Trial Outcomes & Findings for A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease (NCT NCT02151526)
NCT ID: NCT02151526
Last Updated: 2022-04-26
Results Overview
Neutrophil engraftment was defined as the first of absolute neutrophil count (ANC) \> or = 0.5 × 10\^9/ liter (L) for 3 consecutive days (or 3 consecutive measurements done on separate days), after a post-transplant value less than (\<) 0.5 × 10\^9/L. Platelet engraftment was defined as the first of 3 consecutive platelet values \> or =20 × 10\^9/L for participants with TDT and values \> or =50 × 10\^9/L for participants with SCD obtained on different days with no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. The day of engraftment is the first day of the 3 consecutive platelet measurements.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
7 participants
Primary outcome timeframe
From time of drug product infusion through Month 24
Results posted on
2022-04-26
Participant Flow
This study was conducted at a single site in France between 07 June 2013 (First participant signed informed consent) and 26 February 2019 (Last participant last visit).
A total of 7 participants were treated in a single arm and completed the study. Data was planned, analyzed and reported separately for 3 participants with Sickle Cell Disease (SCD) and 4 participants with transfusion-dependent β-thalassemia (TDT).
Participant milestones
| Measure |
LentiGlobin BB305 Drug Product for SCD
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
4
|
|
Overall Study
COMPLETED
|
3
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
Baseline characteristics by cohort
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
Total
n=7 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From time of drug product infusion through Month 24Population: Transplant Population (TP) included all participants in the Intent to treat population who underwent LentiGlobin BB305 Drug Product infusion.
Neutrophil engraftment was defined as the first of absolute neutrophil count (ANC) \> or = 0.5 × 10\^9/ liter (L) for 3 consecutive days (or 3 consecutive measurements done on separate days), after a post-transplant value less than (\<) 0.5 × 10\^9/L. Platelet engraftment was defined as the first of 3 consecutive platelet values \> or =20 × 10\^9/L for participants with TDT and values \> or =50 × 10\^9/L for participants with SCD obtained on different days with no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. The day of engraftment is the first day of the 3 consecutive platelet measurements.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Number of Treated Participants With Successful Neutrophil and Platelet Engraftment
Participants with Neutrophil Engraftment
|
3 Participants
|
4 Participants
|
|
Number of Treated Participants With Successful Neutrophil and Platelet Engraftment
Participants with Platelet Engraftment
|
3 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
Neutrophil engraftment was defined as the first of ANC \> or = 0.5 × 10\^9/ liter (L) for 3 consecutive days (or 3 consecutive measurements done on separate days), after a post-transplant value \< 0.5 × 10\^9/L). Platelet engraftment was defined as the first of 3 consecutive platelet values \> or =20 × 10\^9/L for participants with TDT and values \> or =50 × 10\^9/L for participants with SCD obtained on different days with no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. The day of engraftment is the first day of the 3 consecutive platelet measurements.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Time to Successful Neutrophil and Platelet Engraftment
Time to Neutrophil Engraftment
|
32 Days
Interval 27.0 to 38.0
|
16.5 Days
Interval 14.0 to 29.0
|
|
Time to Successful Neutrophil and Platelet Engraftment
Time to Platelet Engraftment
|
51 Days
Interval 39.0 to 92.0
|
23 Days
Interval 20.0 to 26.0
|
PRIMARY outcome
Timeframe: From screening through 365 days post-transplantPopulation: This outcome measure was evaluated in the Transplant Population (TP).
This was the safety outcome measure related to mortality. Transplant related mortality was defined as any death occurring in the study post drug product infusion deemed related to the transplant by the investigator.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
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Incidence of Transplant Related Mortality
|
0 Number of deaths reported
|
0 Number of deaths reported
|
PRIMARY outcome
Timeframe: From time of drug product infusion through Month 24Population: Intent to Treat (ITT) population consisted of all participants who initiated any study procedures, beginning with mobilization (by Granulocyte colony stimulating factor \[G-CSF\] with or without plerixafor), or bone marrow harvest.
Overall survival was defined as time from date of LentiGlobin BB305 Drug Product infusion (Day 1) to date of death. Overall survival was censored at the date of last visit if the participant was still alive. Number of participants with OS events were reported.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Number of Participants With Overall Survival (OS) Events
Censored Participants
|
3 Participants
|
4 Participants
|
|
Number of Participants With Overall Survival (OS) Events
Participants reported OS events
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in the ITT population.
