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Active & Responsive

Longitudinal Studies of Patient With FPDMM

for
Inherited Hematological Diseases, Rare Diseases, FPDMM
Location: 1 recruiting location
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: 20 recruiting locations
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: 1 recruiting location
Sponsor: Sanford Health

Sex: All

Age: 0+

Code: NCT01793168

Recruiting
Active & Responsive

TCF-001 TRACK (Target Rare Cancer Knowledge) Study

for
Rare Cancers, Cholangiocarcinoma,
Location: 1 recruiting location
Sponsor: TargetCancer Foundation

Sex: All

Age: 18+

Code: NCT04504604

Recruiting
Active & Responsive

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

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

Sex: All

Age: 16 - 70+

Code: NCT04055831

Recruiting

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Active & Responsive

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

for
Hematologic Disorder, Bleeding Disorder,
Location: 62 recruiting locations
Sponsor: American Thrombosis and Hemostasis Network

Sex: All

Age: 0+

Code: NCT04398628

Recruiting
Active & Responsive

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

for
Lysosomal Storage Diseases
Location: 1 recruiting location
Sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Sex: All

Age: 0+

Code: NCT03812055

Recruiting
Active & Responsive

Gene Discovery Core, The Manton Center

for
Undiagnosed Conditions, Rare Disorders, Orphan Diseases
Location: 1 recruiting location
Sponsor: Boston Children's Hospital

Sex: All

Age: 0+

Code: NCT02743845

Recruiting
Active & Responsive

Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms

for
Central Nervous System Neoplasms, Glioblastoma,
Location: 1 recruiting location
Sponsor: Jazz Pharmaceuticals

Sex: All

Age: 18+

Code: NCT04541082

Phase1, Recruiting
Active & Responsive

Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

for
Malignant Solid Tumors, Other Neoplasms Solid Tumors,
Location: 3 recruiting locations
Sponsor: National Cancer Institute (NCI)

Sex: All

Age: 0+

Code: NCT03739827

Recruiting