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Results for “Inherited Genetic Conditions (Diagnosis)”

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Showing 20 of 0 results

Not applicableStudy completedNCT02458937
What this trial is testing

Functional Outcomes Following Hip Core Decompression in Younger Participants With Osteonecrosis

Who this might be right for
Osteonecrosis
St. Jude Children's Research Hospital 17
Testing effectiveness (Phase 2)Looking for participantsNCT07227207
What this trial is testing

TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

Who this might be right for
Alpha-1 Antitrypsin Deficiency (AATD)
Tessera Therapeutics, Inc. 72
Early research (Phase 1)Study completedNCT06186492
What this trial is testing

Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)

Who this might be right for
Alpha-1 Antitrypsin Deficiency (AATD)
Wave Life Sciences USA, Inc. 47
Not applicableStudy completedNCT04262284
What this trial is testing

Respreeza® Self-administration and Learning Program (AmAREtTI Study)

Who this might be right for
Alpha-1 Antitrypsin Deficiency
CSL Behring 15
Testing effectiveness (Phase 2)Study completedNCT00742664
What this trial is testing

Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project

Who this might be right for
Prader Willi SyndromeObsessive-compulsive Disorder
University of South Florida 24
Large-scale testing (Phase 3)Study completedNCT02634931
What this trial is testing

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Who this might be right for
Tuberous SclerosisAngiofibromaHypomelanotic Macule+1 more
Nobelpharma 94
Early research (Phase 1)Study completedNCT00012181
What this trial is testing

Flavopiridol in Treating Children With Relapsed or Refractory Solid Tumors or Lymphomas

Who this might be right for
Recurrent Childhood Brain Stem GliomaRecurrent Childhood Cerebellar AstrocytomaRecurrent Childhood Cerebral Astrocytoma+19 more
National Cancer Institute (NCI) 30
Large-scale testing (Phase 3)WithdrawnNCT01245816
What this trial is testing

A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

Who this might be right for
Familial Adenomatous Polyposis
Cancer Prevention Pharmaceuticals, Inc.
Not applicableNot Yet RecruitingNCT06960499
What this trial is testing

Predictors of Physical Activity in Adult Patients With Hemophilic Arthropathy

Who this might be right for
Hemophilia
Universidad Católica San Antonio de Murcia 88
Not applicableStudy completedNCT04239521
What this trial is testing

The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata

Who this might be right for
Alopecia AreataDepressive EpisodeRecurrent Depressive Disorder+33 more
Momentum Data 51,955
Not applicableLooking for participantsNCT07400757
What this trial is testing

Statin and Vitamin D Treatment in Patients With Thyroid Eye Disease

Who this might be right for
Thyroid Eye Disease, TEDGraves Ophthalmopathy
Taipei Veterans General Hospital, Taiwan 120
Large-scale testing (Phase 3)Not Yet RecruitingNCT07290530
What this trial is testing

24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

Who this might be right for
Retinitis Pigmentosa (RP)Usher Syndrome
Nacuity Pharmaceuticals, Inc. 80
Not applicableStudy completedNCT06867471
What this trial is testing

Effects of Exogenous Ketosis on Proteinuria and Renal Function

Who this might be right for
Renal Insufficiency, ChronicPolycystic Kidney DiseasesProteinuria+1 more
Gødstrup Hospital 45
Testing effectiveness (Phase 2)Study completedNCT00229632
What this trial is testing

Idebenone to Treat Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
National Institute of Neurological Disorders and Stroke (NINDS) 51
Large-scale testing (Phase 3)Ended earlyNCT04485104
What this trial is testing

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Who this might be right for
Seizure in Participants With Tuberous Sclerosis ComplexSeizure in Participants With Dravet SyndromeSeizure in Participants With Lennox-Gastaut Syndrome
Jazz Pharmaceuticals 3
Testing effectiveness (Phase 2)Study completedNCT00470977
What this trial is testing

Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy

Who this might be right for
Coats' DiseaseIdiopathic Retinal TelangiectasiaRetinal Angiomatous Proliferation+7 more
Manhattan Eye, Ear & Throat Hospital 18
Not applicableStudy completedNCT03441685
What this trial is testing

Strategies for Teaching Verbs

Who this might be right for
Language Development DisordersDown SyndromeChild Language
Vanderbilt University Medical Center 44
Not applicableLooking for participantsNCT07435168
What this trial is testing

CHEST-CA: Study of Chest Pain and Hidden Cardiac Amyloidosis

Who this might be right for
Cardiac AmyloidosisChest Pain
Rigshospitalet, Denmark 300
Early research (Phase 1)Looking for participantsNCT06817590
What this trial is testing

Nucleoside Therapy in Patients With Telomere Biology Disorders

Who this might be right for
Telomere Biology DisordersDyskeratosis CongenitaRevesz Syndrome+4 more
Suneet Agarwal 36
Large-scale testing (Phase 3)Study completedNCT01380197
What this trial is testing

Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally

Who this might be right for
PainSickle Cell Disease
Children's Healthcare of Atlanta 40
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