Trial Outcomes & Findings for Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID (NCT NCT02999984)

Last Updated: 2022-08-03

Results Overview

Efficacy of OTL-101 treatment at 6 months post OTL-101 infusion based on the following parameters and thresholds: 1. ADA enzyme activity in erythrocytes above baseline/pretreatment level (i.e., \>0 units). ADA enzyme activity is measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV. 2. Absolute CD3+ T cell counts ≥200 cells/μL. Increase in CD3+ T cell counts is a marker of immune reconstitution. 3. Granulocyte samples positive for vector sequences by quantitative Polymerase Chain Reaction (PCR) (≥1/10,000 cells). Vector copy number (VCN) in the Peripheral Blood (PB) Granulocytes fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

10 participants

Primary outcome timeframe

6 months

Results posted on

2022-08-03

Participant Flow

Participant milestones

Participant milestones
Measure	Gene Therapy Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Overall Study STARTED	10
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Gene Therapy Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Overall Study Treatment Failure	1

Baseline Characteristics

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Gene Therapy n=10 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Age, Categorical <=18 years	10 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	6 Participants n=5 Participants
Race/Ethnicity, Customized Afro-American	2 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	1 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: The efficacy population was a modified intent-to-treat population and consisted of all evaluable subjects at 6-month post-treatment with OTL-101. For outcome measure "% of subjects with CD3+ T-cell count \>=200 cells/μL", only 9 subjects were evaluable as data was not available for 1 subject.

Outcome measures

Outcome measures
Measure	Gene Therapy n=10 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months) % of subjects with an increase from baseline in RBC ADA activity	100 percentage of participants
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months) % of subjects with CD3+ T-cell count >=200 cells/μL	100 percentage of participants
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months) % of subjects with detectable gene-marked granulocytes by quantitative PCR >=1/10,000 cells	90 percentage of participants

PRIMARY outcome

Timeframe: 12 Months

Population: The efficacy population was a modified intent-to-treat population and consisted of all subjects treated with OTL-101 within this study.

Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with cryopreserved OTL-101.

Outcome measures

Outcome measures
Measure	Gene Therapy n=9 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	100 percentage of participants Interval 66.37 to 100.0

PRIMARY outcome

Timeframe: 12 Months

Population: The efficacy population was a modified intent-to-treat population and consisted of all subjects treated with OTL-101 within this study.

Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death.

Outcome measures

Outcome measures
Measure	Gene Therapy n=10 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) Percentage event-free at 12 months	90.00 percentage of participants Interval 55.5 to 99.75
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) Percentage without reinstitution of PEG-ADA by 12 months	90.00 percentage of participants Interval 55.5 to 99.75
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) Percentage without rescue HSCT by 12 months	100 percentage of participants Interval 66.37 to 100.0

SECONDARY outcome

Timeframe: 24 months

OS is defined as the percentage of subjects alive at 24 months post- treatment with cryopreserved OTL-101.

Outcome measures

Outcome measures
Measure	Gene Therapy n=9 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)	100 percentage of participants Interval 66.37 to 100.0

SECONDARY outcome

Timeframe: 24 months

Population: The efficacy population was a modified intent-to-treat population and consisted of all subjects treated with OTL-101 within this study.

Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death.

Outcome measures

Outcome measures
Measure	Gene Therapy n=10 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) Percentage event-free at 24 months	90.00 percentage of participants Interval 55.5 to 99.75
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) Percentage without reinstitution of PEG-ADA by 24 months	90.00 percentage of participants Interval 55.5 to 99.75
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) Percentage without rescue HSCT by 24 months	100 percentage of participants Interval 66.37 to 100.0

SECONDARY outcome

Timeframe: 24 months

Population: The efficacy population was a modified intent-to-treat population and consisted of all subjects treated with OTL-101 within this study, with the exception of 1 subject who was withdrawn prior to Month 24 timepoint and an additional 1 subject for whom CD3+ T cell count data was not available at the Month 24 timepoint.

Immune reconstitution was assessed by change in CD3+ T Cell counts over time.

Outcome measures

Outcome measures
Measure	Gene Therapy n=8 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Change From Baseline in CD3+ T Cell Counts (2 Years)	418.5 cells/μL Interval -332.0 to 1976.0

SECONDARY outcome

Timeframe: 24 months

Population: The efficacy population was a modified intent-to-treat population and consisted of all subjects treated with OTL-101 within this study.

The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning.

Outcome measures

Outcome measures
Measure	Gene Therapy n=10 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Severe Infections Excluding First 3 Months After Treatment	0.12 Infections per person per year

SECONDARY outcome

Timeframe: 24 months

Population: Quality of life data was available for 7/10 subjects at the Month 24 timepoint there only 7 subjects were included in the analysis for this outcome measure.

