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Results for “Single-Gene Disease”

391 trials

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Showing 20 of 391 results

Early research (Phase 1)Study completedNCT04813653
What this trial is testing

Cyclosporine in Combination With Carfilzomib and Dexamethasone in Relapsed Multiple Myeloma Refractory to Carfilzomib and High Expression of PPIA Gene in Myeloma Cells

Who this might be right for
Multiple Myeloma in RelapseMultiple Myeloma, Refractory
Tel-Aviv Sourasky Medical Center 3
Not applicableStudy completedNCT02487615
What this trial is testing

Genetical, Anthropometrical and Biochemical Factors Influencing High Risk Subclinical Atherosclerosis

Who this might be right for
Primary Prevention
Instituto Dante Pazzanese de Cardiologia 340
Not applicableUnknownNCT04701229
What this trial is testing

Haploinsufficiency of the RBM22 and SLU7 Genes in Del(5q) Myelodysplastic Syndromes

Who this might be right for
Myelodysplastic SyndromesMyelodysplastic Syndrome With Isolated Del(5Q)Myelodysplastic Syndrome With Del(5Q)
University Hospital, Brest 100
Testing effectiveness (Phase 2)Study completedNCT02132130
What this trial is testing

Safety, Tolerability and Efficacy for CGF166 in Patients With Unilateral or Bilateral Severe-to-profound Hearing Loss

Who this might be right for
Unilateral Severe to Profound Hearing Loss OR Bilateral Severe to Profound Hearing Loss
Novartis Pharmaceuticals 22
Early research (Phase 1)Ended earlyNCT01620801
What this trial is testing

Hemophilia B Gene Therapy With AAV8 Vector

Who this might be right for
Hemophilia B
Spark Therapeutics, Inc. 4
Very early researchLooking for participantsNCT06650319
What this trial is testing

A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease

Who this might be right for
Wilson Disease
Chaohui Yu 18
Testing effectiveness (Phase 2)Study completedNCT01825811
What this trial is testing

Efficacy and Safety Study of TissueGene-C Mixed With Fibrin-glue for the Patients With Degenerative Arthritis

Who this might be right for
Degenerative Arthritis
Kolon Life Science 18
Not applicableUnknownNCT03589079
What this trial is testing

Delineation of Novel Monogenic Disorders in the United Arab Emirates Population

Who this might be right for
Mendelian DisordersGenetic DisorderNovel Mutation+4 more
Imperial College London Diabetes Centre 150
Testing effectiveness (Phase 2)Study completedNCT00789867
What this trial is testing

Single Dose of pGM169/GL67A in CF Patients

Who this might be right for
Cystic Fibrosis
Imperial College London 35
Large-scale testing (Phase 3)UnknownNCT03153293
What this trial is testing

A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy

Who this might be right for
Leber Hereditary Optic Neuropathy
Huazhong University of Science and Technology 159
Not applicableNo Longer AvailableNCT03672968
What this trial is testing

EAP_GS010_single Patient

Who this might be right for
Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
GenSight Biologics
Testing effectiveness (Phase 2)Enrolling By InvitationNCT07113743
What this trial is testing

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Who this might be right for
Chronic Granulomatous Disease (CGD)
National Institute of Allergy and Infectious Diseases (NIAID) 10
Testing effectiveness (Phase 2)Study completedNCT01252641
What this trial is testing

Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects

Who this might be right for
HIVHIV Infection
Sangamo Therapeutics 21
Testing effectiveness (Phase 2)Study completedNCT02550535
What this trial is testing

A Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients

Who this might be right for
Myelodysplastic Syndromes (MDS)Acute Myeloid Leukaemia (AML)
Cell Medica Ltd 3
Not applicableUnknownNCT02502214
What this trial is testing

Feasibility Study of Preimplantation Genetic Diagnosis for Single-gene Disorders

Who this might be right for
Preimplantation Diagnosis
University Hospital, Strasbourg, France 30
Early research (Phase 1)UnknownNCT05514249
What this trial is testing

Treatment of a Single Patient With CRD-TMH-001

Who this might be right for
Duchenne Muscular Dystrophy
Cure Rare Disease, Inc 1
Early research (Phase 1)Study completedNCT01044654
What this trial is testing

Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients

Who this might be right for
HIV InfectionHIV Infections
Sangamo Therapeutics 19
Large-scale testing (Phase 3)Active Not RecruitingNCT03837483
What this trial is testing

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

Who this might be right for
Wiskott-Aldrich Syndrome
Fondazione Telethon 10
Not applicableStudy completedNCT00898495
What this trial is testing

Genes in Predicting Outcome in Patients With Esophageal Cancer Treated With Cisplatin, Radiation Therapy, and Surgery

Who this might be right for
Esophageal Cancer
ECOG-ACRIN Cancer Research Group 118
Early research (Phase 1)Looking for participantsNCT07246941
What this trial is testing

Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

Who this might be right for
Huntington's Disease
Hoffmann-La Roche 40
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