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ClinicalTrials.gov public records Last synced Oct 5, 2026, 4:35 PM EDT

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Showing 1–16 of 16 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Genetix Biotherapeutics Inc. Clear all
Completed Phase 3 Interventional Results available

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

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 5, 2026, 4:35 PM EDT
Active, not recruiting No phase listed Observational

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

NCT02698579
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Interventions
No interventional drug product utilized in this follow-up study
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 19 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2038
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 5, 2026, 4:35 PM EDT
Terminated No phase listed Observational

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

NCT02204904
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Interventions
Allo-HSCT
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
6
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated May 21, 2020 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
lovo-cel
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Recruiting No phase listed Observational

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

NCT06224413
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2047
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Active, not recruiting No phase listed Observational

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

NCT02633943
View directory record Official record
Conditions
Transfusion-dependent Beta-Thalassemia
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2035
U.S. locations
6
States / cities
Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Recruiting No phase listed Observational

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

NCT06271512
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2043
U.S. locations
6
States / cities
Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Active, not recruiting Phase 3 Interventional

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

NCT04293185
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
bb1111
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 50 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 5, 2026, 4:35 PM EDT
Terminated Phase 2 Interventional Results available

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

NCT05445128
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
MGTA-145, Plerixafor
Biological · Drug
Lead sponsor
Ensoma
Industry
Eligibility
18 Years to 35 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
3
States / cities
Bethesda, Maryland • Boston, Massachusetts • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 25, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2024 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 3 Interventional Results available

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 24, 2024 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT01896102
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 1 Interventional

Study of bb2121 in Multiple Myeloma

NCT02658929
View directory record Official record
Conditions
Multiple Myeloma
Interventions
bb2121
Biological
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
9
States / cities
Stanford, California • Bethesda, Maryland • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jan 23, 2023 · Synced Oct 5, 2026, 4:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

NCT01745120
View directory record Official record
Conditions
β-thalassemia Major
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
4
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated May 8, 2019 · Synced Oct 5, 2026, 4:35 PM EDT
Active, not recruiting Phase 2 Interventional

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

NCT05353647
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 5, 2026, 4:35 PM EDT