Clinical trials: Gene Therapy
Pompe Disease
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and profound muscle weakness.
- Trial name
- NCT00976352
- Testing
- rAAV1-CMV-GAA (study agent) Administration, RMST
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Florida
Metastatic Melanoma, Metastatic Renal Cell Cancer
Chemotherapy Followed by ESO-1 Lymphocytes and Aldesleukin to Treat Metastatic Cancer
Background: -This study uses an experimental cancer treatment that uses the patient s own lymphocytes (type of white blood cell), which are specially selected and genetically modified to target and destroy their tumor.
- Conditions
- Metastatic Melanoma, Metastatic Renal Cell Cancer, Metastatic Cancer
- Trial name
- NCT00670748
- Testing
- Anti-NY ESO-1 T-cell receptor PBL, aldesleukin, Cyclophosphamide
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Cancer Institute (NCI)
KRAS Gene Mutation, Recurrent Lung Non-Small Cell Carcinoma
Trametinib and Docetaxel in Treating Patients With Recurrent or Stage IV KRAS Mutation Positive Non-small Cell Lung Cancer
This phase II trial studies how well trametinib and docetaxel work in treating patients with stage IV KRAS mutation positive non-small cell lung cancer or cancer that has come back.
- Conditions
- KRAS Gene Mutation, Recurrent Lung Non-Small Cell Carcinoma, Stage IV Lung Non-Small Cell Cancer AJCC v7
- Trial name
- NCT02642042
- Testing
- Docetaxel, Laboratory Biomarker Analysis, Trametinib
- Where
- 596 sites in United States
- Volunteers
- 60 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Stage I Breast Cancer, Stage II Breast Cancer
Niraparib + Dostarlimab In BRCA Mutated Breast Cancer
This research study involves pre-operative therapy that is specifically targeted for breast cancer in individuals with BRCA and PALB2 mutations.
- Conditions
- Stage I Breast Cancer, Stage II Breast Cancer, Stage III Breast Cancer, Breast Cancer
- Trial name
- NCT04584255
- Testing
- Niraparib, Dostarlimab
- Where
- 8 sites in United States
- Volunteers
- 66 people
- Sponsor
- Dana-Farber Cancer Institute
Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma
CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)
This study is for patients who have lymphoma or leukemia that has come back or has not gone away after treatment. Because there is no standard treatment for this cancer, patients are being asked to volunteer for a gene transfer…
- Conditions
- Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma, Relapsed Adult ALL, Relapsed CLL
- Trial name
- NCT03774654
- Testing
- CD19.CAR-aNKT cells
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Baylor College of Medicine
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.
Leber Congenital Amaurosis
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
- Trial name
- OPTIRPE65 (NCT02781480)
- Testing
- AAV RPE65
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- MeiraGTx UK II Ltd
Achromatopsia
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia…
- Trial name
- NCT03278873
- Testing
- Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 34 people
- Sponsor
- MeiraGTx UK II Ltd
Erectile Dysfunction
Phase 2A Clinical Trial Evaluating the Potential Activity and Safety of hMaxi-K Gene for ED
To evaluate the safety and efficacy of a single intracavernous injection of hMaxi K (8000 µg and 16000 µg) or placebo upon penile rigidity or erection in males with erectile dysfunction longer than six months that is attributable…
- Trial name
- NCT02713789
- Testing
- hMaxi-K Single Treatment/ 2 escalating dose levels (8000 µg and 16000 µg injection)
- Where
- Kuwait
- Volunteers
- 26 people
- Sponsor
- Urovant Sciences GmbH
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03526250
- Testing
- Laboratory Biomarker Analysis, Palbociclib, Pharmacological Study
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Lung Cancer
Erlotinib Versus Pemetrexed as Second-Line Therapy in Treating Patients With Advanced Lung Adenocarcinoma
Both pemetrexed and erlotinib are second-line treatment options for patients with advanced non-small cell lung cancer. It is controversial that whether it is necessary to detect epidermal growth factor receptor (EGFR) mutation…
- Trial name
- NCT01565538
- Testing
- Erlotinib, Pemetrexed
- Where
- China
- Volunteers
- 123 people
- Sponsor
- Si-Yu Wang
Non Small Cell Lung Cancer, EGFR Gene Mutation
Neo-DIANA: Neoadjuvant Treatment for EGFR Mutated Patients
This is an open-label, non-randomised, phase II, multi-centre clinical trial 26 patients will be enrolled in this trial to evaluate the major pathologic response in patients with neoadjuvant treatment with Carboplatin Pemetrexed…
- Trial name
- NCT04512430
- Testing
- Atezolizumab, Bevacizumab, Carboplatin
- Where
- 18 sites in Spain
- Volunteers
- 4 people
- Sponsor
- Fundación GECP
Diabetic Macular Edema, Diabetic Retinopathy
ADVM-022 Intravitreal Gene Therapy for DME
A Phase 2, Multi-Center, Randomized, Double-Masked*, Active Controlled Study of ADVM-022 (AAV.7m8-aflibercept) in Subjects with Diabetic Macular Edema [INFINITY]
- Trial name
- INFINITY (NCT04418427)
- Testing
- 6E11 vg/eye of ADVM-022, 2E11 vg/eye of ADVM-022, Aflibercept
- Where
- 14 sites in United States, Puerto Rico
- Volunteers
- 36 people
- Sponsor
- Adverum Biotechnologies, Inc.
Large Granular Lymphocytic Leukemia, LGL Leukemia
Effect of Cyclosporine Therapy on Gene Expression in Patients With Large Granular Lymphocyte Leukemia
Background: Large granular lymphocyte (LGL) leukemia is a low-grade non-Hodgkin's lymphoma. LGL is associated with low numbers of white blood cells (leading to recurring infections), red blood cells (causing anemia) and platelets…
- Trial name
- NCT00363779
- Testing
- Cyclosporine, Gene expression analysis, Microarray analysis
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Myeloid Leukemia
Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation
To evaluate the safety and efficacy of subcutaneous administration of omacetaxine mepesuccinate (HHT) in achieving a clinical response in CML patients in chronic, accelerated, or blast phase who have failed prior imatinib therapy…
- Trial name
- NCT00375219
- Testing
- Omacetaxine mepesuccinate
- Where
- 32 sites in United States, Canada and 8 more
- Volunteers
- 103 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Alpha 1-Antitrypsin Deficiency
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Individuals with a deficiency of the alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells with the expectation…
- Trial name
- AAT (NCT00430768)
- Testing
- rAAV1-CB-hAAT
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- University of Massachusetts, Worcester