A Study Evaluating the Safety and Efficacy of the LentiGl... | NCT01745120

Label	Type	Description	Intervention Names
LentiGlobin BB305 Drug Product	Experimental		Genetic: LentiGlobin BB305 Drug Product

Name	Type	Description	Arm Group Labels	Other Names
LentiGlobin BB305 Drug Product	Genetic	Transplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector.	LentiGlobin BB305 Drug Product

Measure	Description	Time Frame
Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months Between Month 18 and Month 24	Percentage of participants with sustained production of >=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months (Month 18 to Month 24) was reported.	Month 18 to Month 24
Percentage of Participants Who Achieved Transfusion Independence (TI)	TI was defined as a weighted average hemoglobin (Hb) >= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of >=12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Percentage of participants who achieved TI from time of drug product infusion up to 24 months was reported.	From time of drug product infusion up to 24 months

Measure	Description	Time Frame
Percentage of Participants Who Achieved Transfusion Independence (TI) at Month 18 and Month 24	TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion.	Month 18, Month 24

Name	Affiliation	Role
Mohammed Asmal, MD	Genetix Biotherapeutics Inc.	Study Director

PubMed Identifier	Type	Citation	Retractions
29669226	Derived	Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.

ID	Title	Description
FG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of beta(β)-globin: β+ or βE (non-β0/β0 genotype) underwent hematopoietic stem cell (HSC) mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of greater than or equal to (>=) 3.0 × 10^6 CD34+ cells per kilogram (cells/kg).

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Jun 29, 2015	Feb 7, 2019

ID	Title	Description
BG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
BG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG00011 BG0018 BG00219

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months Between Month 18 and Month 24

Percentage of participants with sustained production of >=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months (Month 18 to Month 24) was reported.

Transplant Population (TP) included all participants in the ITT population (participants who initiated any study procedures, beginning with mobilization by G-CSF with or without plerixafor) who underwent LentiGlobin BB305 Drug Product infusion.

Posted

Number

Percentage of participants

Month 18 to Month 24

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG002	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Units	Counts
Participants	OG00010 OG0018 OG00218

Title

Denominators

Categories

Title	Measurements
	OG00090.0 OG00187.5 OG00288.9

Primary

Percentage of Participants Who Achieved Transfusion Independence (TI)

TI was defined as a weighted average hemoglobin (Hb) >= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of >=12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Percentage of participants who achieved TI from time of drug product infusion up to 24 months was reported.

TP included all participants in the ITT population (participants who initiated any study procedures, beginning with mobilization by G-CSF with or without plerixafor) who underwent LentiGlobin BB305 Drug Product infusion.

Posted

Number

Percentage of participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Percentage of Participants Who Achieved Transfusion Independence (TI) at Month 18 and Month 24

TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion.

Posted

Number

Percentage of participants

Month 18, Month 24

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG002	Overall

Secondary

Duration of Transfusion Independence (TI)

TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Time period of TI will start when participants achieve a Hb >= 9 g/dL with no transfusions in the preceding 60 days. Duration of TI was calculated as the time from the start of TI (i.e. first Hb >= 9 g/dL with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met.

Posted

Median

Full Range

Months

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Time From LentiGlobin BB305 Drug Product Infusion to Last pRBC Transfusion Prior to Achieving Transfusion Independence (TI)

Posted

Median

Full Range

Months

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Time From LentiGlobin BB305 Drug Product Infusion to Achieving Transfusion Independence (TI)

TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Time from drug product infusion to initial achievement of TI was calculated as the time from drug product infusion to the first Hb at which a participant can be declared as TI.

Posted

Median

Full Range

Months

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Weighted Average Hemoglobin (Hb) During Period of Transfusion Independence (TI)

The weighted average Hb is an average area under the curve during the period of TI, from the start of TI when the Hb is first >= 9 g/dL with no transfusions in the preceding 60 days to the last available Hb at which the TI criteria are still met. TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Weighted average Hb during the period of TI was reported.

Posted

Mean

Standard Deviation

Grams per deciliter (g/dL)

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Percentage Change From Baseline in Annualized Number of Packed Red Blood Cells (pRBC) Transfusions at Month 24

The annualized number of pRBC transfusions over the 2 year period prior to drug product infusion was compared to the annualized number of pRBC transfusions during the Month 6 to Month 24 period post drug product infusion and the percentage change was reported.

