A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
UCB BIOSCIENCES, Inc.
Study ID
NCT07503444
Phase
PHASE3
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
5 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • fenfluramine hydrochloride — DRUG
    Oral solution
  • Placebo — OTHER
    Oral solution

Study Details

The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).

Key Dates

Start date
May 31, 2026
Status verified
May 2026
Primary completion
Aug 1, 2028
Completion
Nov 15, 2030

Study Design

Enrollment
200 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: fenfluramine hydrochloride
    Participants will receive fenfluramine hydrochloride for 14 weeks in a double-blind period (including titration and maintenance), followed by a 2-week double-blind transition period. This is followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.
  • Placebo Comparator: Placebo
    Participants will receive a matching placebo for 14 weeks in a double-blind period (including titration and maintenance). After this period, they transition into a 2-week double-blind period with fenfluramine hydrochloride, followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.

Primary Outcome Measure

Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score [ Time Frame: From Baseline (Day 1) to Week 14 ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Ep0247 21010Little RockArkansas72202-
Ep0247 21005HoustonTexas77030-

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Ep0247 21010· Little Rock, AREp0247 21005· Houston, TX

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