Assessment of quality of life was measured by the Lansky Performance Status Scale. The maximum score on the Lansky Performance Scale is 100 - the child is fully active and able to carry on normal activity with no special care needed. The minimum score is 10 - the child is completely disabled, not even passive play. The scores at baseline (pre-treatment with OTL-101) and scores at Month 24 post-treatment with OTL-101 were compared to establish if there were any changes in the child's score in this timeframe.

Outcome measures

Outcome measures
Measure	Gene Therapy n=7 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Change From Baseline in Quality of Life Measures (2 Years)	0 Lansky Performance Score Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 24 months

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. Indications for considering the discontinuation of immunoglobulin replacement therapy included: absolute CD4+ \>200, absolute B cell \>100/μl, IgA or IgM \> lower limit of normal for age or gene marking \>1% detectable in B cells.

Outcome measures

Outcome measures
Measure	Gene Therapy n=9 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)	78 percentage of participants

SECONDARY outcome

Timeframe: 24 months

Population: IgRT data was available for 7/10 subjects at the Month 24 timepoint there only 7 subjects were included in the analysis for this outcome measure.

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. For subjects who stopped IgRT during the study, the time of cessation was recorded.

Outcome measures

Outcome measures
Measure	Gene Therapy n=7 Participants Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Time to Cessation of IgRT for Those Who Stopped (2 Years)	11.6 months Interval 9.0 to 20.0

Adverse Events

Gene Therapy

Serious events: 3 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Gene Therapy n=10 participants at risk Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Infections and infestations Bacteraemia	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Bronchiolitis	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Respiratory tract infection viral	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Leukopenia	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Neutropenia	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Metabolism and nutrition disorders Dehydration	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.

Other adverse events

Other adverse events
Measure	Gene Therapy n=10 participants at risk Infusion of autologous cryopreserved CD34+ cells genetically modified by the EF1αS-ADA (EFS-ADA) lentiviral vector (LV) Busulfan: Busulfan is used for non-myeloablative conditioning Polyethylene glycol-modified adenosine deaminase (PEG-ADA): PEG-ADA enzyme replacement therapy (ERT) is discontinued at Day 30 +/- 3 days from date of infusion of OTL-101.
Blood and lymphatic system disorders Leukopenia	100.0% 10/10 • Number of events 19 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Neutropenia	100.0% 10/10 • Number of events 17 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Anaemia	80.0% 8/10 • Number of events 10 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Thrombocytopenia	30.0% 3/10 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Blood and lymphatic system disorders Monocytopenia	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Viral upper respiratory tract infection	40.0% 4/10 • Number of events 5 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Upper respiratory tract infection	30.0% 3/10 • Number of events 5 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Otitis media	30.0% 3/10 • Number of events 4 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Conjunctivitis	10.0% 1/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Gastroenteritis	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Gastroenteritis viral	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations H1N1 influenza	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Hordeolum	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Respiratory syncytial virus infection	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Tinea infection	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Infections and infestations Viral infection	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Dermatitis diaper	60.0% 6/10 • Number of events 9 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Eczema	20.0% 2/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Skin lesion	10.0% 1/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Erythema	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Miliaria	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Rash	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Skin and subcutaneous tissue disorders Seborrhoeic dermatitis	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Aspartate aminotransferase increased	50.0% 5/10 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Alanine aminotransferase increased	50.0% 5/10 • Number of events 6 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Human rhinovirus test positive	20.0% 2/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Blood pressure increased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Haematocrit decreased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Mean cell haemoglobin concentration decreased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Mean cell haemoglobin decreased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Investigations Mean cell volume decreased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Respiratory, thoracic and mediastinal disorders Cough	50.0% 5/10 • Number of events 8 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	30.0% 3/10 • Number of events 6 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Respiratory, thoracic and mediastinal disorders Nasal congestion	20.0% 2/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Respiratory, thoracic and mediastinal disorders Asthma	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Gastrointestinal disorders Diarrhoea	20.0% 2/10 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Gastrointestinal disorders Vomiting	20.0% 2/10 • Number of events 3 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Gastrointestinal disorders Constipation	20.0% 2/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Gastrointestinal disorders Nausea	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Gastrointestinal disorders Retching	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
General disorders Pyrexia	20.0% 2/10 • Number of events 2 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
General disorders Gait disturbance	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Eye disorders Eye discharge	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Eye disorders Eye pruritus	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Eye disorders Lacrimation increased	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Injury, poisoning and procedural complications Fall	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Injury, poisoning and procedural complications Skin abrasion	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Ear and labyrinth disorders Cerumen impaction	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Surgical and medical procedures Sinus operation	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.
Vascular disorders Hypertension	10.0% 1/10 • Number of events 1 • Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 2-years post-treatment with OTL-101. The safety population consisted of all subjects treated with OTL-101 within this study.

Additional Information

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Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place