Trial Outcomes & Findings for A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype (NCT NCT02906202)

NCT ID: NCT02906202

Last Updated: 2023-06-18

Results Overview

TI was defined as a weighted average hemoglobin (Hb) \>= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of \>= 12 months at any time during the study after drug product infusion.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 24 participants
• Primary outcome timeframe: From 14 to 24 months post-transplant
• Results posted on: 2023-06-18

Participant Flow

The study was conducted at 9 centers from 08 August 2016 to 31 March 2022.

A total of 24 participants initiated mobilization procedure, of which 23 participants aged less than or equal to (<=) 50 years were treated with LentiGlobin BB305 Drug Product. As appropriate, data were analyzed at times based on Intent-to-Treat (ITT) population which included all 24 participants who initiated mobilization procedure.

Participant milestones

Measure	LentiGlobin BB305 Drug Product Participants aged \<= 50 years received a single intravenous (IV) infusion of LentiGlobin BB305 Drug Product at a dose of greater than or equal to (\>=) 5.0\*10\^6 CD34 plus (+) cells per kilogram (cells/kg) following myeloablative conditioning with busulfan (termed the Transplant population).
Overall Study STARTED	24
Overall Study Transplant Population	23
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	LentiGlobin BB305 Drug Product Participants aged \<= 50 years received a single intravenous (IV) infusion of LentiGlobin BB305 Drug Product at a dose of greater than or equal to (\>=) 5.0\*10\^6 CD34 plus (+) cells per kilogram (cells/kg) following myeloablative conditioning with busulfan (termed the Transplant population).
Overall Study Discontinued after mobilization due to pregnancy	1

Baseline Characteristics

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Baseline characteristics by cohort

Measure	LentiGlobin BB305 Drug Product n=24 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Age, Continuous	15.0 Years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	14 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: From 14 to 24 months post-transplant

Population: Transplant Population (TP) included all participants who received beti-cel. All participants in the TP (23 participants) were evaluable for TI.

TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Percentage of Participants Who Meet the Definition of Transfusion Independence (TI)	91.3 Percentage of participants Interval 72.0 to 98.9

SECONDARY outcome

Timeframe: At Month 24 post-transplant

Population: TP included all participants who received beti-cel. All participants in the TP (23 participants) were evaluable for TI.

TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion. Percentage of participants who met the definition of TI at Month 24 were evaluated

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Percentage of Participants Who Meet the Definition of Transfusion Independence (TI) at Month 24	91.3 Percentage of participants Interval 72.0 to 98.9

SECONDARY outcome

Timeframe: From start of TI up to Month 24

Population: TP included all participants who received beti-cel. All participants in the TP (23 participants) were evaluable for TI. Here, "Overall number of participants analyzed" signifies those participants who achieved TI (i.e. 21 participants as 2 participants did not achieve TI).

Duration of TI was calculated as the time from the start of TI (that is (i.e.), first Hb >=9 with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met using Kaplan-Meier methodology. Duration of TI from start of TI up to Month 24 months was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=21 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Duration of Transfusion Independence (TI)	20.50 months Interval 18.2 to 22.5

SECONDARY outcome

Timeframe: From 14 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel. All participants in the TP (23 participants) were evaluable for TI. Here, "Overall number of participants analyzed" signifies those participants who achieved TI (i.e. 21 participants as 2 participants did not achieve TI).

Time from drug product infusion to achievement of TI was calculated as the time from drug product infusion to the first hemoglobin at which a participant can be declared as TI (that is to 'start of TI + >= 12 months', dependent on Hb lab schedule).

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=21 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Time From Drug Product Infusion to Achievement of Transfusion Independence (TI)	15.51 months Interval 14.8 to 19.4

SECONDARY outcome

Timeframe: From 60 days after the last pRBC transfusion through Month 24

Population: TP included all participants who received beti-cel. All participants in the TP (23 participants) were evaluable for TI. Here, "Overall number of participants analyzed" signifies those participants who achieved TI (i.e. 21 participants as 2 participants did not achieve TI).

