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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

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

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

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

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: Rochester NY, Aurora CO, Portland OR
Sponsor: University of Colorado, Denver

Sex: All

Age: 18 - 70+

Code: NCT07292298

Phase2, Recruiting