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Results for “Congenital, Familial and Genetic Disorders”

977 trials

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Showing 20 of 977 results

Not applicableUnknownNCT05071378
What this trial is testing

Leveraging Family-Based Assets for Black MSM In House Ball Communities

Who this might be right for
HIV I Infection
State University of New York - Downstate Medical Center 168
Post-approval studies (Phase 4)Study completedNCT00916643
What this trial is testing

Low-Density Lipoprotein (LDL) Apheresis Using H.E.L.P. Therapy

Who this might be right for
Familial Hypercholesterolemia
B. Braun Medical Inc. 113
Very early researchStudy completedNCT03832985
What this trial is testing

Pediatric Reporting of Adult-Onset Genomic Results

Who this might be right for
Hereditary Breast and Ovarian Cancer SyndromeLynch SyndromeFamilial Hypercholesterolemia
Geisinger Clinic 162
Not applicableStudy completedNCT05191355
What this trial is testing

Screening for Hypercholesterolemia in Children Using Dried Blood Spot

Who this might be right for
Hypercholesterolemia
Hospices Civils de Lyon 60
Not applicableStudy completedNCT04819490
What this trial is testing

A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH

Who this might be right for
X-Linked Hypophosphataemia
Kyowa Kirin Pharmaceutical Development Ltd 32
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableUnknownNCT05701397
What this trial is testing

Prevalence of Cardiac Amyloidosis in Patients Undergoing Surgery for Lumbar Spinal Stenosis.

Who this might be right for
Cardiac AmyloidosisSpinal Stenosis
Steen Hvitfeldt Poulsen 200
Not applicableUnknownNCT03398954
What this trial is testing

A Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia

Who this might be right for
Familial Hypercholesterolemia
China Cardiovascular Association 1,128
Not applicableStudy completedNCT01070966
What this trial is testing

Vytorin Reexamination Study (0653A-174)

Who this might be right for
Primary Hypercholesterolemia and Homozygous Familial Hypercholesterolemia (HoFH)
Organon and Co 2,089
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Large-scale testing (Phase 3)Study completedNCT00991146
What this trial is testing

Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients With Cryopyrin-associated Periodic Syndromes Followed by an Extension Phase

Who this might be right for
Cryopyrin-associated Periodic SyndromesFamilial Cold Autoinflammatory SyndromeMuckle-Wells Syndrome+1 more
Novartis Pharmaceuticals 19
Early research (Phase 1)Study completedNCT05398029
What this trial is testing

VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

Who this might be right for
Heterozygous Familial HypercholesterolemiaAtherosclerotic Cardiovascular DiseaseHypercholesterolemia
Verve Therapeutics, Inc. 13
Not applicableStudy completedNCT05795400
What this trial is testing

Administration of the SGLT-2 Inhibitor Dapagliflozin in the Patients With Amyloid Cardiomyopathy

Who this might be right for
Heart Failure With Reduced Ejection FractionHeart Failure With Preserved Ejection FractionHeart Failure With Mid Range Ejection Fraction+1 more
National Medical Research Center for Cardiology, Ministry of Health of Russian Federation 50
Not applicableLooking for participantsNCT06866912
What this trial is testing

Observational Epidemiological Study of Patients Suffering From Systemic Amyloidosis

Who this might be right for
Cardiomyopathy
IRCCS Azienda Ospedaliero-Universitaria di Bologna 750
Early research (Phase 1)Study completedNCT00569283
What this trial is testing

Donor Natural Killer Cell Infusion in Preventing Relapse or Graft Failure in Patients Who Have Undergone Donor Bone Marrow Transplant

Who this might be right for
Cancer
Asan Medical Center 18
Not applicableLooking for participantsNCT07196839
What this trial is testing

Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
University of Texas Southwestern Medical Center 50
Not applicableStudy completedNCT04148001
What this trial is testing

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Who this might be right for
Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO Inc. 4
Not applicableStudy completedNCT04862273
What this trial is testing

CMR T1 Mapping for Diagnosis of Cardiac Amyloidosis

Who this might be right for
Heart Failure NYHA Class IIHeart Failure NYHA Class IIIHeart Failure NYHA Class IV+4 more
University of Leipzig 112
Testing effectiveness (Phase 2)Study completedNCT02595983
What this trial is testing

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

Who this might be right for
Transthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more
Alnylam Pharmaceuticals 12
Not applicableUnknownNCT05940922
What this trial is testing

RWE-based Treatment Patterns and Outcomes in CIDP

Who this might be right for
Chronic Inflammatory Demyelinating PolyneuropathyGuillain-Barre SyndromeHereditary Amyloidosis
RwHealth 170
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