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ClinicalTrials.gov public records Last synced Oct 18, 2026, 8:59 AM EDT

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Showing 361–384 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Active, not recruiting Phase 2 Interventional

A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma

NCT01878617
View directory record Official record
Conditions
Medulloblastoma
Interventions
Craniospinal Irradiation with boost to the primary tumor site, Cyclophosphamide, Cisplatin, Vincristine, Vismodegib, Pemetrexed, Gemcitabine, Aerobic Training, Neurocognitive Remediation
Radiation · Drug · Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
3 Years to 39 Years
Enrollment
660 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2031
U.S. locations
14
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Oct 18, 2026, 8:59 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

NCT06314490
View directory record Official record
Conditions
Genetic Disease
Interventions
nL-SCN2A-002
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Firmonertinib Compared With Investigator's Choice of EGFR Inhibitor as First-Line Treatment in Participants Who Have Locally Advanced or Metastatic NSCLC With EGFR P-Loop and Alpha C-Helix Compressing (PACC) Uncommon Mutations

NCT07185997
View directory record Official record
Conditions
Non-Small-Cell Lung Cancer, Metastatic Non-Small-Cell Lung Cancer, Advanced Non-Small-Cell Lung Cancer, EGFR P-Loop and Alpha C-Helix Compressing, EGFR PACC, EGFR Uncommon Mutations
Interventions
Firmonertinib, EGFR-TKI inhibitor based on investigator's choice
Drug
Lead sponsor
ArriVent BioPharma, Inc.
Industry
Eligibility
18 Years and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
21
States / cities
Fullerton, California • Los Angeles, California • Orange, California + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Not applicable Interventional

Droxidopa in Treating Patients With Neurogenic Hypotension

NCT00004478
View directory record Official record
Conditions
Shy-Drager Syndrome, Orthostatic Hypotension
Interventions
droxidopa
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
20 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 18, 2008 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting No phase listed Observational

Genome Medical Sequencing for Gene Discovery

NCT01087320
View directory record Official record
Conditions
Intellectual Disabilities, Congenital Anomaly, Rare Disorders
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Weeks to 99 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Active, not recruiting Phase 1 Interventional

Study of Furmonertinib in Patients With Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) With Activating, Including Uncommon, Epidermal Growth Factor Receptor (EGFR) or Human Epidermal Growth Factor Receptor 2 (HER2) Mutations

NCT05364073
View directory record Official record
Conditions
Non-Small Cell Lung Cancer (NSCLC), Metastatic Non-Small Cell Lung Cancer, Advanced Non-Small Cell Lung Cancer, HER2 Exon 20 Mutations, EGFR Exon 20 Mutations, EGFR Uncommon Mutations, Including G719X and S768I
Interventions
Furmonertinib
Drug
Lead sponsor
ArriVent BioPharma, Inc.
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Prescott, Arizona • Sacramento, California • Whittier, California + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Completed No phase listed Observational

Genetic Study of Familial Epilepsy

NCT00006059
View directory record Official record
Conditions
Epilepsy
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
898 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 8:59 AM EDT
Active, not recruiting Phase 2 Interventional

Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors

NCT03074513
View directory record Official record
Conditions
Appendix Adenocarcinoma, Human Papillomavirus-Related Anal Squamous Cell Carcinoma, Human Papillomavirus-Related Cervical Squamous Cell Carcinoma, Human Papillomavirus-Related Squamous Cell Carcinoma of the Penis, Human Papillomavirus-Related Vulvar Squamous Cell Carcinoma, Neuroendocrine Carcinoma, Pancreatic Neuroendocrine Tumor, Recurrent Merkel Cell Carcinoma, Recurrent Nasopharynx Carcinoma, Recurrent Peritoneal Malignant Mesothelioma, Recurrent Pleural Malignant Mesothelioma, Stage III Merkel Cell Carcinoma AJCC v7, Stage III Nasopharyngeal Carcinoma AJCC v7, Stage III Pleural Malignant Mesothelioma AJCC v7, Stage IV Merkel Cell Carcinoma AJCC v7, Stage IV Nasopharyngeal Carcinoma AJCC v7, Stage IV Pleural Malignant Mesothelioma AJCC v7, Stage IVA Nasopharyngeal Carcinoma AJCC v7, Stage IVB Nasopharyngeal Carcinoma AJCC v7, Stage IVC Nasopharyngeal Carcinoma AJCC v7, Vaginal Squamous Cell Carcinoma, Not Otherwise Specified
Interventions
Atezolizumab, Bevacizumab, Laboratory Biomarker Analysis, Pharmacological Study
Drug · Biological · Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Not listed No phase listed Observational