Blood samples were analyzed for detection of RCL using RCL co-culture assay.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Percentage of Participants With Vector-Derived Replication-Competent Lentivirus (RCL)
|
0 Percentage of participants
|
0 Percentage of participants
|
PRIMARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
Clonal dominance was defined as an ISA result greater than (\>) 90% of the total insertion sites (IS) at any time and a vector copy number (VCN) \> or =0.3, or an initial ISA result of \> 30% of the total IS with a VCN \> or =0.3 followed by a result \> 30% and less than or equal to (\< or =) 90% at first repeat and a result \> 50% at second repeat.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Number of Treated Participants With Greater Than (>) 30 Percent (%) Contribution of an Individual Clone As Per Integration Site Analysis (ISA)
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: From date of Informed Consent signing up to Month 24Population: This outcome measure was evaluated in the ITT population.
An AE was any untoward medical occurrence associated with the use of a drug in participants, whether or not considered drug related. An AE may include a change in physical signs, symptoms, and/or clinically significant laboratory change occurring in any phase of a clinical study. This definition includes inter-current illnesses or injuries, and exacerbation of pre-existing conditions. An SAE was any AE, occurring at any dose and regardless of causality, that resulted in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or was considered an important medical event that may jeopardize the participant and may require medical or surgical intervention to prevent an outcome listed previously. The number of participants with AEs and SAEs were evaluated.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number of Participants with any Adverse Event
|
3 Participants
|
4 Participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number of participants with any SAE
|
3 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT.
TI was defined as a weighted average hemoglobin (Hb) \> or =9 grams per deciliter (g/dL) without any pRBC transfusions for a continuous period of \> or =12 months at any time during the study after drug product infusion, where calculation of time period of TI starts when participants achieve a Hb \> or =9 g/dL with no transfusions in the preceding 60 days.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Percentage of Treated Participants With Transfusion-Dependent β-Thalassemia (TDT) Who Achieved Transfusion Independence (TI)
|
75 Percentage of participants
|
—
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT. The number of participants analyzed signifies participants who were evaluable for this specific outcome measure.
TI was defined as a weighted average hemoglobin (Hb) \> or =9 grams per deciliter (g/dL) without any pRBC transfusions for a continuous period of \> or =12 months at any time during the study after drug product infusion, where calculation of time period of TI starts when participants achieve a Hb \> or =9 g/dL with no transfusions in the preceding 60 days.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Weighted Average Hemoglobin (Hb) During Period of Transfusion Independence (TI) in Participants With Transfusion-Dependent β-Thalassemia (TDT)
|
11.3 grams per deciliter (g/dL)
Interval 10.6 to 13.1
|
—
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT. The number of participants analyzed signifies participants who were evaluable for this specific outcome measure.
TI was defined as a weighted average Hb \> or =9 grams per deciliter (g/dL) without any pRBC transfusions for a continuous period of \> or =12 months at any time during the study after drug product infusion, where calculation of time period of TI starts when participants achieve a Hb \> or =9 g/dL with no transfusions in the preceding 60 days. To meet the initial TI criteria, the weighted Hb must be \> or =9 g/dL at the end of the 12-month period, to remain in the TI state beyond the 12-month period, the treated participant needs to maintain a weighted Hb of \> or =9 g/dL from that point forward, without receiving a pRBC transfusion. This outcome measure reports the duration of TI and was evaluated in the TDT Transplant Population (TP) that reached TI.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Duration of Transfusion Independence (TI) in Participants With Transfusion-Dependent β-Thalassemia (TDT)
|
21.7 Month
Interval 21.2 to 21.8
|
—
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT. The number of participants analyzed signifies participants who were evaluable for this specific outcome measure.
TI was defined as a weighted average Hb \> or =9 grams per deciliter (g/dL) without any pRBC transfusions for a continuous period of \> or =12 months at any time during the study after drug product infusion, where calculation of time period of TI starts when participants achieve a Hb \> or =9 g/dL with no transfusions in the preceding 60 days. To meet the initial TI criteria, the weighted Hb must be \> or =9 g/dL at the end of the 12-month period, to remain in the TI state beyond the 12-month period, the treated participant needs to maintain a weighted Hb of \> or =9 g/dL from that point forward, without receiving a pRBC transfusion. This endpoint reports the time from infusion to the last pRBC transfusion prior to achieving TI.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Time From LentiGlobin BB305 Drug Product Infusion to Last Packed Red Blood Cells (pRBC) Transfusion Prior to Achieving Transfusion Independence (TI) in Participants With Transfusion-Dependent β-Thalassemia (TDT)
|
11 Days
Interval 5.0 to 13.0
|
—
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT. The number of participants analyzed signifies participants who were evaluable for this specific outcome measure.
