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ClinicalTrials.gov public records Last synced Oct 17, 2026, 3:02 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 49–72 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Completed No phase listed Observational

Genetic Studies in the Amish and Mennonites

NCT00359580
View directory record Official record
Conditions
Genetic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2014
U.S. locations
2
States / cities
Bethesda, Maryland • Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed No phase listed Observational

Study of the Pathogenesis of Porphyria Cutanea Tarda

NCT00005103
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Nutritious Eating With Soul at Rare Variety Cafe

NCT04513769
View directory record Official record
Conditions
Obesity
Interventions
Vegan diet
Behavioral
Lead sponsor
University of South Carolina
Other
Eligibility
18 Years to 65 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated May 17, 2022 · Synced Oct 17, 2026, 3:02 PM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting Phase 3 Interventional

A Study to Assess Zipalertinib Versus Placebo in Participants With Early Stage NSCLC With Uncommon EGFR Mutations, Following Complete Tumor Resection

NCT07128199
View directory record Official record
Conditions
Lung Cancer, Adjuvant, Post-surgical, EGFR, Exon 20, Early Stage Lung Cancer, Uncommon EGFR Mutations, NSCLC, Early Stage
Interventions
TAS6417, Zipalertinib Matching-placebo
Drug
Lead sponsor
Taiho Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
30
States / cities
Anchorage, Alaska • Duarte, California • Irvine, California + 26 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed No phase listed Observational

Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors

NCT07374692
View directory record Official record
Conditions
Recurrent CNS Tumors, Brain Cancer, Gliomas, IDH-wildtype Gliomas, IDH-mutant Gliomas, Rare CNS Tumor
Interventions
SmartMatch testing
Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
15 Years to 120 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 17, 2026, 3:02 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

NCT06721871
View directory record Official record
Conditions
Microvillus Inclusion Disease, Congenital Disorders, Rare Diseases
Interventions
Crofelemer Powder for Oral Solution, Placebo Powder for Oral Solution
Drug
Lead sponsor
Napo Pharmaceuticals, Inc.
Industry
Eligibility
Up to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed No phase listed Observational

Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders

NCT03967743
View directory record Official record
Conditions
Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease, Development, Infant, Development, Child
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 4 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed No phase listed Observational

Collecting and Storing Tissue Samples From Patients With Rare or Cutaneous Non-Hodgkin Lymphoma

NCT01000753
View directory record Official record
Conditions
Adult Immunoblastic Lymphoma, Central Nervous System Non-Hodgkin Lymphoma, Childhood Immunoblastic Lymphoma, Grade 1 Follicular Lymphoma, Grade 2 Follicular Lymphoma, Grade 3 Follicular Lymphoma, Lymphoproliferative Disorder, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Non-Hodgkin Lymphoma, Primary Cutaneous B-Cell Non-Hodgkin Lymphoma, Primary Cutaneous T-Cell Non-Hodgkin Lymphoma, Small Lymphocytic Lymphoma
Interventions
Cytology Specimen Collection Procedure
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2023
U.S. locations
119
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 93 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting No phase listed Observational

The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)

NCT01694953
View directory record Official record
Conditions
Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 17, 2025 · Synced Oct 17, 2026, 3:02 PM EDT
Terminated Phase 2 Interventional Results available

Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds

NCT04240886
View directory record Official record
Conditions
Invasive Fungal Infections
Interventions
fosmanogepix
Drug
Lead sponsor
Basilea Pharmaceutica
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
6
States / cities
Duarte, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 16, 2025 · Synced Oct 17, 2026, 3:02 PM EDT
Active, not recruiting No phase listed Observational

Attention and Interpretation Modification (AIM) for Fear of Cancer Recurrence: An Intervention Development Study

NCT01517945
View directory record Official record
Conditions
Breast Cancer, Rare Cancer
Interventions
AIM materials and assessments
Behavioral
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 24, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Not listed Not applicable Interventional

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

NCT00006056
View directory record Official record
Conditions
Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Phase 2 Interventional

Phase II Double-Blind, Placebo-Controlled Study of the Reinforcing Effects of Alprazolam in Patients With Anxiety

NCT00004373
View directory record Official record
Conditions
Anxiety Disorder, Panic Disorder
Interventions
alprazolam
Drug
Lead sponsor
National Institute on Drug Abuse (NIDA)
NIH
Eligibility
18 Years to 50 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 3:02 PM EDT
Not listed No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Diagnosis and Treatment of Patients With Inborn Errors of Metabolism

NCT00369421
View directory record Official record
Conditions
Arterial Calcification Due to Deficiency of CD73
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1978
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting No phase listed Observational

National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

NCT06491615
View directory record Official record
Conditions
Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2054
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting No phase listed Observational

Nephrotic Syndrome Study Network

NCT01209000
View directory record Official record
Conditions
Minimal Change Disease (MCD), Membranous Nephropathy, Glomerulosclerosis, Focal Segmental
Interventions
Kidney Biopsy
Procedure
Lead sponsor
University of Michigan
Other
Eligibility
Up to 80 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2030
U.S. locations
39
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 26 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Recruiting No phase listed Observational

Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors

NCT05868629
View directory record Official record
Conditions
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Interventions
Non-investigational
Other
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
8
States / cities
Torrance, California • Denver, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Not applicable Interventional

Randomized Study of Tauroursodeoxycholic Acid in Prophylactic Therapy of Total Parenteral Nutrition Associated Cholestasis in Infants

NCT00004410
View directory record Official record
Conditions
Cholestasis
Interventions
tauroursodeoxycholic acid
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Up to 20 Days
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
3
States / cities
Minneapolis, Minnesota • Jackson, Mississippi • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Early Phase 1 Interventional Results available

Calcitriol Monotherapy for X-Linked Hypophosphatemia

NCT03748966
View directory record Official record
Conditions
X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant
Interventions
Calcitriol
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
3 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 17, 2026, 3:02 PM EDT
Completed Phase 2 Interventional

Oxandrolone Compared With a Placebo on Growth Rate in Girls With Growth Hormone-Treated Turner's Syndrome

NCT00004275
View directory record Official record
Conditions
Turner's Syndrome
Interventions
growth hormone, oxandrolone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
10 Years to 14 Years · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 10, 2008 · Synced Oct 17, 2026, 3:02 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866
View directory record Official record
Conditions
Fabry Disease, Fabry Disease, Cardiac Variant
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
8
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 17, 2026, 3:02 PM EDT