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ASC618 Gene Therapy in Hemophilia A Patients

for
Hemophilia A
Location: Little Rock AR
Sponsor: ASC Therapeutics

Sex: Male

Age: 18+

Code: NCT04676048

Phase1, Phase2, Recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

for
Beta-Thalassemia
Location: Philadelphia PA
Sponsor: Children's Hospital of Philadelphia

Sex: All

Age: 18 - 40

Code: NCT06364774

Phase1, Phase2, Recruiting

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

for
Friedreich Ataxia, Cardiomyopathy, Secondary
Location: Tampa FL
Sponsor: Lexeo Therapeutics

Sex: All

Age: 6+

Code: NCT07721025

Phase2, Recruiting

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

for
Dilated Cardiomyopathy (DCM)
Location: Charleston SC, Rochester MN, San Diego CA
Sponsor: Rocket Pharmaceuticals Inc.

Sex: All

Age: 18 - 65

Code: NCT07137338

Phase1, Recruiting

Virus-Based Gene Therapy (AdV-HSV1-TK and AdV-Flt3L) in Combination With Valacyclovir for the Treatment of Pediatric and Young Adult Patients With Resectable, Recurrent Primary Malignant Brain Tumors

for
Recurrent Diffuse Hemispheric Glioma, H3 G34-Mutant,
Location: Ann Arbor MI
Sponsor: University of Michigan Rogel Cancer Center

Sex: All

Age: 3 - 39

Code: NCT06914479

Phase1, Recruiting

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Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

for
Pitt Hopkins Syndrome
Location: Chicago IL, Aurora CO, Oakland CA
Sponsor: Mahzi Therapeutics

Sex: All

Age: 2 - 25

Code: NCT07135050

Phase1, Phase2, Recruiting

Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

for
Sickle Cell Disease
Location: Memphis TN
Sponsor: St. Jude Children's Research Hospital

Sex: All

Age: 13+

Code: NCT04416178

Recruiting

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

for
Catecholaminergic Polymorphic Ventricular Tachycardia
Location: Boston MA, Cleveland OH, Rochester MN, Vancouver
Sponsor: Solid Biosciences Inc.

Sex: All

Age: 7+

Code: NCT07148089

Phase1, Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

for
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Location: Philadelphia PA, Durham NC, Dallas TX, Irvine CA
Sponsor: AskBio Inc

Sex: All

Age: 18 - 65

Code: NCT07282847

Phase1, Phase2, Recruiting

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

for
Sickle Cell Disease
Location: Memphis TN
Sponsor: St. Jude Children's Research Hospital

Sex: All

Age: 8+

Code: NCT05810181

Recruiting

Gene Therapy for HER-Positive Cancer (SENTRY-HER2)

for
HER2 Expressing Solid Tumors
Location: New York NY, Hackensack NJ, Philadelphia PA,
Sponsor: Vironexis Biotherapeutics Inc.

Sex: All

Age: 18+

Code: NCT07192432

Phase1, Phase2, Recruiting

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

for
Myotonic Dystrophy
Location: New York NY, Montreal, Richmond VA, Tampa FL,
Sponsor: Sanofi

Sex: All

Age: 10 - 55

Code: NCT06844214

Phase1, Phase2, Recruiting

Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

for
CD40L-HyperIgM Syndrome
Location: Bethesda MD
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Sex: Male

Age: 37 - 70+

Code: NCT06959771

Phase1, Phase2, Recruiting

Adenovirus Mediated Suicide Gene Therapy With Radiotherapy in Progressive Astrocytoma.

for
Malignant Glioma of Brain, Astrocytoma, Malignant Astrocytoma,
Location: Detroit MI
Sponsor: Henry Ford Health System

Sex: All

Age: 18+

Code: NCT05686798

Phase1, Recruiting

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

for
Hemophilia B
Location: Pittsburgh PA, Hamilton, Ann Arbor MI, Houston TX,
Sponsor: CSL Behring

Sex: All

Age: 18+

Code: NCT06003387

Phase3, Recruiting

Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

for
Squamous Cell Carcinoma
Location: Redwood City CA
Sponsor: Stanford University

Sex: All

Age: 18+

Code: NCT06731933

Phase2, Recruiting

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

for
Phenylketonurias
Location: Morristown NJ, Pittsburgh PA, Minneapolis MN, Dallas TX,
Sponsor: NGGT INC.

Sex: All

Age: 18 - 55

Code: NCT06332807

Phase1, Phase2, Recruiting

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

for
Sickle Cell Disease,
Location: Boston MA
Sponsor: Daniel Bauer

Sex: All

Age: 13 - 40

Code: NCT06647979

Phase1, Recruiting

Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)

for
Diamond Blackfan Anemia
Location: New Hyde Park NY
Sponsor: Northwell Health

Sex: All

Age: 3 - 30

Code: NCT07186179

Recruiting

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

for
Hemophilia B
Location: New Haven CT, Montreal, Philadelphia PA, Hamilton,
Sponsor: Regeneron Pharmaceuticals

Sex: Male

Age: 2+

Code: NCT06379789

Phase1, Phase2, Recruiting