TI was defined as a weighted average Hb \> or =9 grams per deciliter (g/dL) without any pRBC transfusions for a continuous period of \> or =12 months at any time during the study after drug product infusion, where calculation of time period of TI starts when participants achieve a Hb \> or =9 g/dL with no transfusions in the preceding 60 days. To meet the initial TI criteria, the weighted Hb must be \> or =9 g/dL at the end of the 12-month period, to remain in the TI state beyond the 12-month period, the treated participant needs to maintain a weighted Hb of \> or =9 g/dL from that point forward, without receiving a pRBC transfusion. This outcome measure reports the time from infusion to achievement of TI.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Time From LentiGlobin BB305 Drug Product Infusion to Achieving Transfusion Independence (TI) in Participants With Transfusion-Dependent β-Thalassemia (TDT)
|
14.9 Month
Interval 14.9 to 15.6
|
—
|
SECONDARY outcome
Timeframe: From 6 months post-drug product infusion through Month 24Population: This outcome measure was evaluated only in Transplant Population (TP) with TDT.
Weighted average Hb nadir was defined as an average area under the curve where the Hb closest but within 3 days prior to a transfusion was used as the Hb nadir. Hb values on the day of the transfusion were considered for nadir calculations.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Weighted Average Nadir Hemoglobin (Hb) in Participants With Transfusion-Dependent β-Thalassemia (TDT)
|
11 grams per deciliter (g/dL)
Interval 8.5 to 13.2
|
—
|
SECONDARY outcome
Timeframe: Baseline, From 6 months post-drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
Percent change from baseline in the average annual transfusion volume from 6 months post-drug product infusion through last visit were reported.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Percentage Change From Baseline in Annualized Packed Red Blood Cell (pRBC) Transfusion Volume
|
-100.0 percent change in Annualized pRBC volume
Interval -100.0 to 90.0
|
-100.0 percent change in Annualized pRBC volume
Interval -100.0 to -100.0
|
SECONDARY outcome
Timeframe: Baseline, From 6 months post-drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
Percentage change from baseline in annualized number of pRBC transfusions from 6 months post-drug product infusion through last visit were reported.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Percentage Change From Baseline in Annualized Number of Packed Red Blood Cell (pRBC) Transfusions
|
-100.0 percentage change in pRBC transfusion
Interval -100.0 to -1.9
|
-100.0 percentage change in pRBC transfusion
Interval -100.0 to -100.0
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in ITT population. This outcome measure was only planned to evaluate in SCD participants. Events of VOC and ACS were from same participant.
Number of VOCs, ACS, and vaso-occlusive events (VOEs; which included both VOC and ACS) through 24 months after drug product infusion.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Number of Participants With Vaso-Occlusive Crisis (VOC) and/or Acute Chest Syndrome (ACS) Events Post Drug Product Infusion in Sickle Cell Disease Participants
Vaso-Occlusive Crisis (VOC)
|
1 Participants
|
—
|
|
Number of Participants With Vaso-Occlusive Crisis (VOC) and/or Acute Chest Syndrome (ACS) Events Post Drug Product Infusion in Sickle Cell Disease Participants
Acute Chest Syndrome (ACS)
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
Therapeutic globin expression was measured by HbA\^T87Q in peripheral blood and the ratio of alpha(α)- globin to all beta (β)-like-globins. The relative amount of each globin produced by a participant (including βA\^A-T87Q globin) was determined in peripheral blood throughout the study.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Therapeutic Globin Expression Measured by Hb Containing β^A -T87Q Globin (HbA^T87Q) in Peripheral Blood
|
2.947 grams per deciliter (g/dL)
Standard Deviation 2.4415
|
8.287 grams per deciliter (g/dL)
Standard Deviation 1.5758
|
SECONDARY outcome
Timeframe: From time of drug product infusion through Month 24Population: This outcome measure was evaluated in the Transplant Population (TP).
LentiGlobin BB305 lentiviral vector (LVV) transduction efficiency was measured by VCN. The presence of vector sequences in the genomic DNA of cells is detected using quantitative polymerase chain reaction (qPCR), and results were expressed as VCN.
