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Results for “Genetic Therapy”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Ended earlyNCT03605069
What this trial is testing

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

Who this might be right for
Epidermolysis Bullosa Dystrophica, RecessiveEpidermolysis Bullosa Dystrophica, Dominant
Phoenicis Therapeutics 2
Testing effectiveness (Phase 2)Study completedNCT01573754
What this trial is testing

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

Who this might be right for
Porphyria Cutanea Tarda
The University of Texas Medical Branch, Galveston 48
Testing effectiveness (Phase 2)Study completedNCT01458470
What this trial is testing

A Trial of Memantine as Symptomatic Treatment for Early Huntington Disease

Who this might be right for
Huntington Disease
University of British Columbia 19
Large-scale testing (Phase 3)Active Not RecruitingNCT05269394
What this trial is testing

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)

Who this might be right for
Alzheimers DiseaseDementiaAlzheimers Disease, Familial
Washington University School of Medicine 197
Testing effectiveness (Phase 2)Active Not RecruitingNCT04551170
What this trial is testing

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Who this might be right for
PseudohypoparathyroidismAlbright Hereditary OsteodystrophyPseudohypoparathyroidism Type 1a
Vanderbilt University Medical Center 34
Not applicableWithdrawnNCT02127905
What this trial is testing

Unrelated HSCT in Patients With Fanconi Anemia

Who this might be right for
Fanconi Anemia
Neena Kapoor, M.D.
Not applicableStudy completedNCT00874172
What this trial is testing

Effectiveness of New Analgesic Strategy Compared to the Usal Antalgic Strategy

Who this might be right for
Sickle Cell Anemia
Assistance Publique - Hôpitaux de Paris 176
Testing effectiveness (Phase 2)Study completedNCT01771731
What this trial is testing

Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease

Who this might be right for
Sickle Cell Disease
University of California, San Francisco 27
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableUnknownNCT05523986
What this trial is testing

Vitamin D Treatment Effect for Atopic Dermatitis in Children

Who this might be right for
Atopic Dermatitis
China Medical University Hospital 300
Not applicableStudy completedNCT02723305
What this trial is testing

Cardiometabolic Profiles of Boys With Klinefelter Syndrome

Who this might be right for
Klinefelter Syndrome47,XXYSex Chromosome Aneuploidy+1 more
University of Colorado, Denver 31
Testing effectiveness (Phase 2)Study completedNCT04945772
What this trial is testing

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

Who this might be right for
Retinitis PigmentosaRetinitisRetinal Diseases+4 more
Nanoscope Therapeutics Inc. 27
Testing effectiveness (Phase 2)Study completedNCT04571970
What this trial is testing

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 6
Testing effectiveness (Phase 2)WithdrawnNCT01932554
What this trial is testing

Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain Crisis

Who this might be right for
Sickle Cell DiseaseHb-SS Disease With Vasoocclusive PainHemoglobin SS Disease With Vasoocclusive Crisis+2 more
St. Louis University
Post-approval studies (Phase 4)Study completedNCT02519556
What this trial is testing

Trial on Effectiveness Combined Probiotics in Atopic Dermatitis in Children

Who this might be right for
Atopic Dermatitis
Casa Espirita Terra de Ismael 60
Post-approval studies (Phase 4)UnknownNCT00103753
What this trial is testing

Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

Who this might be right for
Beta-Thalassemia
Royal Brompton & Harefield NHS Foundation Trust 65
Early research (Phase 1)Looking for participantsNCT05860569
What this trial is testing

Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis

Who this might be right for
Hypertriglyceridemia, Familial
GeneCradle Inc 7
Testing effectiveness (Phase 2)Active Not RecruitingNCT04439344
What this trial is testing

Testing Binimetinib as a Potential Targeted Treatment in Cancers With NRAS Genetic Changes (MATCH-Subprotocol Z1A)

Who this might be right for
Advanced LymphomaAdvanced Malignant Solid NeoplasmHematopoietic and Lymphoid Cell Neoplasm+3 more
National Cancer Institute (NCI) 53
Large-scale testing (Phase 3)UnknownNCT01809444
What this trial is testing

Prednisone Versus Doxycycline in the Treatment of Graves' Orbitopathy

Who this might be right for
Thyroid Associated Opthalmopathies
Sun Yat-sen University 146
Large-scale testing (Phase 3)Looking for participantsNCT07223944
What this trial is testing

A Gaucher Disease Gene Therapy Trial With FLT201

Who this might be right for
Gaucher Disease Type 1
Spur Therapeutics 45
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