An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Key facts

Sponsor

JCR Pharmaceuticals Co., Ltd.

Intervention

JR-446(drug)

Enrollment

10 enrolled

Eligibility

17 years · All sexes

Timeline

2024 – 2030

Study locations (3)

• Hiroshima University Hospital, Hiroshima, Japan
• University of the Ryukyus Hospital, Okinawa, Japan
• National Center for Child Health and Development, Tokyo, Japan

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Mucopolysaccharidosis type 1

Additional recruiting or active studies for the same condition.

• RECRUITINGPHASE1NCT06519552
  A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
  West China Hospital
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06181136
  Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
  Denali Therapeutics Inc.
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06095388
  Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
  JCR Pharmaceuticals Co., Ltd.
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT05665166
  Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
  University of Manchester
• ACTIVE NOT RECRUITINGNCT05634512
  Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
  Masonic Cancer Center, University of Minnesota
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT04201405
  Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
  University of Manchester
• ACTIVE NOT RECRUITINGPHASE2, PHASE3NCT03566043
  CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
  REGENXBIO Inc.
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03488394
  Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
  Orchard Therapeutics

See all trials for Mucopolysaccharidosis type 1 →