Posted

Median

Full Range

Percentage of annualized transfusions

Baseline, Month 24

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Percentage Change From Baseline in Average Annual Packed Red Blood Cells (pRBC) Transfusion Volume at Month 24

The annualized volume of pRBC transfusions over the 2 year period prior to drug product infusion was compared to the annualized volume of pRBC transfusions in the Month 6 to Month 24 period post drug product Infusion and the percentage change from baseline was reported.

Posted

Median

Full Range

Percentage of pRBC transfusion volume

Baseline, Month 24

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Weighted Average Nadir Hemoglobin (Hb)

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 is used as the Hb nadir. If there is a period of more than 60 days without a pRBC transfusion, all Hb records between Day 61 and day of last visit or next transfusion (inclusive) were also considered as nadirs. The weighted average nadir Hb during the period of Month 6 to Month 24 was compared to the weighted average nadir Hb during the 2 years prior to enrollment.

Posted

Mean

Standard Deviation

Grams per deciliter (g/dL)

Baseline, Month 6 to Month 24

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Number of Participants With Successful Neutrophil Engraftment

Neutrophil engraftment was defined as achieving 3 consecutive absolute neutrophil count (ANC) >= 0.5 × 10^9/L on different days after a post-transplant value of < 0.5 × 10^9/L within 42 days after drug product infusion.

Posted

Count of Participants

Participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG002

Secondary

Time to Neutrophil Engraftment

Time to neutrophil engraftment was defined as the time to the first of 3 consecutive absolute neutrophil count (ANC) >= 0.5 × 10^9/L obtained on different days after a post-transplant value of < 0.5 × 10^9/L. The Day of neutrophil engraftment is the first day of the 3 consecutive measurements, where Day 1 is the day of drug product infusion.

TP included all participants in the ITT population who underwent LentiGlobin BB305 Drug Product infusion.

Posted

Median

Full Range

Days

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG002	Overall

Secondary

Number of Participants With Successful Platelet Engraftment

Platelet engraftment was defined as achieving 3 consecutive platelet values >= 20 × 10^9/L on different days after a post-transplant value of < 20 × 10^9/L, while no platelet transfusions administered for 7 days immediately preceding and during the evaluation period.

Posted

Count of Participants

Participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Time to Platelet Engraftment

Time to platelet engraftment was defined as achieving of first 3 consecutive platelet values >= 20 × 10^9/L obtained on different days after a post-transplant value of < 20 × 10^9/L, while no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. The day of platelet engraftment is the first day of the 3 consecutive measurements, where Day 1 is the day of drug product infusion.

Posted

Median

Full Range

Days

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Transplant-related Mortality

Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant.)

ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF with or without plerixafor.

Posted

Count of Participants

Participants

Through 100 and 365 days post-LentiGlobin BB305 Drug Product infusion

ID	Title	Description
OG000	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Units	Counts
Participants	OG000

Secondary

Overall Survival

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. Percentage of participants who survived throughout the study were reported.

ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF with or without plerixafor.

Posted

Number

Percentage of participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Units	Counts
Participants	OG000

Secondary

Percentage of Participants Detected With Replication-competent Lentivirus (RCL)

Blood samples were analyzed for detection of RCL using RCL co-culture assay.

Posted

Number

Percentage of participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Units	Counts
Participants	OG000

Secondary

Number of Participants With Integration Site Analysis (ISA) With >30% Clonal Contribution

Linear amplification-mediated polymerase chain reaction (LAM-PCR) coupled with next generation sequencing and subsequent (semi-) automated data mining allowed high-throughput analysis of vector integration site (IS) in blood cells from treated participants at multiple time points. ISs detected in peripheral blood cells at early time points generally were due to the expansion of transduced short-term progenitor stem cell clones, and gradually shift to include sites detected due to expansion of transduced long-term stem cell clones. An efficient transduction procedure was anticipated to give rise to a polyclonal population in the participant, reflected by the detection of multiple IS. Additionally, ISA allowed monitoring of the relative contribution of individual clones over time. Number of participants who had IS that contributed to >=30% of the total clones at any time was used as a first step to investigating whether clonal dominance was achieved.

Posted

Count of Participants

Participants

From time of drug product infusion up to 24 months

ID	Title	Description
OG000	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Secondary

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE was defined as any unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A SAE was any AE, occurring at any dose and regardless of causality that: results in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect.

ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF with or without plerixafor.

