Clinical trials: Gene Therapy
Severe Combined Immunodeficiency
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA).
- Trial name
- NCT00001255
- Testing
- ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
NF2 Deficiency
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors.
- Trial name
- NCT06834438
- Testing
- ST002
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Galactosialidosis
Characterization of the Patient Population With Galactosialidosis
The late infantile form of galactosialidosis is potentially amenable to treatment by gene transfer with an adeno-associated viral vector encoding Protective Protein Cathepsin A (PPCA) or by infusion of purified protein.
- Trial name
- CPPGAL (NCT01416467)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- St. Jude Children's Research Hospital
Methylmalonic Acidemia
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA…
- Trial name
- SUNRISE (NCT04581785)
- Testing
- hLB-001
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- LogicBio Therapeutics, Inc
Metachromatic Leukodystrophy (MLD)
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem…
- Trial name
- NCT07046338
- Testing
- Lentiviral TYF-ARSA correction of patient's autologous HSCs
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Severe Combined Immunodeficiency Due to ADA Deficiency
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic…
- Trial name
- NCT02999984
- Testing
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Los Angeles
Non-small Cell Lung Cancer
The Recurrence Gene Profiles of Adjuvant Osimertinib Therapy in Resected Non-Small-Cell Lung Cancer
This study was a multicenter, real-world observational study aimed at characterizing the genomic landscape of recurrence in patients with EGFR-sensitizing mutation-positive early-stage (clinical stage IB-IIIA) non-small cell lung…
- Trial name
- RAISE (NCT06477055)
- Where
- 2 sites in China
- Volunteers
- 43 people
- Sponsor
- Hunan Province Tumor Hospital
Hemophilia B
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
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
HIV Infections, Pregnancy
Setting up a Blood Bank for Gene Therapy in HIV-Infected Infants
The purpose of this study is to set up a blood bank for infants who have HIV-positive mothers. This blood may be used in the future to treat the child if he/she turns out to be HIV-positive.
- Trial name
- NCT00000917
- Where
- 8 sites in United States
- Volunteers
- 200 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
SLC13A5 Citrate Transporter Disorder
Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder
Phase 1/2, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of TSHA-105 in individuals with SLC13A5 Citrate Transporter Disorder
- Trial name
- SLC13A5 (NCT07102524)
- Testing
- TSHA-105
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- TESS Research Foundation