Clinical trials: Gene Therapy
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics
MPS IIIA, Sanfilippo Syndrome
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT04088734
- Testing
- ABO-102
- Where
- 3 sites in United States, Australia and 1 more
- Volunteers
- 5 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Breast Cancer Metastatic, BRCA 1 Gene Mutation
Assessment of the Efficacy and Safety of Olaparib Monotherapy Versus Physicians Choice Chemotherapy in the Treatment of Metastatic Breast Cancer Patients With Germline BRCA1/2 Mutations.
This open label, randomised, controlled, multi-centre phase III study will assess the efficacy and safety of single agent olaparib vs standard of care based on physician's choice of capecitabine, vinorelbine or eribulin in…
- Conditions
- Breast Cancer Metastatic, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation
- Trial name
- OlympiAD (NCT02000622)
- Testing
- Olaparib, Physician's choice chemotherapy
- Where
- 171 sites in United States, Bulgaria and 17 more
- Volunteers
- 302 people
- Sponsor
- AstraZeneca
Parkinson's Disease
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of…
- Trial name
- NCT05603312
- Testing
- AAV-GAD Low Dose, AAV-GAD High Dose
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- MeiraGTx, LLC
Cystic Fibrosis
Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR…
- Trial name
- NCT01946412
- Testing
- Ivacaftor
- Where
- 16 sites in United States, Canada and 1 more
- Volunteers
- 33 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Carcinoma, Non-Small-Cell Lung, Lung Diseases
Sitravatinib Plus Pembrolizumab in Patients With Advanced Treatment-Naïve PD-L1+ Non-Squamous NSCLC
This is a multicohort phase 2 study to evaluate the efficacy of pembrolizumab combined with the investigational drug sitravatinib in the frontline treatment of advanced, non-squamous PD-L1 positive NSCLC.
- Conditions
- Carcinoma, Non-Small-Cell Lung, Lung Diseases, Lung Neoplasms, Metastatic Lung Non-Small Cell Carcinoma
- Trial name
- NCT04925986
- Testing
- Sitravatinib, Pembrolizumab
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Sarah Goldberg
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
Trigeminal Neuropathy
Erenumab-aooe for the Management of Trigeminal Neuropathic Pain.
This study is a single center, placebo-controlled, double blind, randomized, phase II pilot to evaluate the efficacy of erenumab-aooe in the management of trigeminal neuropathic pain comparing erenumab-aooe vs Placebo.
- Trial name
- NCT05142228
- Testing
- Erenumab-Aooe
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Maryland, Baltimore
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
Recurrent Acute Leukemia of Ambiguous Lineage, Recurrent Acute Lymphoblastic Leukemia
High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia
This pilot clinical trial studies the feasibility of choosing treatment based on a high throughput ex vivo drug sensitivity assay in combination with mutation analysis for patients with acute leukemia that has returned after a…
- Conditions
- Recurrent Acute Leukemia of Ambiguous Lineage, Recurrent Acute Lymphoblastic Leukemia, Recurrent Acute Myeloid Leukemia, Refractory Acute Lymphoblastic Leukemia
- Trial name
- NCT02551718
- Testing
- Chemosensitivity Assay, Cytology Specimen Collection Procedure, Gene Expression Analysis
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- University of Washington
FGFR1 Gene Amplification, FGFR1 Gene Mutation
Pemigatinib for the Treatment of Metastatic or Unresectable Colorectal Cancer Harboring FGFR Alterations
This phase II trial studies how well pemigatinib works in treating patients with colorectal cancer with mutations (alterations) in a FGFR gene and that has spread to other places in the body (metastatic) or cannot be removed by…
- Conditions
- FGFR1 Gene Amplification, FGFR1 Gene Mutation, FGFR1 Gene Translocation, FGFR2 Gene Amplification
- Trial name
- NCT04096417
- Testing
- Pemigatinib, Quality-of-Life Assessment
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Academic and Community Cancer Research United
Chronic Myeloid Leukemia in Chronic Phase
Efficacy and Safety of LBH589 in Adult Patients With Refractory Chronic Myeloid Leukemia (CML) in Chronic Phase
This study will evaluate the efficacy and safety of LBH589B in adult patients with chronic phase chronic myeloid leukemia with resistant disease following treatment with at least two BCR-ABL tyrosine kinase inhibitors
- Trial name
- NCT00451035
- Testing
- LBH589
- Where
- 10 sites in Belgium, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Cholangiocarcinoma, FGFR2 Gene Rearrangements
Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements
This is an open-label, multinational, parallel 2-arm, randomized Phase 3 study evaluating the efficacy and safety of futibatinib versus gemcitabine-cisplatin chemotherapy as first-line treatment of participants with advanced…
- Trial name
- FOENIX-CCA3 (NCT04093362)
- Testing
- Futibatinib, Cisplatin, Gemcitabine
- Where
- 103 sites in United States, Argentina and 18 more
- Volunteers
- 10 people
- Sponsor
- Taiho Oncology, Inc.
Multiple Myeloma
A Phase I/II Study Evaluating Temferon in Multiple Myeloma Patients With Early Relapse After Front Line Therapy (TEM-MM)
This is a non-randomized, open label, phase I/II, dose-escalation study, involving a single injection of Temferon, an investigational advanced therapy consisting of autologous CD34+-enriched hematopoietic stem and progenitor…
- Trial name
- NCT03875495
- Testing
- Temferon
- Where
- Italy
- Volunteers
- 9 people
- Sponsor
- Genenta Science
Recurrent Glioblastoma Multiforme, Progressive Glioblastoma Multiforme
Genetically Engineered HSV-1 Phase 1 Study for the Treatment of Recurrent Malignant Glioma
To determine the safety and tolerability of the maximum dose for laboratory engineered Herpes Simplex Virus-1 in patients who would not be eligible for surgical resection of recurrent glioma To determine the safety and…
- Conditions
- Recurrent Glioblastoma Multiforme, Progressive Glioblastoma Multiforme, Anaplastic Astrocytoma or Gliosarcoma
- Trial name
- M032-HSV-1 (NCT02062827)
- Testing
- M032 (NSC 733972)
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Alabama at Birmingham
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
Melanoma, Skin Cancer
Phase II Study of Metastatic Melanoma With Lymphodepleting Conditioning and Infusion of Anti-MART-1 F5 TCR-Gene-Engineered Lymphocytes
Background: Human peripheral blood lymphocytes have been engineered to express a T-cell receptor (TCR) that recognizes a blood type,HLA-A 0201 (human leukocyte antigen) derived from the gp100 protein.
- Trial name
- NCT00509288
- Testing
- autologous anti-MART-1 F5 T-cell receptor, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Gaucher Disease
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
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
- NCT00478647
- Testing
- GA-GCB (velaglucerase alfa)
- Where
- 15 sites in United States, Israel and 3 more
- Volunteers
- 40 people
- Sponsor
- Shire
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies