Trial Outcomes & Findings for Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease (NCT NCT03454893)

NCT ID: NCT03454893

Last Updated: 2024-01-05

Results Overview

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. The AE/SAE are also inclusive of any abnormalities in Clinical Laboratory Tests, Vital Signs and in Electrocardiographs (ECGs). Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 15 participants
• Primary outcome timeframe: Baseline to Week 48 post gene therapy
• Results posted on: 2024-01-05

Participant Flow

Total number of subjects who signed the ICF was 16 (one subject who re-screened was not considered enrolled initially), as a result there were 15 distinct subjects who signed ICF. There were 4 participants who failed screening and were not rescreened. The Safety population consists of all 11 enrolled subjects who received any preparatory medication. The Infused population consists of all 9 enrolled subjects who received all preparatory medications and AVR-RD-01.

Participant milestones

Measure	Single Assignment AVR-RD-01 AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg
Overall Study STARTED	15
Overall Study Safety Population	11
Overall Study Infused Population	9
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Measure	Single Assignment AVR-RD-01 AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg
Overall Study Study Terminated by the Sponsor	5
Overall Study Did not pass screening	4

Baseline Characteristics

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Baseline characteristics by cohort

Measure	Single Assignment AVR-RD-01 n=11 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Age, Continuous	31.45 years STANDARD_DEVIATION 10.98 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Safety population consists of all enrolled subjects who received any preparatory medication.

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. The AE/SAE are also inclusive of any abnormalities in Clinical Laboratory Tests, Vital Signs and in Electrocardiographs (ECGs). Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=11 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) AE	354 Number of events
Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) SAE	13 Number of events

PRIMARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The infused population includes all enrolled subjects who received preparatory medications and AVR-RD-01. Four tested positive for anti-AGA antibodies post-AVR-RD-01 infusion at Week 48 or Early Termination visit.

Number of subjects with changes in anti-AGA antibodies from Baseline to post infusion timepoints. Unite of measure: Number of subjects negative at baseline but positive at post-treatment timepoints. A negative or zero result (titer lower or unchanged at post-infusion timepoints compare to Baseline) indicates no immune response to the therapeutic protein.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Immunogenicity of AVR-RD-01	4 Number of positive subjects

PRIMARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

The "Presence of RCL" is a theoretical risk of lentiviral gene therapy treatment based on the theory that it may be possible for inadvertent generation of RCL caused either by recombination of the lentiviral vector plasmids during the vector production process or by mobilization of proviral DNA in vivo by infectious retroviruses (HIV). The absence of RCL is a positive indicator of safety.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Presence of Replication Competent Lentivirus (RCL)	0 Number of positive subjects

PRIMARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Integration Site Analysis (ISA) uses next generation sequencing to identify junction sites between the integrated therapeutic transgene and the host genome. Samples are analyzed for the emergence of clonality (defined as (a single clone accounting for greater than 20% of the population) and whether any integration site is within or near a known oncogene.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Evaluation of Aberrant Clonal Expansion	0 number of abnormal clonal proliferations

PRIMARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. Of the nine subjects, only 2 subjects had baseline and Week 48 results to measure change from baseline. The remaining seven subjects either did not provide both samples or the samples provided were not evaluable.

Globotriaosylceramide (Gb3) Inclusions in Peritubular Capillaries (PTC) on Kidney Biopsy. Electron microscopic images of kidney biopsy samples were taken and read centrally by two independent renal pathologists, each of whom scored the average number of Gb3 inclusions per kidney PTC per subject using a quantification method. Healthy renal tissue would have no Gb3 inclusions. A reduction from baseline is desirable.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=2 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in the Average Number of Gb3 Inclusions (ie, Myelinosomes) Per Kidney Peritubular Capillary (PTC) Per Subject	-3.555 Number of inclusions Interval -4.02 to -3.09

SECONDARY outcome

Timeframe: At Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Vector Copy Number (VCN) is a measurement of the number of copies of the therapeutic transgene found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one [working] copy of the therapeutic transgene per cell.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR) Week 24	0.383 copies/diploid genome Standard Deviation 0.4628
Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR) Week 48	0.313 copies/diploid genome Standard Deviation 0.3447

