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HIT study: IZCARGO (pabinafusp alfa) treatment in Hunter syndrome
jRCT1030250124Active, not recruiting
- Sponsor
- JCR Pharmaceuticals
- Start
- Sep 2025
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Open-label trial of pabinafusp alfa in mucopolysaccharidosis type 2 (2 arms), sponsored by JCR Pharmaceuticals. Primary endpoints: Change in visual function over 104 weeks, Change in auditory function over 104 weeks and 3 more. Started Sep 2025; status: active, not recruiting.
02
Drugs
pabinafusp alfa
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Conditions
mucopolysaccharidosis type 2
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