Weighted average Hb was defined as the weighted average of Hb values without any pRBC transfusions in the proceeding 60 days. The ratio of the time between two Hb values and the time between the first and the last Hb values was used as the weight for calculation.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=21 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Weighted Average Hemoglobin (Hb) During Transfusion Independence (TI)	11.473 grams per deciliter (g/dL) Standard Deviation 1.0395

SECONDARY outcome

Timeframe: 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Percentage of participants reduction in the annualized mL/kg pRBCs transfused from 12 months post-drug product infusion through Month 24 (approximately a 12-month period) of at least 50%, 60%, 75%, 90% or 100% compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 50%	95.7 percentage of participants
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 60%	91.3 percentage of participants
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 75%	91.3 percentage of participants
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 90%	91.3 percentage of participants
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at 100%	91.3 percentage of participants

SECONDARY outcome

Timeframe: From 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Annualized number of pRBC transfusions from 12 months post-drug product infusion through Month 24 were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Annualized Number of pRBC Transfusions	0.95 pRBC transfusions per year Standard Deviation 3.195

SECONDARY outcome

Timeframe: From 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Annualized volume of pRBC transfusions from 12 months post-drug product infusion through Month 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Annualized Volume of pRBC Transfusions	11.607 milliliter/kilogram/year (mL/kg/year) Standard Deviation 40.2964

SECONDARY outcome

Timeframe: From start of drug product infusion up to Month 24

Population: TP included all participants who received beti-cel.

Time from drug product infusion to last pRBC transfusion was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Time From Drug Product Infusion to Last pRBC Transfusion	0.953 months Interval 0.46 to 23.98

SECONDARY outcome

Timeframe: From last pRBC Transfusion up to Month 24 (actual maximum time frame of up to approximately 27 months due to visit window)

Population: TP included all participants who received beti-cel. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. Notably due to institutional COVID-19 restrictions and scheduling conflicts one participant had a Month 24 visit 42 days out of this window.

Time From Last pRBC Transfusion to Month 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Time From Last pRBC Transfusion to Month 24	23.228 months Interval 0.07 to 26.64

SECONDARY outcome

Timeframe: 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

The weighted average nadir Hb was defined as the most recent Hb prior to each pRBC transfusion, on the day of transfusion or within 3 days and, if there was a period of more than 60 days without transfusion, all Hb records between Day 61 and last follow-up or next transfusion (inclusive) was included. The weighted average nadir Hb during the period of 12 months post-drug product infusion to Month 24 was compared to the weighted average nadir Hb during the 24 months prior to enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Weighted Average Nadir Hemoglobin (Hb)	9.546 g/dL Standard Deviation 0.7007

SECONDARY outcome

Timeframe: At Month 6, 9, 12, 18 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-207 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint.

Unsupported total Hb level was defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=22 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 6	11.24 g/dL Standard Deviation 1.249
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 9	11.22 g/dL Standard Deviation 1.138
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 12	11.45 g/dL Standard Deviation 1.314
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 18	11.80 g/dL Standard Deviation 1.167
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 24	11.77 g/dL Standard Deviation 1.224

SECONDARY outcome

Timeframe: At Month 6, 9, 12, 18 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-207 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint.

The number of participants with unsupported total Hb levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) meeting the thresholds were reported at at Months 6, 9, 12, 18 and 24. Participants were evaluable if they had an unsupported total Hb measurement at the specific timepoint, where unsupported total Hb level is defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=22 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=10 g/dL)	17 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=11 g/dL)	15 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=12 g/dL)	7 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=13 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=10 g/dL)	20 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=11 g/dL)	14 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=12 g/dL)	5 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=13 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=10 g/dL)	18 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=11 g/dL)	14 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=12 g/dL)	10 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=13 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=10 g/dL)	18 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=11 g/dL)	15 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=12 g/dL)	8 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=13 g/dL)	4 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=10 g/dL)	18 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=11 g/dL)	14 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=12 g/dL)	10 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=13 g/dL)	5 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=14 g/dL)	0 Participants

SECONDARY outcome

Timeframe: Up to Month 24

Population: TP included all participants who received beti-cel.

