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Trial Match

Clinical trials test new treatments โ€” sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

๐ŸŽฏ Match trials to me

11 recruiting trials

for MPS I

Cardiac Structure and Function in MPS NCT07361536n/a3 sites๐Ÿ“ Orange๐Ÿ“ Minneapolis๐Ÿ“ St Louis Children's Hospital of Orange County Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH NCT02716246Phase 2, Phase 35 sites๐Ÿ“ Columbus๐Ÿ“ Pittsburgh๐Ÿ“ North Adelaide Ultragenyx Pharmaceutical Inc ยท Tests: UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I NCT05682144Phase 12 sites๐Ÿ“ Oakland๐Ÿ“ Minneapolis Immusoft of CA, Inc. ยท Tests: Autologous Plasmablasts (B cells) Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC NCT04528355n/a1 site๐Ÿ“ Pittsburgh Paul Szabolcs ยท Tests: data collection PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) NCT04532047Phase 11 site๐Ÿ“ San Francisco University of California, San Francisco ยท Tests: Aldurazyme (laronidase) A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I NCT06519552Phase 11 site๐Ÿ“ Chengdu West China Hospital ยท Tests: JWK008 Single intravenous infusion administration MPS (RaDiCo Cohort) (RaDiCo-MPS) NCT06036693n/a23 sites๐Ÿ“ Angers๐Ÿ“ Bordeaux๐Ÿ“ Brest Institut National de la Santรฉ Et de la Recherche Mรฉdicale, โ€ฆ A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB) NCT07640984Phase 1, Phase 25 sites๐Ÿ“ Oakland๐Ÿ“ Pittsburgh๐Ÿ“ Fairfax JCR Pharmaceuticals Co., Ltd. ยท Tests: JR-446 Registry of Patients Diagnosed With Lysosomal Storage Diseases NCT05619900n/a1 site๐Ÿ“ San Francisco University of California, San Francisco ยท Tests: There is no intervention Longitudinal Study of Neurodegenerative Disorders NCT03333200n/a1 site๐Ÿ“ Pittsburgh University of Pittsburgh ยท Tests: Palliative Care, Hematopoetic Stem Cell Transplantation Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415Phase 21 site๐Ÿ“ Pittsburgh Paul Szabolcs ยท Tests: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
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