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Results for “Genetic Therapy”

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Enrolling by Invitation

Showing 20 of 0 results

Not applicableStudy completedNCT00446862
What this trial is testing

The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 36
Not applicableEnded earlyNCT01144741
What this trial is testing

Survey Study and Records Review of Treatment Outcomes in Freeman-Sheldon Syndrome

Who this might be right for
Freeman-Sheldon SyndromeArthrogryposis Distal Type 2AWhistling Face Syndrome+13 more
Freeman-Sheldon Research Group, Inc. 2
Testing effectiveness (Phase 2)Active Not RecruitingNCT06308159
What this trial is testing

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

Who this might be right for
Beta-Thalassemia
Lantu Biopharma 6
Large-scale testing (Phase 3)Study completedNCT01145586
What this trial is testing

A Non-inferiority, Multicenter and Randomized, Multiple-Dose Study About a Treatment to Hypolactasia

Who this might be right for
Lactose Intolerance
Eurofarma Laboratorios S.A. 140
Testing effectiveness (Phase 2)Study completedNCT02428296
What this trial is testing

Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation

Who this might be right for
PIK3CA-Related Overgrowth Spectrum (PROS)Growth DisorderGenetics
National Human Genome Research Institute (NHGRI) 39
Early research (Phase 1)Study completedNCT04551950
What this trial is testing

Bintrafusp Alfa Combination Therapy in Participants With Cervical Cancer (INTR@PID 046)

Who this might be right for
Cervical Cancer
EMD Serono Research & Development Institute, Inc. 25
Testing effectiveness (Phase 2)Active Not RecruitingNCT06314490
What this trial is testing

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Who this might be right for
Genetic Disease
University of California, San Diego 1
Testing effectiveness (Phase 2)Looking for participantsNCT06290713
What this trial is testing

Vasodilator and Exercise Study for DMD (VASO-REx)

Who this might be right for
Duchenne Muscular DystrophyDuchenne DiseaseMuscular Dystrophy+4 more
University of Florida 50
Not applicableStudy completedNCT00695279
What this trial is testing

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Who this might be right for
Severe Combined ImmunodeficiencyMalignancy, HematologicNeuroblastoma+2 more
St. Jude Children's Research Hospital 41
Testing effectiveness (Phase 2)Enrolling By InvitationNCT07640503
What this trial is testing

Lamivudine as a Novel Clinical Effort for Rett Syndrome

Who this might be right for
Rett Syndrome
Maria Denise Fernandes Carvalho de Andrade 10
Not applicableStudy completedNCT04694456
What this trial is testing

Pro-inflammatory Cytokines in Facioscapulohumeral Muscular Dystrophy (CYTOKINE-FSH)

Who this might be right for
Muscular Dystrophies
Centre Hospitalier Universitaire de Nice 30
Not applicableStudy completedNCT01053156
What this trial is testing

Trial of Minocycline to Treat Children With Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
University of California, Davis 66
Not applicableAvailableNCT07250737
What this trial is testing

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

Who this might be right for
Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal Diseases+6 more
Avidity Biosciences, Inc.
Large-scale testing (Phase 3)Not Yet RecruitingNCT07823595
What this trial is testing

Abrocitinib Treatment of Atopic Dermatitis in Children 2 to Less Than 6 Years of Age

Who this might be right for
Atopic Dermatitis
Pfizer 90
Post-approval studies (Phase 4)Study completedNCT02500641
What this trial is testing

Intensive Urate Lowering Therapy of Febuxostat Compared to Allopurinol on Cardiovascular Risk in Patients With Gout

Who this might be right for
Gout
Menarini International Operations Luxembourg SA 196
Not applicableUnknownNCT03584841
What this trial is testing

Implementation of a Non-invasive Version of the Imaging β-adrenergic-dependent Sweat Secretion Test

Who this might be right for
Mucoviscidosis Involving the Lung
Cliniques universitaires Saint-Luc- Université Catholique de Louvain 120
Not applicableStudy completedNCT00562705
What this trial is testing

Effects of Growth Hormone (GH) Treatment on Eating Regulation

Who this might be right for
Short Stature
Rabin Medical Center 32
Very early researchNot Yet RecruitingNCT06293729
What this trial is testing

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

Who this might be right for
Refractory HypercholesterolemiaFamilial Hypercholesterolemia
Suzhou Municipal Hospital 9
Testing effectiveness (Phase 2)Study completedNCT07095712
What this trial is testing

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

Who this might be right for
Amyotrophic Lateral Sclerosis
n-Lorem Foundation 1
Testing effectiveness (Phase 2)Study completedNCT00798369
What this trial is testing

Targeted Dose Finding of Canakinumab (ACZ885) for Management of Acute Flare in Refractory or Contraindicated Gout Patients

Who this might be right for
Acute Gout
Novartis 200
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