Posted

Count of Participants

Participants

From signing of informed consent to 24 months after the drug product infusion

ID	Title	Description
OG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.
OG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Thrombocytopenia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG00010 affected11 at risk EG0018 affected8 at risk EG00218 affected19 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0009 affected11 at risk EG0018 affected8 at risk EG00217 affected19 at risk
Febrile neutropenia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0007 affected11 at risk EG0014 affected8 at risk EG00211 affected19 at risk
Neutropenia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0006 affected11 at risk EG0014 affected8 at risk EG00210 affected19 at risk
Leukopenia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0005 affected11 at risk EG0011 affected8 at risk EG0026 affected19 at risk
Leukocytosis	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Lymphadenitis	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Lymphopenia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Neutrophilia	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Splenomegaly	Blood and lymphatic system disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Cardiac flutter	Cardiac disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Palpitations	Cardiac disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Sinus tachycardia	Cardiac disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Conjunctivitis	Eye disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Myopia	Eye disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Punctate keratitis	Eye disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Stomatitis	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0008 affected11 at risk EG0015 affected8 at risk EG00213 affected19 at risk
Nausea	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0007 affected11 at risk EG0015 affected8 at risk EG00212 affected19 at risk
Vomiting	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0017 affected8 at risk EG00211 affected19 at risk
Constipation	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0015 affected8 at risk EG0029 affected19 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0014 affected8 at risk EG0027 affected19 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0013 affected8 at risk EG0027 affected19 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0012 affected8 at risk EG0026 affected19 at risk
Toothache	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Abdominal distension	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Gastritis	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Gastrointestinal inflammation	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Gingival bleeding	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Proctalgia	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Anal fissure	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Aphthous ulcer	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Dysphagia	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Haemorrhoids	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Lip swelling	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Oesophagitis	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Paraesthesia oral	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Rectal haemorrhage	Gastrointestinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Catheter site pain	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0015 affected8 at risk EG0029 affected19 at risk
Fatigue	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0012 affected8 at risk EG0026 affected19 at risk
Pyrexia	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0012 affected8 at risk EG0025 affected19 at risk
Non-cardiac chest pain	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0011 affected8 at risk EG0023 affected19 at risk
Pain	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Chest discomfort	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Catheter site haemorrhage	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Catheter site inflammation	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Cyst	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Drug withdrawal syndrome	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Dysplasia	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Face oedema	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Influenza like illness	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Injection site pain	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Injection site reaction	General disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Cholecystitis	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hepatic congestion	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Hepatomegaly	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Jaundice	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Periportal sinus dilatation	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Venoocclusive liver disease	Hepatobiliary disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Drug hypersensitivity	Immune system disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Anaphylactic reaction	Immune system disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0014 affected8 at risk EG0024 affected19 at risk
Anal abscess	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Paronychia	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Anorectal infection bacterial	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Bacterial vaginosis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Bronchitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Catheter site cellulitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Cellulitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Eye infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Folliculitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Gastroenteritis viral	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Herpes simplex	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Influenza	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Moraxella infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Nasopharyngitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Pharyngitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Pneumonia	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Pseudomonal bacteraemia	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Rash pustular	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Respiratory tract infection viral	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Sialoadenitis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Staphylococcal infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Syphilis	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Viral upper respiratory tract infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Vulvovaginal mycotic infection	Infections and infestations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0006 affected11 at risk EG0014 affected8 at risk EG00210 affected19 at risk
Transfusion reaction	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0006 affected11 at risk EG0013 affected8 at risk EG0029 affected19 at risk
Skin abrasion	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0010 affected8 at risk EG0023 affected19 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Infusion related reaction	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Laceration	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Anaphylactic transfusion reaction	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Citrate toxicity	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Iron overload	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Ligament rupture	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Transfusion-related circulatory overload	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Vascular access complication	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Wound	Injury, poisoning and procedural complications	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Alanine aminotransferase increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0011 affected8 at risk EG0024 affected19 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0010 affected8 at risk EG0023 affected19 at risk
Blood bilirubin increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0003 affected11 at risk EG0010 affected8 at risk EG0023 affected19 at risk
