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Active & Responsive

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

for
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis,
Location: Los Altos CA
Sponsor: xCures

Sex: All

Age: 0+

Code: NCT06539169

Recruiting
Active & Responsive

Longitudinal Studies of Patient With FPDMM

for
Inherited Hematological Diseases, Rare Diseases, FPDMM
Location: Bethesda MD
Sponsor: National Human Genome Research Institute (NHGRI)

Sex: All

Age: 0 - 70+

Code: NCT03854318

Recruiting
Active & Responsive

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

for
Inherited Retinal Degeneration, Retinitis Pigmentosa
Location: Boston MA, Philadelphia PA, Baltimore MD, Durham NC,
Sponsor: Jaeb Center for Health Research

Sex: All

Age: 4+

Code: NCT05589714

Recruiting
Active & Responsive

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

for
Rare Disorders, Undiagnosed Disorders,
Location: Sioux Falls SD
Sponsor: Sanford Health

Sex: All

Age: 0+

Code: NCT01793168

Recruiting
Active & Responsive

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

for
Lysosomal Storage Diseases
Location: Fairfax VA
Sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Sex: All

Age: 0+

Code: NCT03812055

Recruiting

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Active & Responsive

TCF-001 TRACK (Target Rare Cancer Knowledge) Study

for
Rare Cancers, Cholangiocarcinoma,
Location: Cambridge MA
Sponsor: TargetCancer Foundation

Sex: All

Age: 18+

Code: NCT04504604

Recruiting
Active & Responsive

Investigating Phenotypic, Epigenetic, and NeuroGenetic Traits in Rare and Ultra-rare Neurodevelopmental Disorders (Project PENGUIN)

for
Baker Gordon Syndrome, Rare Neurodevelopmental Conditions,
Location: Columbia MO
Sponsor: University of Missouri-Columbia

Sex: All

Age: 0 - 70+

Code: NCT07329257

Recruiting
Active & Responsive

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

for
Gaucher Disease Type 1
Location: Fairfax VA
Sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Sex: All

Age: 16 - 70+

Code: NCT04055831

Recruiting
Active & Responsive

Gene Discovery Core, The Manton Center

for
Undiagnosed Conditions, Rare Disorders, Orphan Diseases
Location: Boston MA
Sponsor: Boston Children's Hospital

Sex: All

Age: 0+

Code: NCT02743845

Recruiting
Active & Responsive

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

for
Spinal Muscular Atrophy Type 3,
Location: Boston MA, New York NY, Palo Alto CA
Sponsor: Columbia University

Sex: All

Age: 5+

Code: NCT06839469

Recruiting
Active & Responsive

Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates

for
Rectal Adenocarcinoma, Rectal Cancer
Location: Aurora CO
Sponsor: University of Colorado, Denver

Sex: All

Age: 18 - 70+

Code: NCT07292298

Phase2, Recruiting

Baker Gordon Syndrome Natural History Study

for
Rare Diseases, Autism or Autistic Traits, Development Delay,
Location: Columbia MO
Sponsor: University of Missouri-Columbia

Sex: All

Age: 0 - 70+

Code: NCT06399952

Recruiting

Umbrella Study for Single Patient Treatments in Oncology

for
Advanced Cancer, Rare Diseases
Location: Toronto
Sponsor: University Health Network, Toronto

Sex: All

Age: 18+

Code: NCT06285500

Recruiting

A Multi-Site Leukopak Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives

for
Rare Diseases
Location: Waltham MA
Sponsor: Sanguine Biosciences

Sex: All

Age: 18 - 70+

Code: NCT05794217

Recruiting

Epilepsy Learning Healthcare System (ELHS)

for
Epilepsy, Seizure Disorder, Neurologic Disorder, Rare Diseases
Location: Boston MA, Philadelphia PA, Bowie MD, Hershey PA,
Sponsor: Epilepsy Foundation of America

Sex: All

Age: 0+

Code: NCT06265103

Recruiting

Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas

for
Rare Diseases
Location: Houston TX
Sponsor: Baylor College of Medicine

Sex: All

Age: 0

Code: NCT07102966

Recruiting

Stakeholders of Rare Diseases Informing Values In Neuroethics

for
Rare Disorder, Disorder, Neurologic
Location: Memphis TN
Sponsor: St. Jude Children's Research Hospital

Sex: All

Age: 0+

Code: NCT07314736

Recruiting

Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

for
Myelodysplastic Syndrome
Location: Hershey PA, Tampa FL, Cleveland OH
Sponsor: Office of Rare Diseases (ORD)

Sex: All

Age: 18+

Code: NCT00466843

Phase2, Recruiting

Angioedema Biomarker Research Study

for
Angioedema, Angioedemas, Hereditary, Urticaria, Mastocytosis,
Location: Wheaton MD
Sponsor: Foundation For Rare Disease Research

Sex: All

Age: 12 - 70+

Code: NCT06210698

Recruiting

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

for
Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
Location: New York NY, Washington DC, Tampa FL, Indianapolis IN,
Sponsor: Recordati Rare Diseases

Sex: All

Age: 0+

Code: NCT05040178

Recruiting