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Treatment-naïve Fabry patients are deficient in alpha-galactosidase A (AGA) enzyme activity due to mutations in the GLA gene. Any therapeutic option offered should aim to increase the amount of available AGA enzyme. This assay measured the AGA enzyme activity levels in plasma and PBLs. It should be noted that the measurement in plasma reflects the amount of "free" AGA enzyme that has been released from cells into the extracellular space and is therefore considered a more indirect measure of AGA enzyme activity, compared to the result in PBLs which is more of a direct measure of enzyme within cells. In both cases, enzyme activity is expected to increase from Baseline to the post-infusion timepoints.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs) CFB Plasma W48	1.548 nmol/h/mg Standard Deviation 2.152
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs) CFB Plasma W24	1.973 nmol/h/mg Standard Deviation 2.585
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs) CFB PBL Week 24	29.524 nmol/h/mg Standard Deviation 39.194
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs) CFB PBL Week 48	18.973 nmol/h/mg Standard Deviation 32.800

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in plasma. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of circulating Gb3 in plasma.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Plasma Week 24	-3334.0 nM Standard Deviation 3846.8
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Plasma Week 48	-5067.8 nM Standard Deviation 4067.0

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in urine. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of excreted Gb3 in urine.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Urine Week 24	-11.73 nmol/mmol creatinine Standard Deviation 309.78
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Urine Week 48	-188.73 nmol/mmol creatinine Standard Deviation 262.90

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in tissue samples. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of measured Gb3 in tissues.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in superficial dermal capillary endothelial cells (Week 24)	-0.4 number of myelinosomes Standard Deviation 1.3
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in superficial dermal capillary endothelial cells (Week 48)	-0.5 number of myelinosomes Standard Deviation 1.0
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in deep dermal capillary endothelial cells (Week 24)	0 number of myelinosomes Standard Deviation 1.3
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in deep dermal capillary endothelial cells (Week 48)	-0.5 number of myelinosomes Standard Deviation 1.0
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in vascular smooth muscle cells (Week 24)	0.1 number of myelinosomes Standard Deviation 1.2
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in vascular smooth muscle cells (Week 48)	-0.5 number of myelinosomes Standard Deviation 1.0
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in perineurium (Week 24)	0 number of myelinosomes Standard Deviation 1.4
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy Myelinosomes in perineurium (Week 48)	-0.5 number of myelinosomes Standard Deviation 1.0

SECONDARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

mGFR is a measure of the time the kidney takes to filter products that the body does not naturally produce.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Renal Function as Assessed by Measured Glomerular Filtration Rate (mGFR)	-2.358 mL/min/1.73m^2 Standard Deviation 14.502

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

eGFR is the measure to evaluate kidney function. It is the estimated amount of blood that is filtered through all glomeruli in a given time.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Renal Function as Assessed by Estimated Glomerular Filtration Rate (eGFR) Week 24	-6.386 mL/min/1.73 m^2 Standard Deviation 9.699
Change From Baseline in Renal Function as Assessed by Estimated Glomerular Filtration Rate (eGFR) Week 48	-3.561 mL/min/1.73 m^2 Standard Deviation 7.332

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. No data were collected for any subjects since samples were not analyzed in order to produce data (Refer to "Limitations and Caveat" section.")

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

A healthy kidney only allows very small amounts of albumin to pass from the blood into the urine. An increased level of albumin in urine (albuminuria) is a marker of renal damage.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Renal Function as Assessed by Urine Albumin Levels Week 24	0.915 mg/dL Standard Deviation 6.704
Change From Baseline in Renal Function as Assessed by Urine Albumin Levels Week 48	0.643 mg/dL Standard Deviation 5.789

SECONDARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

LVMI is a surrogate of left ventricular hypertrophy. An increase in LVMI is an independent risk factor for cardiovascular morbidity and mortality.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Left Ventricular Mass Index (LVMI) as Assessed by Cardiac Magnetic Resonance Imaging (MRI)	7.270 g/m^2 Standard Deviation 6.055

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

DIBSS-D assesses bowel habits and abdominal symptoms over a period of time. It was administered daily for 14 days commencing at each study visit.

Stool Consistency scale has been converted to a numeric rating scale for ease of analysis, where 1=Very hard; 2=Hard; 3=Neither too hard nor too soft; 4=Loose but not lumpy; 5=Very loose and watery. The median of each 14-day period was derived per patient, per visit before deriving the group median. Group median at Baseline (pre-treatment) was 3.540 (n=9). Change from baseline (CFB) is presented below. An increase CFB indicates softening of the stools, and a decrease CFB indicates hardening of the stools. Abdominal Pain measure asked the patient to rate the worst level of pain within the past 24hrs (0=no pain; 10=worst possible pain). A mean score was derived for each 14-day period per patient, per visit, and these means used to derive a group mean. An increase CFB indicates more abdominal pain; a decrease CFB indicates less abdominal pain.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D) Abdominal Pain Week 24	-0.445 units on a scale Standard Deviation 0.583
Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D) Abdominal Pain Week 48	-0.497 units on a scale Standard Deviation 0.626
Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D) Stool Consistency Week 24	-0.373 units on a scale Standard Deviation 0.216
Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D) Stool Consistency Week 48	-0.530 units on a scale Standard Deviation 0.104

SECONDARY outcome

Timeframe: Baseline to Week 24 and Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.