Percentage of participants who have not received chelation therapy for at least 6 months following drug product infusion were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Percentage of Participants Who Have Not Received Chelation Therapy for At Least 6 Months Following Drug Product Infusion	43.5 percentage of participants

SECONDARY outcome

Timeframe: Time from last iron chelation up to Month 24 (actual maximum time frame of up to approximately 27 months due to visit window)

Population: TP population. Here, "Overall number of participants analyzed" signifies those participants who have not received chelation therapy for at least 6 months following drug product infusion. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. Notably due to institutional COVID-19 restrictions and scheduling conflicts one participant had a Month 24 visit 42 days out of this window.

Time from last iron chelation use to last follow-up to 24 months was reported. Participants were evaluable for this outcome if they had not received iron chelation therapy for at least 6 months following drug product infusion.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=10 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Time From Last Iron Chelation Use to Last Follow-up	23.56 months Interval 17.6 to 24.6

SECONDARY outcome

Timeframe: Up to Month 24

Population: TP included all participants who received beti-cel.

Therapeutic phlebotomy could be used in lieu of chelation in participants who had Hb consistently >= 11 g/dL and who were no longer receiving regular transfusions, at the discretion of the investigator. Percentage of participants who used therapeutic phlebotomy post DP infusion for up to Month 24 were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Percentage of Participants Who Used Therapeutic Phlebotomy Post Drug Product (DP) Infusion	30.4 percentage of participants

SECONDARY outcome

Timeframe: Up to Month 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who received therapeutic phlebotomy.

Annualized phlebotomy therapy usage (number of procedures per year, calculated from DP infusion through last follow-up) were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=7 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Annualized Phlebotomy Therapy Usage Following Drug Product Infusion	6.29 number of procedures per year Standard Deviation 4.786

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Participants were evaluable for this outcome if they had available liver iron content measurements at baseline and at the applicable follow-up timepoints. Here, "number of participants analyzed" signifies those participants who had LIC data available at Baseline, Months 12 and 24.

Change From Baseline in liver Iron Content by Magnetic Resonance Imaging (MRI) at Months 12 and 24 were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=22 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Liver Iron Concentration by Magnetic Resonance Imaging (MRI) At Month 12	2.490 milligrams per gram (mg/g) Standard Deviation 4.1546
Change From Baseline in Liver Iron Concentration by Magnetic Resonance Imaging (MRI) At Month 24	0.494 milligrams per gram (mg/g) Standard Deviation 3.9957

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Participants were evaluable for this outcome if they had available cardiac T2\* measurements at baseline and at the applicable follow-up timepoints. Here, "Overall number of participants analyzed" signifies those participants who had Cardiac T2\* data available at Baseline, Months 12 and 24 and "number analyzed" signifies those participants at specific timepoint.

Change From Baseline in Cardiac T2* on MRI at baseline, Month 12 and 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=21 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Cardiac T2* on MRI At Month 12	-1.0 milliseconds Standard Deviation 5.93
Change From Baseline in Cardiac T2* on MRI At Month 24	-1.6 milliseconds Standard Deviation 7.42

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Participants were evaluable for this endpoint if they had available serum ferritin measurements at baseline and at the applicable follow-up timepoints. Here, "number analyzed" signifies those participants who were evaluable at specific timepoint.

Serum ferritin was commonly used for an indirect estimation of body iron stores. Although sensitive, it is not specific for iron overload as it can be elevated in a variety of infectious and inflammatory states, and in the presence of cytolysis. Change from baseline in serum ferritin at Months 12 and 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=23 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Serum Ferritin at Months 12 and 24 At Month 24	-1163.8 picomole per Liter (pmol/L) Standard Deviation 2435.58
Change From Baseline in Serum Ferritin at Months 12 and 24 At Month 12	167.3 picomole per Liter (pmol/L) Standard Deviation 1887.67

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were \<18 years at specific timepoint.

