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Rapid Analysis and Response Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE CANCER) Trial: RARE 1 Nilotinib and Paclitaxel

for
Neoplasms
Location: Bethesda MD
Sponsor: National Cancer Institute (NCI)

Sex: All

Age: 18 - 70+

Code: NCT04449549

Phase2, Recruiting

Testing the Effectiveness of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) With One Anti-cancer Targeted Drug (Cabozantinib) for Rare Genitourinary Tumors

for
Bladder Adenocarcinoma, Bladder Clear Cell Adenocarcinoma,
Location: Peabody MA, Burlington MA, Commack NY, Uniondale NY,
Sponsor: National Cancer Institute (NCI)

Sex: All

Age: 18+

Code: NCT03866382

Phase2, Recruiting

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

for
Sanfilippo Syndrome, MPS3
Location: Fairfax VA
Sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Sex: All

Age: 5 - 70+

Code: NCT05705674

Recruiting

Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy

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

Sex: All

Age: 18 - 70

Code: NCT04724083

Recruiting

UW Undiagnosed Genetic Diseases Program

for
Rare Diseases, Genetic Disease, Undiagnosed Disease
Location: Madison WI
Sponsor: University of Wisconsin, Madison

Sex: All

Age: 0 - 70+

Code: NCT04586075

Recruiting

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Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers

for
Adrenal Hyperplasia, Congenital
Location: New York NY
Sponsor: Office of Rare Diseases (ORD)

Sex: All

Age: 12+

Code: NCT00617292

Recruiting

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

for
Adrenal Hyperplasia, Congenital
Location: New York NY
Sponsor: Office of Rare Diseases (ORD)

Sex: Female

Age: 20 - 35

Code: NCT00694525

Recruiting

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

for
Anemia, Aplastic
Location: Hershey PA, Tampa FL, Cleveland OH, Los Angeles CA
Sponsor: Office of Rare Diseases (ORD)

Sex: All

Age: 21+

Code: NCT00319878

Phase1, Phase2, Recruiting

Getting Global Rare Disease Insights Through Technology Study

for
Metabolic Disease, Fabry Disease, Gaucher Disease,
Location: Calgary
Sponsor: M.A.G.I.C. Clinic LTD

Sex: All

Age: 18+

Code: NCT04758130

Recruiting

Dabrafenib and/or Trametinib Rollover Study

for
Melanoma, Non Small Cell Lung Cancer, Solid Tumor, Rare Cancers,
Location: Toronto
Sponsor: Novartis Pharmaceuticals

Sex: All

Age: 18 - 70+

Code: NCT03340506

Recruiting