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ClinicalTrials.gov public records Last synced Oct 18, 2026, 6:57 AM EDT

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Showing 289–312 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC

NCT06010329
View directory record Official record
Conditions
Non-small Cell Lung Cancer
Interventions
Sutetinib Maleate Capsule
Drug
Lead sponsor
Teligene US
Industry
Eligibility
18 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
9
States / cities
Glendale, California • La Jolla, California • Tampa, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Completed No phase listed Observational

Health-related Quality of Life in Rare Kidney Stone

NCT02124395
View directory record Official record
Conditions
Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
Interventions
Health-related Quality of Life in Rare Kidney Stone Questionnaire
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
365 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
2
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 18, 2026, 6:57 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Research for Individualized Therapeutics in Rare Genetic Disease

NCT05236595
View directory record Official record
Conditions
Rare Genetic Disease, Undiagnosed Diseases
Interventions
Individualized drug matching per genetic disease
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Minneota, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Completed Phase 2 Interventional Results available

Repetitive Transcranial Magnetic Stimulation for "Voices"

NCT00004980
View directory record Official record
Conditions
Schizophrenia
Interventions
repetitive transcranial magnetic stimulation (rTMS), sham stimulation
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2005
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 7, 2013 · Synced Oct 18, 2026, 6:57 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing AZD9291 as Potentially Targeted Treatment in Cancers With EGFR Genetic Changes (MATCH-Subprotocol E)

NCT06303167
View directory record Official record
Conditions
Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Multiple Myeloma
Interventions
Biopsy Procedure, Biospecimen Collection, Echocardiography Test, Multigated Acquisition Scan, Osimertinib, Radiologic Examination
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

NCT02867592
View directory record Official record
Conditions
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue, Clear Cell Sarcoma of Soft Tissue, Ewing Sarcoma, Hepatoblastoma, Hepatocellular Carcinoma, Osteosarcoma, Recurrent Adrenal Cortical Carcinoma, Recurrent Alveolar Soft Part Sarcoma, Recurrent Clear Cell Sarcoma of Soft Tissue, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Kidney Wilms Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Recurrent Renal Cell Carcinoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Medullary Carcinoma, Refractory Adrenal Cortical Carcinoma, Refractory Alveolar Soft Part Sarcoma, Refractory Clear Cell Sarcoma of Soft Tissue, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm, Refractory Renal Cell Carcinoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Medullary Carcinoma, Refractory Wilms Tumor, Renal Cell Carcinoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Solid Neoplasm, Thyroid Gland Medullary Carcinoma, Wilms Tumor
Interventions
Cabozantinib, Cabozantinib S-malate, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 30 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
142
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 104 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Clinical Study to Test if an Investigational Treatment Called BNT326 is Safe and Potentially Beneficial When Used Alone or in Combination With Other Investigational Treatments Such as BNT327, for People With Advanced Malignant Tumors

NCT07070232
View directory record Official record
Conditions
Advanced Solid Tumor
Interventions
BNT326, Pumitamig, Itraconazole, Paroxetine
Drug
Lead sponsor
BioNTech SE
Industry
Eligibility
18 Years and older
Enrollment
1,438 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
19
States / cities
Phoenix, Arizona • San Francisco, California • Hartford, Connecticut + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting No phase listed Observational

Delayed Toxicities Post-CAR-T

NCT07665307
View directory record Official record
Conditions
Multiple Myeloma
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
San Francisco, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Not listed No phase listed Observational

Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries

NCT00006128
View directory record Official record
Conditions
Head Injuries, Closed
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 12 Years
Enrollment
374 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
4
States / cities
La Jolla, California • Dallas, Texas • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 6:57 AM EDT
Completed Phase 1 Interventional

Indocyanine Green (ICG) Guided Tumor Resection

NCT04084067
View directory record Official record
Conditions
Neoplastic Disease, Solid Tumor
Interventions
Indocyanine Green
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
215 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting Phase 4 Interventional

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

NCT07531251
View directory record Official record
Conditions
Barth Syndrome
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
5 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Needham, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Completed Not applicable Interventional

Palliative Care Coaching for Families With Rare Advanced Lung Diseases

NCT03813667
View directory record Official record
Conditions
Lung Diseases, Rare Non-Neoplastic Disorder
Interventions
Intervention FamPALcare
Behavioral
Lead sponsor
West Virginia University
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Morgantown, West Virginia
Source: ClinicalTrials.gov public record
Updated Oct 21, 2019 · Synced Oct 18, 2026, 6:57 AM EDT
Completed No phase listed Observational

The Experience of Uncertainty in Parents of Children With an Undiagnosed Medical Condition

NCT00955370
View directory record Official record
Conditions
Multiple Abnormalities
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Vascular Disease Discovery Protocol

NCT03538639
View directory record Official record
Conditions
Vascular Dysfunction, Genetic Mutations, Genetic Predisposition
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Not listed No phase listed Observational

Studies in Porphyria I: Characterization of Enzyme Defects

NCT00004331
View directory record Official record
Conditions
Porphyria
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1992
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 6:57 AM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting Not applicable Interventional

IntelliWell: An AI-Assisted Imaging Platform for Detection and Location of Ultra-Rare Testicular Sperm in Surgical Specimens

NCT07074015
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 Jun 22, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Recruiting Phase 2 Interventional

A Study of Zipalertinib in Patients With Advanced Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions or Other Uncommon Mutation.

NCT05967689
View directory record Official record
Conditions
Advanced or Metastatic NSCLC Harboring Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertion (ex20ins) Mutations
Interventions
TAS6417, CYP Cocktail, Transporter Cocktail
Drug
Lead sponsor
Taiho Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
18
States / cities
Birmingham, Alabama • Duarte, California • Boston, Massachusetts + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Enrolling by invitation No phase listed Observational

SLC13A5 Deficiency Natural History Study - Remote Only

NCT04681781
View directory record Official record
Conditions
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
Interventions
Not listed
Lead sponsor
TESS Research Foundation
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2024 · Synced Oct 18, 2026, 6:57 AM EDT
Completed Not applicable Interventional

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

NCT00004495
View directory record Official record
Conditions
End Stage Renal Disease, Hyperhomocysteinemia
Interventions
cyanocobalamin, folic acid, pyridoxine
Drug
Lead sponsor
Georgetown University
Other
Eligibility
21 Years to 89 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2000
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 18, 2026, 6:57 AM EDT
Terminated Phase 2 Interventional Results available

A Study of Soticlestat in Adults and Children With Rare Epilepsies

NCT03635073
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
Interventions
Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years and older
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
27
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 18, 2026, 6:57 AM EDT
Completed No phase listed Observational

Genetic Study of Patients With Primary Ciliary Dyskinesia

NCT00005650
View directory record Official record
Conditions
Primary Ciliary Dyskinesia
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 6:57 AM EDT
Completed No phase listed Observational

Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases

NCT00001686
View directory record Official record
Conditions
Ewing's Sarcoma, Leukemia, Lymphoma, Brain Tumors, Rhabdomyosarcoma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 40 Years
Enrollment
482 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2020 · Synced Oct 18, 2026, 6:57 AM EDT