PedsQL GCS designed to measure health-related quality of life in pediatric and adolescents (2 to 18 years). It encompassed 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), Teens (13-18 years). The questionnaire was also completed by parent/caregiver to assess parents' perceptions of their children's quality of life. The Toddler group consisted of 21 items, using a 5-point Likert scale (0 to 4); all other groups consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for young pediatric, a 5-point Likert scale for pediatric and teens groups. All reported scores were transformed on a scale from 0 to 100 for each domain where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores correspond with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=14 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Parent total score: Change at Month 12	8.76 Score on a scale Standard Deviation 12.071
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Parent total score: Change at Month 24	6.03 Score on a scale Standard Deviation 9.753
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Patient total score: Change at Month 12	6.82 Score on a scale Standard Deviation 16.079
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Patient total score: Change at Month 24	9.96 Score on a scale Standard Deviation 16.997

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were 11 to \<18 years at specific timepoint.

EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life questionnaire in cost effectiveness and health technologies assessment. EQ-5D-Y was a version of instrument specifically developed and validated for use by youths aged 12 through 17 years. The EQ-5D-Y visual analog scale (VAS) consisted of a 20-cm vertical VAS, with anchors of 0 ("worst imaginable health state") and 100 ("best imaginable health state"). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Higher scores equated to better outcomes.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=7 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 Health State: Change at Month 12	15.8 Score on a scale Standard Deviation 20.60
Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 Health State: Change at Month 24	20.9 Score on a scale Standard Deviation 18.86

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this endpoint and number analyzed signifies those participants who were \>= 18 years at specific timepoint.

EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life (HRQoL) questionnaire in cost effectiveness and health technologies assessment. Participants age >=18 at time of informed consent were eligible to complete the EQ-5D-3L which is a visual analog scale (VAS) which consists of a 20-cm vertical VAS, with anchors of 0 ("worst imaginable health state") and 100 ("best imaginable health state"). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they feel most accurately reflects their current health state.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=9 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 Health state: Change at Month 12	6.9 Score on a scale Standard Deviation 11.93
Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 Health state: Change at Month 24	9.7 Score on a scale Standard Deviation 14.91

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who were \>= 18 years for this outcome measure.

FACT-BMT is assessed bone marrow transplant related quality of life in adults. It. Total score was sum of sub-scale scores for 5 domains: Physical Well-Being, Social/Family Well-Being, Emotional Well-Being, Functional Well-Being, and Bone Marrow Transplantation Subscale. Each item scored on a 5-point Likert scale based on participant agreement with each statement: 0 for "not at all," 1 for "a little bit," 2 for "somewhat," 3 for "quite a bit," and 4 for "very much. Reported scores were transformed as follows: After taking into account reverse scores for questions constructed in negative form, subscale score for each domain was calculated by multiplying sum of item scores by number of items in subscale, then dividing by number of items answered. Total score was sum of subscale total added together and ranges from 0-148. Higher scores corresponded with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=9 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Total Score: Change at Month 12	3.78 Score on a scale Standard Deviation 16.994
Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score Total Score: Change at Month 24	2.15 Score on a scale Standard Deviation 13.695

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who were \>= 18 years for this outcome measure.

SF-36 was designed to measure health-related quality of life in adults. The instrument consisted of 36 items, were aggregated into 8 multi-item scales (physical functioning [1=yes, limited a lot to 3=no, not limited at all], role-physical [1=all of time to 5=none of time], bodily pain [1=very severe to 6=none], general health [1=poor to 5=excellent], vitality [1=none of time to 5=all of time], social functioning [1=all of time: to 5=none of time], role emotional [1=all of time to 5=none of time] and mental health [1=all of time to 5=none of the time]). Four domains comprised physical component summary (PCS) score (physical functioning, role-physical, bodily pain, general health) and remaining 4 domains comprised mental component summary (MCS) score (vitality, social functioning, role-emotional, mental health). Reported summary scores were transformed on a scale from 0 to 100. Higher scores corresponded with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=9 Participants Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0\*10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Physical Component: Change at Month 12	0.80 Score on a scale Standard Deviation 4.838
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Physical Component: Change at Month 24	0.82 Score on a scale Standard Deviation 3.672
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Mental Component: Change at Month 12	1.88 Score on a scale Standard Deviation 8.201
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Mental Component: Change at Month 24	0.93 Score on a scale Standard Deviation 10.805

