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Results for “Genetic Therapy”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT00059280
What this trial is testing

The Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
Testing effectiveness (Phase 2)UnknownNCT02175589
What this trial is testing

Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation

Who this might be right for
Familial Mediterranean Fever
Rambam Health Care Campus 80
Early research (Phase 1)Ended earlyNCT03685591
What this trial is testing

PF-06952229 Treatment in Adult Patients With Advanced Solid Tumors

Who this might be right for
Breast NeoplasmsProstate NeoplasmsNeoplasms, Squamous Cell+6 more
Pfizer 49
Early research (Phase 1)Ended earlyNCT02574910
What this trial is testing

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

Who this might be right for
Congenital Adrenal Hyperplasia
University of Texas Southwestern Medical Center 4
Not applicableUnknownNCT02614820
What this trial is testing

The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
General Hospital of Chinese Armed Police Forces 100
Not applicableAvailableNCT06931262
What this trial is testing

Expanded Access Treatment Protocol With DCA for Patients With PDCD

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Saol Therapeutics Inc
Not applicableUnknownNCT05463562
What this trial is testing

Multimodal Biomarkers of Electroconvulsive Therapy in Severe and Treatment-resistant Depression

Who this might be right for
Depressive DisorderBipolar Disorder
Max-Planck-Institute of Psychiatry 134
Not applicableStudy completedNCT03771235
What this trial is testing

Online Mindfulness-based Tic Reduction (Phase Two)

Who this might be right for
Tourette SyndromePersistent Tic Disorder
Bowdoin College 37
Early research (Phase 1)Active Not RecruitingNCT04737460
What this trial is testing

Study for the Treatment for CLN7 Disease

Who this might be right for
CLN7
Benjamin Greenberg 4
Testing effectiveness (Phase 2)Study completedNCT00454818
What this trial is testing

Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure

Who this might be right for
Heart Failure, CongestiveDilated Cardiomyopathy
Celladon Corporation 51
Testing effectiveness (Phase 2)WithdrawnNCT02535962
What this trial is testing

Probiotics and Corticosteroids for Treating Periodic Fever, Aphthous Stomatitis, Pharyngitis, Cervical Adenitis (PFAPA)

Who this might be right for
Periodic FeverAphthous StomatitisPharyngitis+1 more
Connecticut Children's Medical Center
Not applicableUnknownNCT04775888
What this trial is testing

Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies

Who this might be right for
Health Services AccessibilityBlood Coagulation Factor Deficiencies
RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône 500
Not applicableStudy completedNCT04440410
What this trial is testing

Evaluation of Cutaneous and Circulating Inflammatory Biomarkers in Hidradenitis Suppurativa and Atopic Dermatitis

Who this might be right for
Hidradenitis SuppurativaDermatitis, Atopic
Kymera Therapeutics, Inc. 40
Large-scale testing (Phase 3)Ended earlyNCT01425307
What this trial is testing

Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea

Who this might be right for
Sickle Cell Anemia
Children's Hospital Medical Center, Cincinnati 159
Not applicableLooking for participantsNCT06305234
What this trial is testing

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 200
Post-approval studies (Phase 4)UnknownNCT02525172
What this trial is testing

Immune Modulation Therapy for Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 8
Testing effectiveness (Phase 2)Active Not RecruitingNCT02650401
What this trial is testing

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

Who this might be right for
Solid TumorsCNS Tumors
Hoffmann-La Roche 69
Not applicableStudy completedNCT03875924
What this trial is testing

REAL-LIFE DATA OF CONSTITUTIONAL VON WILLEBRAND DISEASE IN WESTERN FRANCE (HOPSCOTcH-WILL)

Who this might be right for
Von Willebrand Diseases
Nantes University Hospital 926
Not applicableStudy completedNCT01132079
What this trial is testing

Pimecrolimus and Epidermal Barrier Function

Who this might be right for
Atopic Dermatitis
University of Kiel 15
Testing effectiveness (Phase 2)Not Yet RecruitingNCT05126888
What this trial is testing

SCI-110 in the Treatment of Tourette Syndrome

Who this might be right for
Tourette Syndrome
Neurothera Labs Inc. 164
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