Clinical trials: Gene Therapy
Breast Cancer
Autologous Vaccination With Lethally Irradiated, Autologous Breast Cancer Cells Engineered to Secrete GM-CSF in Women With Operable Breast Cancer
The purpose of this trial is to test the safety of a vaccine made from a patient's own breast cancer cells, and determine if this vaccine will delay or stop the growth of the cancer.
- Trial name
- NCT00880464
- Testing
- Autologous, Lethally Irradiated Breast Cancer Cells
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Dana-Farber Cancer Institute
Ductal Breast Carcinoma in Situ, Lobular Breast Carcinoma in Situ
Hormone Therapy Or Chemotherapy Before Surgery Based on Gene Expression Analysis in Treating Patients With Breast Cancer
This randomized pilot clinical trial studied whether the Oncotype DX gene expression "Recurrence Score" (RS) would be useful for helping make a decision about which type of pre-operative treatment, hormone therapy or chemotherapy…
- Conditions
- Ductal Breast Carcinoma in Situ, Lobular Breast Carcinoma in Situ, Stage II Breast Cancer, Stage IIA Breast Cancer
- Trial name
- NCT01293032
- Testing
- Neoadjuvant Therapy, Therapeutic Conventional Surgery, Laboratory Biomarker Analysis
- Where
- 8 sites in United States, Canada
- Volunteers
- 59 people
- Sponsor
- Virginia Commonwealth University
Papillomavirus Infections, Cervical Intraepithelial Neoplasia
E7 TCR T Cells for Human Papillomavirus-Associated Cancers
Background: Human papillomavirus (HPV) can cause cervical, throat, anal, and genital cancers. Cancers caused by HPV have an HPV protein called E7 inside of their cells.
- Conditions
- Papillomavirus Infections, Cervical Intraepithelial Neoplasia, Carcinoma In Situ, Vulvar Neoplasms
- Trial name
- NCT02858310
- Testing
- E7 TCR cells, Aldesleukin, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 224 people
- Sponsor
- National Cancer Institute (NCI)
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
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen.
- Trial name
- NCT01263379
- Testing
- LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
Prostate Cancer
Effect of Dutasteride on Androgen-Response Gene Expression in Patients With Advanced Prostate Cancer
The purpose of this study is to determine if the drug dutasteride increases expression of genes that slow the growth of prostate cancer during treatment with intermittent androgen ablation therapy (hormone therapy).
- Trial name
- NCT00668642
- Testing
- Dutasteride
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Endeavor Health
Breast Adenocarcinoma, Deleterious BRCA1 Gene Mutation
Talazoparib Before Standard Therapy in Treating Patients With Invasive, BRCA-Mutated Breast Cancer
This phase II trial studies the side effects of talazoparib when given before standard therapy in treating patients with breast cancer that has spread to nearby healthy tissue and has a mutation in a breast cancer, early onset…
- Conditions
- Breast Adenocarcinoma, Deleterious BRCA1 Gene Mutation, Deleterious BRCA2 Gene Mutation, HER2/Neu Negative
- Trial name
- NCT02282345
- Testing
- Laboratory Biomarker Analysis, Talazoparib
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- M.D. Anderson Cancer Center
Chronic Lymphocytic Leukemia (CLL)
A Study Evaluating the Impact of Venetoclax on the Quality of Life for Subjects With Relapsed (Your Cancer Has Come Back) or Refractory (no Response to Previous Cancer Treatments) Chronic Lymphocytic Leukemia (CLL) While Receiving Venetoclax Monotherapy (a Single Agent).
The purpose of this open-label, single-arm study was to evaluate the impact of venetoclax on the quality of life of participants including those with with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL; a type of…
- Trial name
- VENICE II (NCT02980731)
- Testing
- Venetoclax
- Where
- 33 sites in Argentina, Australia and 8 more
- Volunteers
- 210 people
- Sponsor
- AbbVie
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Malignant Solid Neoplasm
MPACT Study to Compare Effects of Targeted Drugs on Tumor Gene Variations
This phase II trial studies molecular profiling-based assignment of cancer therapy (MPACT) in treating patients with solid tumors that have spread to other places in the body and usually cannot be cured or controlled with…
- Trial name
- NCT01827384
- Testing
- Adavosertib, Carboplatin, Everolimus
- Where
- 8 sites in United States
- Volunteers
- 208 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Esophageal Carcinoma, Esophagogastric Junction Carcinoma
Study of Pembrolizumab (MK-3475) Versus Investigator's Choice Standard Therapy for Participants With Advanced Esophageal/ Esophagogastric Junction Carcinoma That Progressed After First-Line Therapy (MK-3475-181/KEYNOTE-181)
In this study, participants with advanced or metastatic adenocarcinoma or squamous cell carcinoma of the esophagus or Siewert type I adenocarcinoma of the esophagogastric junction (EGJ) that had progressed after first-line…
- Trial name
- NCT02564263
- Testing
- pembrolizumab, paclitaxel, docetaxel
- Volunteers
- 628 people
- Sponsor
- Merck Sharp & Dohme LLC
Rheumatoid Arthritis
Rheumatoid Arthritis Treatment and Biopsy Study Assessing Certolizumab Pegol (Cimzia)
The purpose of this study is to conduct an Investigator-Sponsored Study to determine the potential immunomodulatory effects of Certolizumab Pegol (CZP) treatment at the site of disease activity (synovial lining) in subjects with…
- Trial name
- NCT01374971
- Testing
- Certolizumab Pegol (CZP), Arthroscopic synovial tissue biopsy
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Nathan Wei, MD, FACP, FACR:
Prostatic Neoplasms
A Study of Niraparib in Combination With Abiraterone Acetate and Prednisone Versus Abiraterone Acetate and Prednisone for the Treatment of Participants With Deleterious Germline or Somatic Homologous Recombination Repair (HRR) Gene-MutatedProstatic Neoplasms (mCSPC)
The purpose of the study is to determine if the combination of niraparib with Abiraterone Acetate (AA) plus prednisone compared with AA plus prednisone in participants with deleterious germline or somatic Homologous Recombination…
- Trial name
- AMPLITUDE (NCT04497844)
- Testing
- Niraparib+ Abiraterone acetate fixed dose combination (FDC), Abiraterone acetate (AA), Prednisone
- Where
- 387 sites in United States, Argentina and 31 more
- Volunteers
- 696 people
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Idiopathic Parkinson's Disease
Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease.
- Trial name
- NCT00985517
- Testing
- CERE-120: Adeno-Associated Virus Delivery of Neurturin
- Where
- 11 sites in United States
- Volunteers
- 57 people
- Sponsor
- Sangamo Therapeutics