The short version of the BPI (Short form) includes 9 items: Q1 - Q9, Question 9 includes 7 sub-items (Q9a - Q9g). It uses a 0 to 10 numeric rating scales for item rating. The pain severity score is calculated as the average of questions answered: Q3 (worst pain), Q4 (least pain), Q5 (average pain) and Q6 (current pain). The pain interference score is calculated as the average of the answered Q9 sub-items, which represents pain interference with general activity (Q9a), mood (Q9b), walking ability (Q9c), normal work (Q9d), relations with other people (Q9e), sleep (Q9f), and enjoyment of life (Q9g). A reduction in score from baseline indicates less pain.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores Week 48 (pain interference)	-0.573 score on a scale Standard Deviation 0.996
Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores Week 24 (pain severity)	-0.188 score on a scale Standard Deviation 0.874
Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores Week 48 (pain severity)	-0.500 score on a scale Standard Deviation 0.612
Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores Week 24 (pain interference)	0.073 score on a scale Standard Deviation 1.007

SECONDARY outcome

Timeframe: Baseline to Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. For Week 24 data, please see comment in "Limitations and Caveats" section.

The original version of the SF-36 was administered to the participants and consisted of eight subscales (Vitality, Physical Functioning, Bodily Pain, General Health Perceptions, Physical Role Functioning, Emotional Role Functioning, Social Role Functioning and Mental Health) each scored from 0 (worst health) to 100 (best heath). These scores were normalized (re-scaled) against mean scores obtained in the US general population (Mean=50, Standard deviation 10). The summary health components PCS and MCS are derived from the eight subscales mentioned above and summarize information from all eight subscales but with different weights. For PCS, highest weights are given to the physical subscales while some mental subscales are given negative weights. For MCS, highest weights are given to the mental subscales while some physical subscales are given negative weights. An increase in the normalized score from baseline indicates improvement in physical and mental functioning.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=9 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Change From Baseline in Physical and Mental Functioning as Assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores Physical Functioning Week 48	3.235 score on a scale Standard Deviation 4.810
Change From Baseline in Physical and Mental Functioning as Assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores Mental Functioning Week 48	-1.580 score on a scale Standard Deviation 4.652

SECONDARY outcome

Timeframe: At Week 48 post gene therapy

Population: The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. No analysis performed (refer to "Limitations and Caveats" section)

VCN is defined as the average number of copies of the therapeutic gene (transgene) in a sample of cells and is a measurement of the number of copies of the vector found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one [working] copy of the therapeutic transgene per cell. This measurement was for VCN in a sample of Bone marrow progenitor cells obtained from an aspirate.

Outcome measures

Measure	Single Assignment AVR-RD-01 n=1 Participants AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Average Vector Copy Number (VCN) in Bone Marrow / Progenitor Cells as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)	1.14 copies/diploid genome Interval 1.14 to 1.14

Adverse Events

Single Assignment AVR-RD-01

Serious events: 8 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	Single Assignment AVR-RD-01 n=11 participants at risk AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Blood and lymphatic system disorders Febrile neutropenia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Thrombocytopenia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Odynophagia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Vomiting	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Mucosal inflammation	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Pyrexia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Hepatobiliary disorders Cholelithiasis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Dehydration	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Seizure	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Choking	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Lung infiltration	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.

