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Results for “Genetic Therapy”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT07222371
What this trial is testing

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

Who this might be right for
Genetic DiseaseTUBB4A-Related Leukodystrophy
University of California, San Diego 1
Large-scale testing (Phase 3)Study completedNCT00945009
What this trial is testing

Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor

Who this might be right for
Adult Kidney Wilms TumorBeckwith-Wiedemann SyndromeChildhood Kidney Wilms Tumor+7 more
Children's Oncology Group 249
Testing effectiveness (Phase 2)Active Not RecruitingNCT07223632
What this trial is testing

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

Who this might be right for
Charcot Marie Tooth Disease (CMT)Neuromuscular Diseases (NMD)
Vanda Pharmaceuticals 1
Testing effectiveness (Phase 2)Study completedNCT00876863
What this trial is testing

Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease

Who this might be right for
Alzheimer's Disease
Sangamo Therapeutics 49
Testing effectiveness (Phase 2)Study completedNCT01307098
What this trial is testing

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Alexion Pharmaceuticals, Inc. 9
Testing effectiveness (Phase 2)Ended earlyNCT01104298
What this trial is testing

Doxorubicin vs. Trabectedin Plus Doxorubicin in Non Operable and/or Metastatic STS

Who this might be right for
Sarcoma
Grupo Espanol de Investigacion en Sarcomas 115
Not applicableUnknownNCT02441387
What this trial is testing

Predictors of the Outcome of Late Life Depression

Who this might be right for
Major Depression
University of Sao Paulo General Hospital 120
Large-scale testing (Phase 3)Looking for participantsNCT07232134
What this trial is testing

The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia

Who this might be right for
AML (Acute Myelogenous Leukemia)Down Syndrome (DS)
Federal Research Institute of Pediatric Hematology, Oncology and Immunology 100
Large-scale testing (Phase 3)Study completedNCT05139316
What this trial is testing

Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Who this might be right for
Glycogen Storage Disease Type IA
Ultragenyx Pharmaceutical Inc 49
Testing effectiveness (Phase 2)Study completedNCT00532844
What this trial is testing

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Who this might be right for
Endothelial Dysfunction
BioMarin Pharmaceutical 52
Testing effectiveness (Phase 2)Study completedNCT04416516
What this trial is testing

Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor

Who this might be right for
Basal Cell CarcinomaBasal Cell Nevus Syndrome
Ascend Biopharmaceuticals Ltd 21
Not applicableUnknownNCT03847532
What this trial is testing

MUTYH-associated Polyposis

Who this might be right for
MutYH-associated Polyposis
State Scientific Centre of Coloproctology, Russian Federation 25
Post-approval studies (Phase 4)Study completedNCT00180141
What this trial is testing

Elidel-Study: Elidel in Patients With Atopic Dermatitis

Who this might be right for
Atopic Dermatitis
Technische Universität Dresden 24
Testing effectiveness (Phase 2)Study completedNCT00029731
What this trial is testing

Arginine Treatment of Acute Chest Syndrome (Pneumonia) in Sickle Cell Disease Patients

Who this might be right for
Anemia, Sickle CellPneumonia
UCSF Benioff Children's Hospital Oakland 30
Not applicableActive Not RecruitingNCT03281902
What this trial is testing

Genetic Analysis in Blood and Tumor Samples From Patients With Advanced or Metastatic Estrogen Receptor Positive and HER2 Negative Breast Cancer Receiving Palbociclib and Endocrine Therapy

Who this might be right for
Advanced Breast CarcinomaLocally Advanced Breast CarcinomaMetastatic Breast Carcinoma+6 more
Mayo Clinic 68
Post-approval studies (Phase 4)Ended earlyNCT03661983
What this trial is testing

Trial to Evaluate the Long-term Efficacy of Oral Aripiprazole in the Treatment of Pediatric Participants With Tourette's Disorder

Who this might be right for
Tourette's Disorder (TD)
Otsuka Pharmaceutical Development & Commercialization, Inc. 36
Not applicableUnknownNCT06355622
What this trial is testing

Prevalence and Characterization of Pain in RASopathies

Who this might be right for
RASopathyCostello SyndromeCardio-Facio-Cutaneous Syndrome+1 more
Fondazione Policlinico Universitario Agostino Gemelli IRCCS 150
Not applicableWithdrawnNCT03642951
What this trial is testing

Multi-site Transcranial Magnetic Stimulation Therapy of the Supplementary Motor Area in Children With Tourette Syndrome

Who this might be right for
Tourette Syndrome
Children's Mercy Hospital Kansas City
Testing effectiveness (Phase 2)Study completedNCT02654483
What this trial is testing

Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients

Who this might be right for
Epidermolysis BullosaPruritus
Jean Yuh Tang 14
Testing effectiveness (Phase 2)Active Not RecruitingNCT04411654
What this trial is testing

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Who this might be right for
Gaucher Disease, Type 2
Prevail Therapeutics 7
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