Clinical trials: Genes
Human Immunodeficiency Virus (HIV)
Repeat Doses of SB-728mR-T After Cyclophosphamide Conditioning in HIV-Infected Subjects on HAART
The purpose of this study is to evaluate the safety and tolerability of repeat doses of T-cell immunotherapy (SB-728mR-T) following cyclophosphamide conditioning. CCR5 is a major co-receptor for HIV entry into T-cells.
- Trial name
- NCT02225665
- Testing
- SB-728mR-T, Cyclophosphamide
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Relapsed Ovarian Cancer, BRCA Mutation, Platinum Sensitivity
Olaparib Treatment in Relapsed Germline Breast Cancer Susceptibility Gene (BRCA) Mutated Ovarian Cancer Patients Who Have Progressed at Least 6 Months After Last Platinum Treatment and Have Received at Least 2 Prior Platinum Treatments
Comparison of olaparib vs. physician's choice of single agent standard of care non-platinum based chemotherapy in patients with germline Breast Cancer susceptibility gene (gBRCA) mutated ovarian cancer who have progressed at…
- Trial name
- SOLO3 (NCT02282020)
- Testing
- OLAPARIB, Single agent chemotherapy
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 266 people
- Sponsor
- AstraZeneca
Ischemic Heart Disease
Safety Study of Gene Therapy for Ischemic Heart Disease in Korea
The purpose of this study is to evaluate the safety of VM202 (Engensis) direct injection into the cardiac muscles of the coronary artery territory where complete revascularization could not be done even through Coronary Artery…
- Trial name
- Engensis (NCT01422772)
- Testing
- VM202-0.5 mg, VM202-1.0 mg, VM202-2.0 mg
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- Helixmith Co., Ltd.
Choroideremia
Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).
- Trial name
- STAR (NCT03496012)
- Testing
- BIIB111
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 169 people
- Sponsor
- Biogen
Osteoarthritis, Knee
Efficacy and Safety of XT-150 in Osteoarthritis of the Knee
This is a Phase 2 safety and efficacy study of XT-150 in adult participants experiencing moderate to severe pain due to osteoarthritis of the knee.
- Trial name
- NCT04124042
- Testing
- XT-150
- Where
- 6 sites in United States, Australia
- Volunteers
- 289 people
- Sponsor
- Xalud Therapeutics, Inc.
AADC Deficiency
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of…
- Trial name
- NCT04903288
- Testing
- Eladocagene Exuparvovec
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Receptors, Estrogen, Genes, Erbb-2
A Study of H3B-6545 in Combination With Palbociclib in Women With Advanced or Metastatic Estrogen Receptor-Positive Human Epidermal Growth Factor Receptor-2 (HER2)-Negative Breast Cancer
The primary objective of this study is to evaluate the safety and tolerability of H3B-6545 and palbociclib when administered in combination in order to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2…
- Conditions
- Receptors, Estrogen, Genes, Erbb-2, Breast Neoplasms
- Trial name
- NCT04288089
- Testing
- Palbociclib (75, 100, 125 milligram [mg]), H3B-6545 (150, 300, 450 mg)
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 31 people
- Sponsor
- Eisai Inc.
Limb-Girdle Muscular Dystrophy, Type 2D
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
- Trial name
- NCT01976091
- Testing
- SRP-9004
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Pancreatic Neoplasms
Phase 1/2a DTA-H19 in Patients With Unresectable Pancreatic Cancer
This study is designed to assess the safety, tolerability, pharmacokinetics (PK) and preliminary efficacy of DTA-H19 administered intratumorally in patients with unresectable, locally advanced pancreatic cancer.
- Trial name
- NCT00711997
- Testing
- DTA-H19
- Where
- 4 sites in United States, Israel
- Volunteers
- 9 people
- Sponsor
- Anchiano Therapeutics Israel 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
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Ovarian Cancer, Breast Cancer
A Crossover Bioequivalence Study of Olaparib Tablets, 150 mg (Lek Pharmaceuticals d.d.) and Lynparza® (Olaparib) Tablets 150 mg (AstraZeneca Pharmaceuticals LP), in Patients With Breast Cancer Gene (BRCA) Mutated Ovarian Cancer, Recurrent Ovarian Cancer or Metastatic Breast Cancer
This is a two-way crossover bioequivalence study between test and reference product in patients diagnosed with BRCA mutated ovarian cancer, recurrent ovarian cancer or metastatic breast cancer.
- Trial name
- NCT05258747
- Testing
- Olaparib tablets, 150 mg, Lynparza® (olaparib) tablets 150 mg
- Where
- India
- Volunteers
- 70 people
- Sponsor
- Sandoz
Alzheimer's Disease
The Clinical Response of Choline Acetyltransferase and Apolipoprotein Epsilon Gene Polymorphisms to Donepezil in Alzheimer's Disease
This study attempts to differentiate the clinical responses of Choline Acetyltransferase and Apolipoprotein Epsilon gene polymorphism to donepezil in Alzheimer's Disease patients.
- Trial name
- NCT00381381
- Testing
- Donepezil
- Where
- 15 sites in South Korea
- Volunteers
- 199 people
- Sponsor
- Eisai Korea Inc.
Attention Deficit Hyperactivity Disorder
PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations
This is PART B of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes implicated in glutamatergic signaling and…
- Trial name
- NCT03609619
- Testing
- AEVI-001
- Where
- United States
- Volunteers
- 109 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Advanced NSCLC
Real-World Study on Gene Profile in Patients With Advanced NSCLC Who Progressed on First-Line Osimertinib Therapy(GPS).
Although some small sample studies have reported the possible resistance mechanisms of Osimertinib in the first-line treatment, it is still an urgent need to explore the whole gene profile in EGFRm advanced NSCLC patients post…
- Trial name
- GPS (NCT05219162)
- Testing
- Gene Profile explore
- Where
- 16 sites in China
- Volunteers
- 182 people
- Sponsor
- AstraZeneca
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
X-linked Retinoschisis
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis.
- Trial name
- NCT02416622
- Testing
- rAAV2tYF-CB-hRS1
- Where
- 9 sites in United States
- Volunteers
- 27 people
- Sponsor
- Beacon Therapeutics
LMNA-Related Dilated Cardiomyopathy
A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy
This is a rollover study designed to investigate the safety and effectiveness of investigational study drug ARRY-371797 in patients who previously received ARRY-371797 in a study for patients with LMNA-related dilated…
- Trial name
- NCT02351856
- Testing
- ARRY-371797, p38 inhibitor, oral
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer