First-in-human Study of UX016 in GNEM

Conditions

• GNE Myopathy

Eligibility Criteria

Sex

ALL

Age

18 Years - 55 Years

Healthy Volunteers

Not accepted

Interventions

• UX016 — DRUG
  Tablets for oral use
• Placebo — OTHER
  Tablets for oral use. Tablets will match the UX016 tablets, but contain no active ingredients

Study Details

The main goal of this study is to evaluate the safety of UX016 and to evaluate the impact of UX016 on muscle strength in adults with GNE Myopathy (GNEM).

Key Dates

Start date

Oct 31, 2026

Status verified

Mar 2026

Primary completion

Dec 31, 2028

Completion

Dec 31, 2028

Study Design

Enrollment

24 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Lower Dose UX016 -> Extension Period
  Participants will be randomized 3:1. Those randomized to lower dose will receive UX016 daily following up to two single dose administrations. After 48 weeks of daily dosing, participants will be eligible to enter the extension period.
• Experimental: Higher Dose UX016 -> Extension Period
  Participants will be randomized 3:1. Those randomized to higher dose will receive UX016 daily following a single dose administration. After 48 weeks of daily dosing, participants will be eligible to enter the extension period.
• Placebo Comparator: Placebo -> Extension Period
  Participants will be randomized 3:1. Those randomized to placebo will receive placebo per the same Lower or Higher Dose UX016 cohorts. After 48 weeks of daily dosing, participants will be eligible to enter the extension period and receive UX016 at the cohort assigned dose level.

Primary Outcome Measure

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 104 ]

Central Contacts

• Patient Contact: Trial Recruitment
  1-888-756-8657
  [email protected]
• HCP Contact: Medical Information
  1-888-756-8657
  [email protected]

Locations (2)

Find similar trials in Orange, CA