Clinical trials: Gene Therapy
Head and Neck Neoplasms
Recombinant Human Endostatin Adenovirus Combined With Chemotherapy for Advanced Head and Neck Malignant Tumors
This study will investigate the efficacy and safety of recombinant human endostatin adenovirus combined with chemotherapy for advanced head and neck malignant tumors.
- Trial name
- NCT02283489
- Testing
- recombinant human endostatin adenovirus, Cisplatin injection, Paclitaxel injection
- Where
- 4 sites in China
- Volunteers
- 180 people
- Sponsor
- Renmiao Zhang
Retinitis Pigmentosa
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients.
- Trial name
- MOON (NCT06292650)
- Testing
- ZM-02-L, ZM-02-H, ZM-02-S
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Zhongmou Therapeutics
HER2-positive, Adenocarcinoma
CAR-macrophages for the Treatment of HER2 Overexpressing Solid Tumors
Phase 1, first-in-human, open label study of CAR macrophages in HER2 overexpressing solid tumors.
- Conditions
- HER2-positive, Adenocarcinoma, Bile Duct Cancer, Biliary Tract Cancer
- Trial name
- NCT04660929
- Testing
- CT-0508, Pembrolizumab
- Where
- 7 sites in United States
- Volunteers
- 48 people
- Sponsor
- Carisma Therapeutics Inc
Hemophilia B
A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing.
- Trial name
- NCT03641703
- Testing
- FLT180a
- Where
- 6 sites in United Kingdom
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
Coronary Artery Disease
Evaluation of the GTX™ 5126 DES Coronary Stent System in the Treatment of Patients With a Lesion in the Coronary Artery
The GTX™ Drug Eluting Coronary Stent System is intended for the treatment of patients with a lesion in the coronary artery.
- Trial name
- NCT00777842
- Testing
- GTX™ Drug Eluting Coronary Stent System
- Where
- Germany
- Volunteers
- 19 people
- Sponsor
- Cook Group Incorporated
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies
Migraine
Head-to-head Study of Erenumab Against Topiramate in Patients With Episodic and Chronic Migraine
This study used a single-cohort, 2-treatment arm, parallel-group randomized, double-blind, double-dummy design in adult patients with episodic migraine and chronic migraine, who had to be either naïve or not suitable for or could…
- Trial name
- HER-MES (NCT03828539)
- Testing
- Erenumab, Topiramate
- Where
- 79 sites in Germany
- Volunteers
- 777 people
- Sponsor
- Novartis Pharmaceuticals
OTC Deficiency
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
- Trial name
- NCT05345171
- Testing
- DTX301, Oral Corticosteroids, Sodium Acetate
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 37 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing…
- Trial name
- NCT06241950
- Testing
- delandistrogene moxeparvovec, imlifidase
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Sarepta Therapeutics, Inc.
Diabetic Retinopathy (DR), Center-Involved Diabetic Macular Edema (CI-DME)
RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)
ABBV-RGX-314 is being developed as a novel, potential one-time gene therapy treatment for the treatment of Diabetic Retinopathy (DR) with and without Center-Involved Diabetic Macular Edema (CI-DME).
- Trial name
- ALTITUDE® (NCT04567550)
- Testing
- ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3
- Where
- 25 sites in United States
- Volunteers
- 139 people
- Sponsor
- AbbVie
HER-2 Gene Amplification
A Study of MM-111 in Combination With Multiple Treatments in Patients With HER2 Positive Cancer
This study is an open-label, dose-escalation study of MM-111 with five different combination treatments with the main goal of determining the safety of MM-111 with each combination.
- Trial name
- NCT01304784
- Testing
- Cisplatin, Capecitabine, Trastuzumab and MM-111, Lapatinib +/- Trastuzumab and MM-111, Paclitaxel, Trastuzumab and MM-111
- Where
- 15 sites in United States
- Volunteers
- 100 people
- Sponsor
- Merrimack Pharmaceuticals
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- NCT06333249
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD
Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration
The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to the contralateral (second) eye of adult participants (≥ 50 years of…
- Trial name
- NCT07630649
- Testing
- Ixo-vec
- Where
- 12 sites in United States
- Volunteers
- 15 people
- Sponsor
- Adverum Biotechnologies, Inc.
Retinitis Pigmentosa
Promising ROd-cone DYstrophy Gene therapY
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a…
- Trial name
- PRODYGY (NCT05748873)
- Testing
- SPVN06
- Where
- 6 sites in United States, France
- Volunteers
- 33 people
- Sponsor
- SparingVision
Clear Cell RCC
A Clinical Gene Therapy Study With Hematopoietic Stem Cells for the Treatment, With Single Dose of Temferon, of Patients Suffering From Metastatic Renal Cell Carcinoma
This is an open label, single-centre phase 1/2 study involving a single dose of Temferon, an investigational Advanced Therapy Medicinal Product (ATMP), to treat patients with metastatic clear cell renal cell carcinoma (RCC) with…
- Trial name
- TEM-GU (NCT06716853)
- Testing
- Temferon, Pembrolizumab, Cabozantinib
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Genenta Science
Angelman Syndrome
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
- Trial name
- ASCEND-AS (NCT07181837)
- Testing
- MVX-220
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- MavriX Bio, LLC
Gaucher Disease, Type 1
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00954460
- Testing
- velaglucerase alfa
- Where
- 32 sites in United States
- Sponsor
- Shire
Head and Neck Cancer
Safety Study of Adenovirus/PNP Coupled With Fludarabine Phosphate to Treat Solid Tumors
This study will test whether it is possible to introduce new genetic material into a small portion of a tumor and have the product of the new gene not only kill those tumor cells that were infected initially, but also the…
- Trial name
- NCT01310179
- Testing
- Ad/PNP and fludarabine monophosphate
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- PNP Therapeutics, Inc.
Actinic Keratosis
Cellular and Molecular Events During Treatment of Actinic Keratosis With Imiquimod 5% Cream
Treatment of actinic keratosis with imiquimod cream is expected to cause changes in the genes that are turned on, and turned off, by skin cells.
- Trial name
- NCT00335179
- Testing
- Aldara (imiquimod 5% cream), Vehicle cream
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Graceway Pharmaceuticals, LLC