Clinical trials: Gene Therapy
Alzheimer Disease
Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease
The primary purpose of this long-term follow-up study is to assess the long-term safety profile of APOE4 homozygote participants who were administered gene therapy (LX1001) for the treatment of Alzheimer's disease in Study…
- Trial name
- LEADLTFU (NCT05400330)
- Testing
- LX1001
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Lexeo Therapeutics
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
HPV-Related Squamous Cell Carcinoma
A Phase 1/2 Study in Patients With HPV16+ Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma and Other Cancers
This is a First in Human (FIH) Phase I/II, multinational, multicenter, open-label study of HB-201 single vector therapy and HB-201 \& HB-202 two-vector therapy in patients with HPV 16+ confirmed cancers comprising two parts…
- Trial name
- NCT04180215
- Testing
- HB-201 intravenous administration., HB-202 intravenous administration alternating with HB-201 intravenous administration., HB-201 intravenous administration + standard of care regimen including pembrolizumab.
- Where
- 34 sites in United States, Netherlands and 1 more
- Volunteers
- 198 people
- Sponsor
- Hookipa Biotech GmbH
FGFR Inhibition, Pharmacokinetics, Biomarkers, ER+ Breast Cancer
Safety and Efficacy of AZD4547 in Combination With Fulvestrant vs. Fulvestrant Alone in ER+ Breast Cancer Patients
The purpose of this study is to assess the safety and effectiveness of AZD4547 in combination with fulvestrant vs. fulvestrant alone in ER+ breast cancer patients with FGFR1 polysomy (FISH4/5) or gene amplification (FISH 6)
- Trial name
- GLOW (NCT01202591)
- Testing
- AZD4547, Exemestane, Fulvestrant
- Where
- 21 sites in Belgium, Czechia and 6 more
- Volunteers
- 127 people
- Sponsor
- AstraZeneca
Myelodysplastic Syndrome
A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.
Several previous studies (clinical and non-clinical) hypothesize that treatment with deferasirox causes a hematological improvement in transfused patients with low and intermediate-1 risk myelodysplastic syndrome.
- Trial name
- EXPHAR (NCT02663752)
- Testing
- Bone marrow aspirate, Deferasirox
- Where
- Belgium
- Volunteers
- 1 people
- Sponsor
- Novartis Pharmaceuticals
Ischemic Heart Disease, Acute Myocardial Infarction
Safety and Efficacy Study of Gene Therapy for Acute Myocardial Infarction in Korea
The purpose of this study is to evaluate the safety and clinical efficacy of VM202RY injected via transendocardial route using C-Cathez® catheter (Celyad, S.A., Belgium) in subjects with AMI.
- Trial name
- NCT03404024
- Testing
- Stage 1-Low dose VM202RY, Stage 1-Middle dose VM202RY, Stage 1-High dose VM202RY
- Where
- 4 sites in South Korea
- Sponsor
- Helixmith Co., Ltd.
Hemophilia B, Blood Coagulation Disorders, Inherited
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB).
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Trial name
- ECLIPSE (NCT04272554)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 37 people
- Sponsor
- Spur Therapeutics
Stargardt Disease Type 1 (STGD1)
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence.
- Trial name
- START (NCT07063251)
- Testing
- HG005
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Mucopolysaccharidosis IH
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of…
- Trial name
- TigetT10_MPSIH (NCT03488394)
- Testing
- Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Orchard Therapeutics
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Choroideremia
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).
- Trial name
- GEMINI (NCT03507686)
- Testing
- BIIB111
- Where
- 6 sites in United States, France and 1 more
- Volunteers
- 66 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Osteoarthritis, Knee
A Study Evaluating the Safety, Tolerability, and Range of Biologically Active Doses of ICM-203 in Mild to Moderate Knee Osteoarthritis
The purpose of this study is to determine the safety, tolerability, and activity of ICM-203, a recombinant adeno-associated viral (AAV) vector that expresses a therapeutic gene that promotes cartilage formation, reduces joint…
- Trial name
- NCT04875754
- Testing
- ICM-203
- Where
- 2 sites in Australia
- Volunteers
- 12 people
- Sponsor
- ICM Biotech Australia Pty Ltd.
Parkinson's Disease (PD)
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Injection of LY-N001 Injection for the Treatment of Moderate to Advanced Parkinson's Disease With GBA1 Mutations
This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with…
- Trial name
- NCT07685444
- Testing
- LY-N001 Injection
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Lingyi Biotech Co., Ltd.
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A.
- Trial name
- NCT04418414
- Testing
- Gene therapy, Biological
- Volunteers
- 7 people
- Sponsor
- Expression Therapeutics, LLC
AMD, nAMD
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD).
- Conditions
- AMD, nAMD, Wet Age-related Macular Degeneration, wAMD
- Trial name
- ATMOSPHERE (NCT04704921)
- Testing
- ABBV-RGX-314, Ranibizumab (LUCENTIS®)
- Where
- 89 sites in United States
- Volunteers
- 671 people
- Sponsor
- AbbVie
Breast Cancer
Plant Extracted Natural Compounds Impact on Stage IV Breast Cancer Survival Time and Remission.
RATIONALE: Plant extracted natural compounds, in an adjunct therapy position, slow the growth and reproduction of Stage IV Breast Cancer tumor cells. May help eradicate different types of cancers.
- Trial name
- NCT00910884
- Testing
- laboratory biomarker analysis, therapeutic dietary intervention
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Brabant Research
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.