Gamma-glutamyltransferase increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Activated partial thromboplastin time prolonged	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Blood albumin decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood alkaline phosphatase decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood creatinine increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood follicle stimulating hormone increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood iron increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood magnesium decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Blood potassium decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Blood sodium decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
C-reactive protein increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Carbon dioxide decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Forced expiratory flow decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
International normalised ratio increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Mean cell haemoglobin concentration increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Mean cell volume decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Monocyte count decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Protein total decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Red blood cell count increased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Reticulocyte count decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Reticulocyte percentage decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Weight decreased	Investigations	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0013 affected8 at risk EG0024 affected19 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0011 affected8 at risk EG0023 affected19 at risk
Hypocalcaemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Hyponatraemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Fluid overload	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hyperglycaemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Hyperphosphataemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hypoalbuminaemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hypomagnesaemia	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Iron overload	Metabolism and nutrition disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0012 affected8 at risk EG0024 affected19 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0011 affected8 at risk EG0023 affected19 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Musculoskeletal discomfort	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Neck pain	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Flank pain	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Osteopenia	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Pain in jaw	Musculoskeletal and connective tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Headache	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0006 affected11 at risk EG0014 affected8 at risk EG00210 affected19 at risk
Peripheral sensory neuropathy	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0014 affected8 at risk EG0026 affected19 at risk
Dizziness	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Dysgeusia	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Head discomfort	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Lethargy	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Somnolence	Nervous system disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Insomnia	Psychiatric disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0012 affected8 at risk EG0026 affected19 at risk
Agitation	Psychiatric disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Anxiety	Psychiatric disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Haematuria	Renal and urinary disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Dysuria	Renal and urinary disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Urinary retention	Renal and urinary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Urinary tract pain	Renal and urinary disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Vaginal haemorrhage	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0014 affected8 at risk EG0026 affected19 at risk
Menstruation irregular	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0004 affected11 at risk EG0010 affected8 at risk EG0024 affected19 at risk
Ovarian failure	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Menorrhagia	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Ovarian hyperstimulation syndrome	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Vulvovaginal pruritus	Reproductive system and breast disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0006 affected11 at risk EG0013 affected8 at risk EG0029 affected19 at risk
Pharyngeal inflammation	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0015 affected8 at risk EG0027 affected19 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Hypoxia	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Laryngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Rales	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Respiratory tract irritation	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Rhinitis allergic	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Throat irritation	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Upper-airway cough syndrome	Respiratory, thoracic and mediastinal disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Alopecia	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0009 affected11 at risk EG0018 affected8 at risk EG00217 affected19 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0013 affected8 at risk EG0025 affected19 at risk
Skin hyperpigmentation	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0013 affected8 at risk EG0024 affected19 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0013 affected8 at risk EG0024 affected19 at risk
Petechiae	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0012 affected8 at risk EG0023 affected19 at risk
Dermatitis contact	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0012 affected8 at risk EG0022 affected19 at risk
Dry skin	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0011 affected8 at risk EG0022 affected19 at risk
Ecchymosis	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0002 affected11 at risk EG0010 affected8 at risk EG0022 affected19 at risk
Eczema	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hyperhidrosis	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Pain of skin	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Palpable purpura	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Pruritus allergic	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Pruritus generalised	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Purpura	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Rash follicular	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Skin abrasion	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Skin discolouration	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Skin hypopigmentation	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Sweat gland disorder	Skin and subcutaneous tissue disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Deep vein thrombosis	Vascular disorders	MedDRA 19.0	Non-systematic Assessment	EG0000 affected11 at risk EG0011 affected8 at risk EG0021 affected19 at risk
Flushing	Vascular disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hot flush	Vascular disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Hypotension	Vascular disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk
Vena cava thrombosis	Vascular disorders	MedDRA 19.0	Non-systematic Assessment	EG0001 affected11 at risk EG0010 affected8 at risk EG0021 affected19 at risk

Facility	Status	City	State	ZIP	Country	Contacts
		Los Angeles	California		United States

ID	Term
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Non-β0/β0	Participants who had at least 1 mutation in the HBB gene that results in reduced but detectable expression of β-globin: β+ or βE (non-β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.	11	6	11	10	11
EG001	β0/β0	Participants who were bi-allelic for mutations in the HBB gene that results in absence of expression of β-globin: β0 (β0/β0 genotype) underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.	8	4	8	8	8
EG002	Overall	Participants with a β0/β0 genotype or non-β0/β0 genotypes underwent HSC mobilization with G-CSF and plerixafor, followed by myeloablative conditioning and infusion with LentiGlobin BB305 Drug Product at a dose of >= 3.0 × 10^6 CD34+ cells/kg.	19	10	19	18	19