Clinical trials: Gene Therapy
Advanced Cancer, Breast Cancer
First-in-Human Study of OKI-219 in Advanced Solid Tumors and Advanced Breast Cancer
OKI-219-101 is a Phase 1a/1b, open-label, multicenter, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of OKI-219 as monotherapy and in combination…
- Conditions
- Advanced Cancer, Breast Cancer, Advanced Solid Tumors, PI3K Gene Mutation
- Trial name
- PIKture-01 (NCT06239467)
- Testing
- OKI-219, Fulvestrant, Trastuzumab
- Where
- 33 sites in United States, Belgium and 4 more
- Volunteers
- 200 people
- Sponsor
- OnKure, Inc.
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Observational
This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing…
- Trial name
- NCT04591834
- Testing
- Observational
- Where
- 3 sites in United States, Canada
- Sponsor
- REGENXBIO Inc.
Osteoarthritis, Knee
A Long Term Follow-up Study of Subjects Who Received ICM-203 or Matching Placebo
This is an observational study of the long term safety and efficacy of ICM-203.
- Trial name
- NCT05752032
- Testing
- ICM-203
- Where
- 2 sites in Australia
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- ICM Co. Ltd.
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the…
- Trial name
- NCT01474343
- Testing
- SAF-301
- Where
- 2 sites in France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Parkinson Disease, Parkinson's Disease
Phase 1 Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of IPS101A in Parkinson's Disease Patients
The purpose of this study is to evaluate the dose-related safety and tolerability of IPS101A, an adeno-associated virus (AAV) gene therapy, in patients with Parkinson's disease who exhibit severe functional impairment…
- Trial name
- NCT07371338
- Testing
- IPS101A
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Innopeutics Corporation
Bietti Crystalline Dystrophy, Inherited Retinal Diseases
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects.
- Trial name
- NCT06699108
- Testing
- VGR-R01
- Where
- China
- Volunteers
- 45 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Myelodysplastic Syndromes (MDS), Acute Myeloid Leukaemia (AML)
A Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients
This is a Phase I/II trial to determine safety, clinical efficacy and feasibility of a gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML).
- Trial name
- NCT02550535
- Testing
- Autologous WT1 TCR transduced T cells
- Where
- 6 sites in Belgium, Germany and 1 more
- Volunteers
- 3 people
- Sponsor
- Cell Medica Ltd
Adverse Drug Events, Adverse Drug Reactions
YouScript IMPACT Registry
This multicenter observational study aims to investigate the benefits of providing pharmacogenetic testing with the YouScript Personalized Prescribing System which includes a clinical decision support tool and individualized…
- Conditions
- Adverse Drug Events, Adverse Drug Reactions, Drug Interaction Potentiation, Drug Metabolism, Poor, CYP2D6-RELATED
- Trial name
- IMPACT (NCT02191358)
- Where
- 7 sites in United States
- Volunteers
- 800 people
- Sponsor
- Genelex Corporation
Non Hodgkin Lymphoma, Refractory Diffuse Large B Cell Lymphoma (DLBCL)
Lentiviral Gene Therapy (Zamtocabtagene Autoleucel) LTFU
This is an observational long-term follow-up (LTFU) study for subjects who previously received zamtocabtagene autoleucel, known as MB-CART2019.1.
- Conditions
- Non Hodgkin Lymphoma, Refractory Diffuse Large B Cell Lymphoma (DLBCL), Relapsed Diffuse Large B Cell Lymphoma, High Grade B-cell Lymphoma
- Trial name
- NCT06116110
- Testing
- Long-term Follow-Up
- Where
- 11 sites in United States
- Volunteers
- 150 people
- Sponsor
- Miltenyi Biomedicine GmbH
HR-positive, HER2-negative Breast Cancer, TNBC - Triple-Negative Breast Cancer
Gene-edited T Regulatory Cells (CRG-150) for the Treatment of Relapsed/Refractory Malignancies
The goal of this Phase 1/2a interventional study is to evaluate the safety and tolerability of CRG-150 in relapsed/refractory HR+HER2- breast cancer, Triple Negative Breast Cancer (TNBC) and prostate cancer.
- Conditions
- HR-positive, HER2-negative Breast Cancer, TNBC - Triple-Negative Breast Cancer, Prostate Cancer
- Trial name
- NCT07730125
- Testing
- CRG-150
- Volunteers
- 67 people
- Sponsor
- CoRegen, Inc.
Acute Myeloblastic Leukaemia, Leukemia, T-Cell
Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).
This Phase I/IIa clinical study is testing an experimental treatment called GYA01 (CART84) for people with acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia (T-ALL) whose disease has come back after treatment…
- Trial name
- NCT07471789
- Testing
- CART84
- Where
- 2 sites in Spain
- Volunteers
- 33 people
- Sponsor
- Gyala Therapeutics
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.
X-Linked Retinitis Pigmentosa
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants…
- Trial name
- NCT04850118
- Testing
- rAAV2tYF-GRK1-hRPGRco, Control
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 85 people
- Sponsor
- Beacon Therapeutics
Degenerative Arthritis
Efficacy and Safety Study of TissueGene-C to Degenerative Arthritis
To assess the efficacy and safety of the intra-articular injection of TissueGene-C in patients with degenerative arthritis of the knee
- Trial name
- NCT02341378
- Testing
- TissueGene-C(Low dose), TissueGene-C(High dose)
- Where
- 3 sites in South Korea
- Volunteers
- 28 people
- Sponsor
- Kolon Life Science
Rett Syndrome
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
- Trial name
- ASPIRE (NCT07480564)
- Testing
- TSHA-102
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Taysha Gene Therapies, Inc.
Lung Neoplasm, Carcinoma, Non-Small-Cell Lung
(HARMONY) Study of BLU-701 in EGFR-mutant NSCLC
This is a Phase 1/2, open-label, first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of BLU-701 as monotherapy or in combination with either…
- Conditions
- Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Neoplasms
- Trial name
- NCT05153408
- Testing
- BLU-701, osimertinib, carboplatin
- Where
- 7 sites in United States
- Volunteers
- 20 people
- Sponsor
- Blueprint Medicines Corporation
Transfusion-dependent Beta-Thalassemia
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent…
- Trial name
- NCT02633943
- Testing
- Safety and efficacy assessments
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Genetix Biotherapeutics Inc.
X-Linked Retinitis Pigmentosa
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Trial name
- NCT04671433
- Testing
- Genetic: AAV5-hRKp.RPGR
- Where
- 28 sites in United States, Belgium and 9 more
- Volunteers
- 105 people
- Sponsor
- MeiraGTx Ocular UK Ltd