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ClinicalTrials.gov public records Last synced May 21, 2026, 5:33 PM EDT

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

Showing 1–24 of 404 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Rare Diseases Clear all
Terminated Phase 2 Interventional Results available
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 15, 2025 · Synced May 21, 2026, 5:33 PM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational
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 13, 2024 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Aspergillosis, Invasive Fungal Infections
Interventions
isavuconazole
Drug
Lead sponsor
Astellas Pharma Inc
Industry
Eligibility
18 Years and older
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
24
States / cities
Birmingham, Alabama • Duarte, California • Sacramento, California + 19 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases
Interventions
Stem Cell Transplantation, CD34 selection
Procedure · Device
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
2 Years to 18 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced May 21, 2026, 5:33 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Infertility, Azoospermia
Interventions
Use of IntelliWell to identify and localize sperm for ICSI
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 21, 2026, 5:33 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Choroideremia
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
10 Years and older
Enrollment
1,178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 7, 2021 · Synced May 21, 2026, 5:33 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Part 1: Bladder, Biliary Tract, Cervical, Endometrial, Ovarian, Pancreatic Cancer, Rare Tumors, Any Tumor Type Excluding Breast, Gastric, Colorectal Cancer, Part 2: HER2 Expressing/Amplified Solid Tumors Excluding Breast, Gastric, Colorectal Cancer
Interventions
Trastuzumab deruxtecan
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 120 Years
Enrollment
477 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
12
States / cities
Duarte, California • Santa Rosa, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced May 21, 2026, 5:33 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Trigeminal Neuralgia
Interventions
L-baclofen
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced May 21, 2026, 5:33 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Cancer
Interventions
Sirolimus, Celecoxib, Etoposide, Cyclophosphamide
Drug
Lead sponsor
Emory University
Other
Eligibility
12 Months to 30 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
6
States / cities
Phoenix, Arizona • Wilmington, Delaware • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced May 21, 2026, 5:33 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Congenital Portosystemic Shunt, CPSS (Congenital Portosystemic Shunt)
Interventions
Shunt Closure
Procedure
Lead sponsor
Prof. Valérie Mc Lin
Other
Eligibility
1 Day and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
2
States / cities
New Haven, Connecticut • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 17, 2025 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Adrenocortical Carcinoma, Peritoneal Carcinomatosis
Interventions
Cisplatin, Cytoreductive surgery, sodium thiosulfate
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 29, 2019 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 4 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
zinc acetate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1993
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
cyclophosphamide, filgrastim, methylprednisolone, Autologous Stem Cell Transplantation
Drug · Procedure
Lead sponsor
Northwestern Memorial Hospital
Other
Eligibility
0 Years to 59 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
2
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 5:33 PM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Lymphoma, Adult T-Cell Leukemia/Lymphoma, Lymphatic Diseases
Interventions
Brentuximab Vedotin, CHEP
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
4
States / cities
Hollywood, Florida • Boston, Massachusetts • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 9, 2025 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Rare Disorder, Disorder, Neurologic
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
385 participants
Timeline
2026 – 2031
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 21, 2026, 5:33 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Systemic Lupus Erythematosus, Antiphospholipid Antibody Syndrome
Interventions
Cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 70 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 5:33 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic, Ewing's Tumor Recurrent, Rare Diseases, Sarcoma, Neoplasms, Connective and Soft Tissue, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective Tissue, Sarcoma, Ewing, Neoplasms
Interventions
Vigil, Irinotecan, Temozolomide
Biological · Drug
Lead sponsor
Gradalis, Inc.
Industry
Eligibility
2 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
15
States / cities
Little Rock, Arkansas • Los Angeles, California • Jacksonville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 25, 2023 · Synced May 21, 2026, 5:33 PM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Carcinoma of Unknown Primary, Metastatic Adrenal Gland Pheochromocytoma, Metastatic Kidney Medullary Carcinoma, Metastatic Malignant Germ Cell Tumor, Metastatic Malignant Solid Neoplasm, Metastatic Paraganglioma, Metastatic Penile Carcinoma, Metastatic Skin Squamous Cell Carcinoma, Small Cell Carcinoma, Stage III Adrenal Cortex Carcinoma AJCC v7, Stage IV Adrenal Cortex Carcinoma AJCC v7, Stage IV Penile Cancer AJCC v7, Stage IV Renal Cell Cancer AJCC v7, Unresectable Adrenal Gland Pheochromocytoma, Unresectable Paraganglioma, Unresectable Skin Squamous Cell Carcinoma, Unresectable Solid Neoplasm, Vascular Neoplasm
Interventions
Laboratory Biomarker Analysis, Pembrolizumab, Questionnaire Administration
Other · Biological
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
PI3KCD, CTLA4, STAT3GOF, MAGT1
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational
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 May 3, 2026 · Synced May 21, 2026, 5:33 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
clotrimazole, hydroxyurea
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2002
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced May 21, 2026, 5:33 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Hematologic Cancer
Interventions
Molecularly targeted treatment matched to genomic/immunophenotypic tumor profile (chosen by treating physician)
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Feb 14, 2022 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 30, 2020 · Synced May 21, 2026, 5:33 PM EDT