Adverse Events

LentiGlobin BB305 Drug Product

Serious events: 14 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Measure	LentiGlobin BB305 Drug Product n=24 participants at risk Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0 × 10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
Injury, poisoning and procedural complications Contusion	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Femur fracture	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Transfusion reaction	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Vascular disorders Hypotension	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Cardiac disorders Atrial fibrillation	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Epistaxis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Hypoxia	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Febrile neutropenia	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Neutropenia	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Thrombocytopenia	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Pyrexia	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Catheter site haemorrhage	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Stomatitis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Hepatobiliary disorders Venoocclusive liver disease	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Hypokalaemia	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Lower respiratory tract infection	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Neutropenic sepsis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Pneumonia viral	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Sepsis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Appendicitis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Device related sepsis	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Gastroenteritis viral	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Vascular device infection	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Viral infection	4.2% 1/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.

Other adverse events

Measure	LentiGlobin BB305 Drug Product n=24 participants at risk Participants aged \<= 50 years received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>= 5.0 × 10\^6 CD34+ cells/kg following myeloablative conditioning with busulfan (termed the Transplant population).
General disorders Catheter site pain	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Chest pain	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Fatigue	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Mucosal inflammation	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Non-cardiac chest pain	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Pain	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
General disorders Pyrexia	50.0% 12/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Psychiatric disorders Anxiety	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Psychiatric disorders Insomnia	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Ligament sprain	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Procedural pain	45.8% 11/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Skin abrasion	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Injury, poisoning and procedural complications Transfusion reaction	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Activated partial thromboplastin time prolonged	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Alanine aminotransferase increased	37.5% 9/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Aspartate aminotransferase increased	25.0% 6/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Blood alkaline phosphatase increased	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Blood magnesium decreased	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Gamma-glutamyltransferase increased	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Investigations Blood bilirubin increased	20.8% 5/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Cardiac disorders Palpitations	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Bronchospasm	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Cough	41.7% 10/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Dyspnoea	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Epistaxis	41.7% 10/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Hypoxia	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	20.8% 5/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Pharyngeal inflammation	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Respiratory, thoracic and mediastinal disorders Nasal congestion	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Anaemia	70.8% 17/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Febrile neutropenia	33.3% 8/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Leukocytosis	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Leukopenia	54.2% 13/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Lymphopenia	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Neutropenia	75.0% 18/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Blood and lymphatic system disorders Thrombocytopenia	100.0% 24/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Nervous system disorders Headache	41.7% 10/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Ear and labyrinth disorders Vertigo	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Abdominal pain	37.5% 9/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Abdominal pain upper	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Anal fissure	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Anal haemorrhage	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Anal inflammation	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Constipation	29.2% 7/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Diarrhoea	37.5% 9/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Dyspepsia	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Gingival bleeding	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Nausea	58.3% 14/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Stomatitis	75.0% 18/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Gastrointestinal disorders Vomiting	66.7% 16/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Renal and urinary disorders Haematuria	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Hepatobiliary disorders Venoocclusive liver disease	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Alopecia	33.3% 8/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Pigmentation disorder	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Pruritus	29.2% 7/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Rash	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Skin hypopigmentation	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Skin and subcutaneous tissue disorders Skin ulcer	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Musculoskeletal and connective tissue disorders Arthralgia	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Musculoskeletal and connective tissue disorders Back pain	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Musculoskeletal and connective tissue disorders Bone pain	20.8% 5/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Decreased appetite	20.8% 5/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Fluid overload	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Hypocalcaemia	20.8% 5/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Hypomagnesaemia	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Hypophosphataemia	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Cellulitis	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Folliculitis	16.7% 4/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Gingivitis	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Molluscum contagiosum	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Nasopharyngitis	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Pneumonia	12.5% 3/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Rhinitis	8.3% 2/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Infections and infestations Upper respiratory tract infection	25.0% 6/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.
Metabolism and nutrition disorders Hypokalaemia	25.0% 6/24 • From date of informed consent up to Month 24 ITT population included all participants who initiated mobilization procedure.

Additional Information

Study Medical Director

bluebird bio, Inc

Phone: 339-499-9300

Email: medinfo@bluebirdbio.com

Results disclosure agreements

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