A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients with Wolfram Syndrome

The purpose of this research study is to find out how safe and tolerable the investigational drug AMX0035 is in participants with Wolfram syndrome. It will also see if AMX0035 may slow the worsening of clinical signs and symptoms of Wolfram syndrome.

This study is actively recruiting.

Principal Investigator: Fumihiko Urano, MD

Study Contact
Stacy Hurst

ClinicalTrials.gov Identifier
NCT05676034

The purpose of this research study is to find out how safe and tolerable the investigational drug AMX0035 is in participants with Wolfram syndrome. It will also see if AMX0035 may slow the worsening of clinical signs and symptoms of Wolfram syndrome. There are currently no approved drugs for Wolfram syndrome. Study participation will last about 8 months and participants will take the study medication for about 24 weeks.

This study consists of 3 periods:
• A screening period starting from time of consent and lasting up to 28 days, during which tests and procedures will be done to determine eligibility to participate.
• A treatment period starting at baseline visit and lasting 24 weeks, or 168 days, and ending at Week 24/End of Treatment (EOT) visit.
• A post-treatment period starting from Week 24/EOT visit and lasting 4 weeks, and ending at Week 28 visit.