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Results for “Congenital, Familial and Genetic Disorders”

1,002 trials

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Showing 20 of 1,002 results

Early research (Phase 1)Study completedNCT00685568
What this trial is testing

Celecoxib in Preventing Colorectal Cancer in Young Patients With a Genetic Predisposition for Familial Adenomatous Polyposis

Who this might be right for
Colorectal CancerPrecancerous Condition
M.D. Anderson Cancer Center 22
Not applicableLooking for participantsNCT06096688
What this trial is testing

Discovering New Targets for Colorectal and Endometrial Cancer Risk Reduction

Who this might be right for
Colorectal CancerEndometrial CancerHereditary Cancer Syndromes
Weill Medical College of Cornell University 1,120
Not applicableStudy completedNCT02131623
What this trial is testing

Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Who this might be right for
Alagille SyndromeProgressive Familial Intrahepatic Cholestasis
Mirum Pharmaceuticals, Inc. 23
Not applicableEnrolling By InvitationNCT06664866
What this trial is testing

AI Echocardiographic Screening of Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
Cedars-Sinai Medical Center 500
Not applicableUnknownNCT04080050
What this trial is testing

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Who this might be right for
Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO Inc. 8
Large-scale testing (Phase 3)Study completedNCT01302860
What this trial is testing

Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

Who this might be right for
Cryopyrin-associated Periodic SyndromesFamilial Cold Autoinflammatory SyndromeMuckle-Wells Syndrome+1 more
Novartis Pharmaceuticals 17
Early research (Phase 1)Study completedNCT05014360
What this trial is testing

JNJ-64251330 in Participants With Familial Adenomatous Polyposis

Who this might be right for
Adenomatous Polyposis Coli
Janssen Research & Development, LLC 42
Not applicableStudy completedNCT04661800
What this trial is testing

Study of Olfactory Disorders in Patients With Cardiac Amyloidosis

Who this might be right for
AmyloidosisCardiac AmyloidosisOlfactory Loss+4 more
Assistance Publique - Hôpitaux de Paris 51
Not applicableLooking for participantsNCT07689331
What this trial is testing

Hungarian National Systemic Amyloidosis Registry

Who this might be right for
Systemic AmyloidosisATTR AmyloidosisAL Amyloidosis+1 more
Semmelweis University 300
Not applicableWithdrawnNCT07607600
What this trial is testing

Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia

Who this might be right for
X-linked Hypophosphatemia (XLH)
Novo Nordisk A/S
Testing effectiveness (Phase 2)Ended earlyNCT05217667
What this trial is testing

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

Who this might be right for
Homozygous Familial Hypercholesterolemia
Arrowhead Pharmaceuticals 18
Not applicableStudy completedNCT03596554
What this trial is testing

X-linked Hypophosphatemia and FGF21

Who this might be right for
X-linked Hypophosphatemia
Hospices Civils de Lyon 39
Testing effectiveness (Phase 2)Study completedNCT03526250
What this trial is testing

Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)

Who this might be right for
Advanced Malignant Solid NeoplasmRecurrent Childhood EpendymomaRecurrent Ewing Sarcoma+29 more
National Cancer Institute (NCI) 23
Not applicableStudy completedNCT02206360
What this trial is testing

Pancreatic Cancer Early Detection Program

Who this might be right for
Pancreatic CancerPancreas CancerPancreatic Adenocarcinoma+8 more
White Plains Hospital 181
Early research (Phase 1)Study completedNCT00036959
What this trial is testing

ABT-751 in Treating Young Patients With Refractory Solid Tumors

Who this might be right for
Brain and Central Nervous System TumorsChildhood Germ Cell TumorExtragonadal Germ Cell Tumor+6 more
National Institutes of Health Clinical Center (CC) 90
Early research (Phase 1)Study completedNCT00770991
What this trial is testing

Lyophilized Black Raspberries in Adults With Familial Adenomatous Polyposis (FAP)

Who this might be right for
Familial Adenomatous Polyposis
The Cleveland Clinic 34
Large-scale testing (Phase 3)Study completedNCT02434497
What this trial is testing

Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia

Who this might be right for
Homozygous Familial Hypercholesterolemia (HoFH)
AstraZeneca 9
Not applicableNot Yet RecruitingNCT07606118
What this trial is testing

An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia

Who this might be right for
HypercholesterolaemiaFamilial HypercholesterolaemiaCVD Risk
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) 2,500
Not applicableLooking for participantsNCT06544018
What this trial is testing

Circadian Rhythm Deregulation in Patients With CAPS

Who this might be right for
Cryopyrin Associated Periodic SyndromeFamilial Cold UrticariaMuckle-Wells Syndrome+1 more
Hospices Civils de Lyon 30
Not applicableLooking for participantsNCT07232459
What this trial is testing

[18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis

Who this might be right for
AmyloidCardiac AmyloidosisAmyloidosis+1 more
Tianjin Medical University 25
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