Outcome measures
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 Participants
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Vector Copy Number (VCN) in Peripheral Blood
|
0.898 copies per diploid genome (c/dg)
Standard Deviation 1.1655
|
1.796 copies per diploid genome (c/dg)
Standard Deviation 1.3665
|
Adverse Events
LentiGlobin BB305 Drug Product for SCD
Serious events: 3 serious events
Other events: 3 other events
Deaths: 0 deaths
LentiGlobin BB305 Drug Product for TDT
Serious events: 3 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 participants at risk
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 participants at risk
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Procedural pain
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Hepatic enzyme increased
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Acute chest syndrome
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Presyncope
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Seizure
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Psychiatric disorders
Major depression
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Hepatobiliary disorders
Cholestasis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Staphylococcal bacteraemia
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Tooth infection
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Pneumonia
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
Other adverse events
| Measure |
LentiGlobin BB305 Drug Product for SCD
n=3 participants at risk
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 milligram per kilogram per day (mg/kg/day) busulfan intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of greater than or equal to (\> or =) 2.0×10\^6 cluster of differentiation (CD) 34+ cells/kg LentiGlobin BB305 Drug Product was administered to participants with SCD by IV infusion.
|
LentiGlobin BB305 Drug Product for TDT
n=4 participants at risk
Following myeloablative conditioning with a dose (dose may be adjusted as per protocol) of 3.2 mg/kg/day busulfan Intravenous (IV) for 4 consecutive days and subsequent daily monitoring of busulfan levels for confirmation of adequate washout, a single dose of \> or =3.0 × 10\^6 CD34+ cells/kg LentiGlobin BB305 Drug Product (betibeglogene autotemcel) was administered to participants with TDT by IV infusion.
|
|---|---|---|
|
Vascular disorders
Hot flush
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Vascular disorders
Hypotension
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Xerosis
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Non-cardiac chest pain
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Puncture site pain
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Pyrexia
|
100.0%
3/3 • Number of events 8 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 5 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Chills
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Catheter site pain
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Fatigue
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Feeling cold
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Hypothermia
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Pain
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
General disorders
Injection site inflammation
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Psychiatric disorders
Anxiety
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Psychiatric disorders
Disturbance in attention
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
Reproductive system and breast disorders
Ovarian cyst
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Reproductive system and breast disorders
Premature menopause
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Injury, poisoning and procedural complications
Procedural pain
|
66.7%
2/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Injury, poisoning and procedural complications
Catheter site pain
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Alanine aminotransferase increased
|
66.7%
2/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
75.0%
3/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Aspartate aminotransferase increased
|
66.7%
2/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
75.0%
3/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Gamma-glutamyltransferase increased
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Staphylococcus test positive
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Investigations
Aspergillus test positive
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Congenital, familial and genetic disorders
Dolichocolon
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive pulmonary disease
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
100.0%
3/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Neutropenia
|
100.0%
3/3 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Anaemia
|
100.0%
3/3 • Number of events 15 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 5 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
66.7%
2/3 • Number of events 7 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Headache
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Presyncope
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Syncope
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Nervous system disorders
Tremor
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Abdominal pain
|
66.7%
2/3 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 6 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Vomiting
|
100.0%
3/3 • Number of events 5 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Nausea
|
100.0%
3/3 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
75.0%
3/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Diarrhoea
|
100.0%
3/3 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
75.0%
3/4 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Stomatitis
|
100.0%
3/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 7 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Anal fissure
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Colitis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Constipation
|
33.3%
1/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Gingival bleeding
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Lip dry
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Odynophagia
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Gastrointestinal disorders
Anal inflammation
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
100.0%
3/3 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
100.0%
4/4 • Number of events 4 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Melanoderma
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
33.3%
1/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
66.7%
2/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
33.3%
1/3 • Number of events 2 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
50.0%
2/4 • Number of events 3 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Folliculitis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Bronchitis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Genital candidiasis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Nasopharyngitis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Rhinitis
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Tooth abscess
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Wound infection fungal
|
33.3%
1/3 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
0.00%
0/4 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Clostridium difficile infection
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Mastitis
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Oral herpes
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Tooth infection
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/3 • From date of Informed Consent signing up to Month 24
|
25.0%
1/4 • Number of events 1 • From date of Informed Consent signing up to Month 24
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60