Other adverse events

Measure	Single Assignment AVR-RD-01 n=11 participants at risk AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human. Drug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.
Blood and lymphatic system disorders Anaemia	27.3% 3/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Febrile neutropenia	72.7% 8/11 • Number of events 8 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Leukopenia	81.8% 9/11 • Number of events 11 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Lymphopenia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Neutropenia	81.8% 9/11 • Number of events 14 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Blood and lymphatic system disorders Thrombocytopenia	81.8% 9/11 • Number of events 12 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Cardiac disorders Sinus tachycardia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Cardiac disorders Tachycardia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Congenital, familial and genetic disorders Fabry's disease	9.1% 1/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Ear and labyrinth disorders Ear congestion	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Endocrine disorders Primary hypogonadism	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Eye disorders Dry eye	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Abdominal distension	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Abdominal pain	45.5% 5/11 • Number of events 7 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Anal fissure	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Constipation	45.5% 5/11 • Number of events 6 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Dental caries	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Diarrhoea	72.7% 8/11 • Number of events 9 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Dry mouth	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Dysphagia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Gastrooesophageal reflux disease	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Haematemesis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Haemorrhoids	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Hiatus hernia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Nausea	90.9% 10/11 • Number of events 15 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Odynophagia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Stomatitis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Tooth impacted	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Gastrointestinal disorders Vomiting	54.5% 6/11 • Number of events 10 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Face oedema	9.1% 1/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Fatigue	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Feeling abnormal	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Influenza like illness	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Injection site induration	9.1% 1/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Injection site pain	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Mucosal inflammation	45.5% 5/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Nodule	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Oedema	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Oedema peripheral	36.4% 4/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Pyrexia	45.5% 5/11 • Number of events 6 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
General disorders Vaccination site pain	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Hepatobiliary disorders Cholelithiasis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Hepatobiliary disorders Hepatic steatosis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Hepatobiliary disorders Hypertransaminasaemia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Immune system disorders Immunisation reaction	9.1% 1/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Escherichia urinary tract infection	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Folliculitis	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Gingivitis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Nasopharyngitis	9.1% 1/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Post-acute COVID-19 syndrome	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Postoperative wound infection	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Rhinitis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Tinea cruris	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Tooth infection	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Upper respiratory tract infection	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Urinary tract infection bacterial	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Infections and infestations Vascular access site infection	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Citrate toxicity	54.5% 6/11 • Number of events 7 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Meniscus cyst	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Post procedural constipation	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Procedural pain	36.4% 4/11 • Number of events 9 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Tooth fracture	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Tooth injury	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Traumatic haematoma	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Vascular access site erythema	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Vascular access site haemorrhage	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Injury, poisoning and procedural complications Vascular access site pain	36.4% 4/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Alanine aminotransferase increased	9.1% 1/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Aspartate aminotransferase increased	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Blood alkaline phosphatase increased	18.2% 2/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Blood follicle stimulating hormone increased	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Blood lactate dehydrogenase increased	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Blood phosphorus increased	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Blood potassium decreased	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations CD4 lymphocytes decreased	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Cardiac murmur	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Gamma-glutamyltransferase increased	9.1% 1/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Investigations Urine analysis abnormal	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Abnormal loss of weight	36.4% 4/11 • Number of events 4 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Abnormal weight gain	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Decreased appetite	36.4% 4/11 • Number of events 4 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Dehydration	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hyperlipidaemia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hypocalcaemia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hypokalaemia	54.5% 6/11 • Number of events 7 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hypomagnesaemia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hyponatraemia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hypophosphataemia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Hypovolaemia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Metabolism and nutrition disorders Vitamin D deficiency	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Arthralgia	27.3% 3/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Back pain	27.3% 3/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Bone pain	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Joint swelling	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Muscle spasms	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Myalgia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Neck pain	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Musculoskeletal and connective tissue disorders Pain in extremity	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Dizziness	27.3% 3/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Dysgeusia	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Headache	72.7% 8/11 • Number of events 9 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Lethargy	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Neuralgia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Parosmia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Nervous system disorders Seizure	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Psychiatric disorders Anxiety	18.2% 2/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Psychiatric disorders Depressed mood	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Psychiatric disorders Insomnia	45.5% 5/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Renal and urinary disorders Acute kidney injury	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Renal and urinary disorders Dysuria	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Renal and urinary disorders Haematuria	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Renal and urinary disorders Renal haematoma	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Renal and urinary disorders Urinary hesitation	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Azoospermia	45.5% 5/11 • Number of events 5 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Erectile dysfunction	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Gynaecomastia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Oligospermia	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Scrotal discomfort	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Reproductive system and breast disorders Scrotal erythema	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Choking	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Cough	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Dry throat	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Epistaxis	9.1% 1/11 • Number of events 3 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Hiccups	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Increased viscosity of upper respiratory secretion	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Lung infiltration	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Painful respiration	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Pharyngeal inflammation	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Alopecia	72.7% 8/11 • Number of events 8 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Dermatosis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Dry skin	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Hair growth abnormal	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Hyperhidrosis	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Lichen planus	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Nail ridging	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Neurodermatitis	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Pain of skin	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Pigmentation disorder	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Rash	18.2% 2/11 • Number of events 2 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Skin and subcutaneous tissue disorders Skin plaque	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Vascular disorders Flushing	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Vascular disorders Hypotension	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.
Vascular disorders Orthostatic hypotension	9.1% 1/11 • Number of events 1 • Up to week 48 Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.

Additional Information

AVROBIO MedInfo

AVROBIO, Inc

Phone: 617-914-8419

Email: medinfo@avrobio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place