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Results for “Inherited Genetic Conditions (Diagnosis)”

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Showing 20 of 0 results

Not applicableStudy completedNCT00004831
What this trial is testing

Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Who this might be right for
Erythropoietic Protoporphyria
FDA Office of Orphan Products Development 20
Testing effectiveness (Phase 2)Study completedNCT05059327
What this trial is testing

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

Who this might be right for
Tuberous Sclerosis Complex
Noema Pharma AG 61
Not applicableStudy completedNCT02654886
What this trial is testing

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Who this might be right for
Pompe DiseaseMuscle Weakness
University of California, Irvine 10
Not applicableStudy completedNCT03876522
What this trial is testing

Natural History and Functional Status Study of Patients With Lafora Disease

Who this might be right for
Lafora Disease
Ionis Pharmaceuticals, Inc. 33
Not applicableStudy completedNCT05274646
What this trial is testing

Impact on Risk Stratification of Overlap Syndrome Phenotype in Patients With E1784K Mutation in SCN5A

Who this might be right for
Long QT SyndromeBrugada SyndromeCardiac Conduction System Disease+1 more
Centre Hospitalier Universitaire de la Réunion 47
Not applicableLooking for participantsNCT05354622
What this trial is testing

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

Who this might be right for
Hereditary Spastic ParaplegiaNeurodegenerative DiseasesPediatric Disorder+3 more
Boston Children's Hospital 200
Testing effectiveness (Phase 2)Study completedNCT01223222
What this trial is testing

Evaluate Safety, Tolerability and Efficacy of Lytixar™ (LTX-109) on Uncomplicated, Gram-positive, Skin Infection

Who this might be right for
Gram-positive, Skin InfectionsMild Eczema/DermatosesAtopic Dermatitis
Lytix Biopharma AS 24
Testing effectiveness (Phase 2)WithdrawnNCT02900183
What this trial is testing

Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels

Who this might be right for
Alpha-1 Antitrypsin Deficiency
Arrowhead Pharmaceuticals
Testing effectiveness (Phase 2)Looking for participantsNCT07280156
What this trial is testing

Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)

Who this might be right for
Gout
Protalix 150
Testing effectiveness (Phase 2)Study completedNCT00872729
What this trial is testing

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 9
Not applicableStudy completedNCT01405859
What this trial is testing

MRI Study of Tic Remission in Tourette Syndrome

Who this might be right for
Tourette Syndrome
University of Utah 21
Not applicableUnknownNCT02144870
What this trial is testing

Efficacy of Psychotherapy Treatment of Children With Tics

Who this might be right for
Tic-Disorder
University of Cologne 100
Not applicableStudy completedNCT02895841
What this trial is testing

Patient-Empowered Mobile Technology in Hospitalized Patients (TRU-PAIN)

Who this might be right for
Pain
Duke University 174
Large-scale testing (Phase 3)Study completedNCT01467362
What this trial is testing

Efficacy Emollient on Xerosis in Children With Atopic Dermatitis

Who this might be right for
Atopic Dermatitis
Pierre Fabre Medicament 251
Testing effectiveness (Phase 2)WithdrawnNCT01381003
What this trial is testing

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)

Who this might be right for
Granulomatous Disease, Chronic, X-linked, Variant
Great Ormond Street Hospital for Children NHS Foundation Trust
Not applicableStudy completedNCT01404624
What this trial is testing

Characterization of Transcriptional Regulators of Ghrelin Hormone Which Causes Genetic Obesity

Who this might be right for
Prader Willi SyndromeObesity
Samsung Medical Center 58
Testing effectiveness (Phase 2)Looking for participantsNCT06526923
What this trial is testing

A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

Who this might be right for
Cystic Fibrosis
Spirovant Sciences, Inc. 15
Not applicableLooking for participantsNCT07417137
What this trial is testing

A Natural History Study of Angelman Syndrome

Who this might be right for
Angelman SyndromeNeurodevelopmental Conditions
Massachusetts General Hospital 40
Testing effectiveness (Phase 2)UnknownNCT02933437
What this trial is testing

The Response To Ajmaline Provocation in Healthy Subjects

Who this might be right for
Brugada SyndromeSudden Death
St George's, University of London 100
Not applicableLooking for participantsNCT07014007
What this trial is testing

Tolerance and Efficacy Open-label Study With RV4421B, in Various Populations With Atopic Dermatitis for 12 Weeks

Who this might be right for
Atopic Dermatitis
Pierre Fabre Dermo Cosmetique 105
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