Study of Depression, Peptides, and Steroids in Cushing's Syndrome

NCT00004334
View directory record Official record
Conditions
Cushing's Syndrome
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
20 Years to 60 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1994
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Phase 2 Interventional

Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

NCT00004464
View directory record Official record
Conditions
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 70 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 18, 2019 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

NCT05361811
View directory record Official record
Conditions
Neurofibromatosis 1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome
Interventions
Waitlist, ACT Intervention
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

NCT03680365
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
Interventions
Not listed
Lead sponsor
Jett Foundation, Inc.
Other
Eligibility
11 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 18, 2026, 8:59 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2048
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Completed No phase listed Observational

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

NCT01784679
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM), Distal Myopathy With Rimmed Vacuoles (DMRV)
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
319 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Apr 27, 2018 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting Phase 2 Interventional

Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors

NCT01356290
View directory record Official record
Conditions
Medulloblastoma Recurrent, Ependymoma Recurrent, ATRT Recurrent, Rare CNS Tumor Recurrent
Interventions
Bevacizumab, Thalidomide, Celecoxib, Fenofibric acid, Etoposide, Cyclophosphamide, Cytarabine, Temozolomide (TMZ), Irinotecan
Drug
Lead sponsor
Medical University of Vienna
Other
Eligibility
Up to 19 Years
Enrollment
232 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • Boston, Massachusetts • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Not listed Phase 2 Interventional Accepts healthy volunteers

Ultraviolet Light Therapy for Systemic Lupus Erythematosus

NCT00004375
View directory record Official record
Conditions
Systemic Lupus Erythematosus
Interventions
ultraviolet A-1 light treatment
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
15 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1991
U.S. locations
2
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Phase 2 Interventional

Phase II Study of High-Dose Cyclophosphamide in Patients With Severe Autoimmune Hematologic Disease

NCT00010387
View directory record Official record
Conditions
Anemia, Hemolytic, Autoimmune, Felty Syndrome, Purpura, Thrombocytopenic, Autoimmune Diseases
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2008 · Synced Oct 18, 2026, 8:59 AM EDT
Enrolling by invitation No phase listed Observational

Idiopathic Diseases of Man

NCT01440218
View directory record Official record
Conditions
Rare Disease, Idiopathic Disease
Interventions
Not listed
Lead sponsor
Scripps Translational Science Institute
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2030
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jan 16, 2025 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Not applicable Interventional

Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease

NCT00007033
View directory record Official record
Conditions
Alagille Syndrome, Cholestasis, Biliary Atresia
Interventions
magnesium gluconate, magnesium sulfate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
3 Years to 18 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 22, 2009 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Phase 1 Interventional

Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders

NCT00010335
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 6, 2015 · Synced Oct 18, 2026, 8:59 AM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 18, 2026, 8:59 AM EDT
Completed Not applicable Interventional

Randomized Study of Fluoxetine in Children and Adolescents With Autism

NCT00004486
View directory record Official record
Conditions
Autism
Interventions
fluoxetine
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
5 Years to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
3
States / cities
New York, New York • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 18, 2026, 8:59 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Energy Expenditure in Infants With Ventricular Septal Defects

NCT00006272
View directory record Official record
Conditions
Heart Septal Defects, Ventricular
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
3 Months to 4 Months
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1994
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 8:59 AM EDT