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Long-term Safety of Sirolimus (Rapalimus granules) in Pendred Syndrome/DFNB4
jRCTs031240673Recruiting
- Sponsor
- Nobelpharma Co., Ltd.
- Start
- May 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of sirolimus in Pendred syndrome (PDS) and autosomal recessive nonsyndromic deafness 4 (DFNB4), sponsored by Nobelpharma Co., Ltd. Primary endpoint: Adverse events and side effects. Started May 2025; status: recruiting.
02
Drugs
sirolimus
03
Conditions
Pendred syndrome (PDS)autosomal recessive nonsyndromic deafness 4 (DFNB4)
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