A PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, PARALLEL GROUP, MULTICENTER STUDY WITH OPEN-LABEL EXTENSION TO EVALUATE THE EFFICACY AND SAFETY OF FENFLURAMINE HYDROCHLORIDE IN STUDY PARTICIPANTS WITH RETT SYNDROME
A PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, PARALLEL GROUP, MULTICENTER STUDY WITH OPEN-LABEL EXTENSION TO EVALUATE THE EFFICACY AND SAFETY OF FENFLURAMINE HYDROCHLORIDE IN STUDY PARTICIPANTS WITH RETT SYNDROME
-
Clinical Trial Information
Trial Contact: Guerrero, Richard; Castro, Gabriela; Saint Pierre, Loudmy
Trial Phone: 3218411361 ; 1234567890 ; 321-841-3938
-
IRB No: S25.329.11
Protocol Abbrev: EP0247-Rett Syndrome
Principal Investigator: Vikram Prakash, MD
Phase: Drug: Phase III
Age Group: Pediatric
Secondary Protocol No: EP0247
Treatment: Fenfluramine Hydrochloride
Therapies Involved: Medication
ClinicalTrials.gov ID: NCT07503444
-
Objective
-To investigate the efficacy of fenfluramine HCl versus placebo in study participants with RTT
-To investigate the efficacy of fenfluramine HCl versus placebo on sleep in study participants with RTT
-To investigate the efficacy of fenfluramine HCl versus placebo on the ability to communicate in study participants with RTT
-To investigate the efficacy of fenfluramine HCl versus placebo on seizure in study participants with RTT and seizures
-To investigate the safety and tolerability of fenfluramine HCl versus placebo in study participants with RTT
-To investigate the efficacy of treatment with fenfluramine HCl versus placebo on the QoL of study participants with RTT
-To investigate the efficacy of fenfluramine HCl versus placebo on motor behavior in study participants with RTT
-To investigate the efficacy of fenfluramine HCl versus placebo on a global assessment of the severity of illness in study participants with RTT
-To investigate the benefit of treatment with fenfluramine HCl versus placebo on respiratory function in study participants with RTT
-To investigate the benefit of treatment with fenfluramine HCl versus placebo in study participants with RTT as reported by caregivers on an exit interview
-To investigate the long-term effectiveness of fenfluramine HCl in study participants with RTT
-To investigate the long-term effectiveness of fenfluramine HCl on the ability to communicate in study participants with RTT
-To investigate the long-term effectiveness of fenfluramine HCl on sleep in study participants with RTT
-To investigate the long-term effectiveness of fenfluramine HCl on seizures in study participants with RTT and seizures
-To investigate the safety and tolerability of fenfluramine HCl versus placebo in study participants with RTT
-To characterize the PK of fenfluramine and norfenfluramine in study participants with RTT
-To assess the PK/PD relationship using safety and efficacy endpoints in study participants with RTT receiving fenfluramine HCl -
Key Eligibility
Type of participant and disease characteristics
1. Participant can swallow the study intervention provided as a liquid solution or can take it by
G-tube.
2. Participant has typical or classic RTT according to the RettSearch Consortium 2010 revised
criteria.
3. Participant has a documented disease-causing mutation in the MECP2 gene.
4. Participant meets criteria for postregression for at least 6 months prior to Screening, defined
as:
UCB 28 Jul 2025
Clinical Study Protocol Fenfluramine Hydrochloride EP0247
Confidential Page 46 of 119
− No loss or degradation of ambulation (including gait, coordination, or independence of
walking/standing);
− No loss or degradation of hand function; no loss or degradation of speech (including
babbling, words, or previously developed communicative vocalizations);
− No loss or degradation of nonverbal communicative or social skills (including eye gaze,
using body to indicate communicative intent, or social attentiveness).
5. Participant has an RTT-CSS rating of 10 to 36 (inclusive).
6. Participant has a CGIS score of ≥4.
Informed consent
7. Participant has a legal representative capable of providing signed informed consent on behalf
of the participant as described in Section 10.1, which includes compliance with the
requirements and restrictions listed in the ICF and in this protocol.
Age
8. Participant is aged 5 to 35 years of age (inclusive) at the time of first administration of
investigational intervention.
Sex
9. Male or female.
− A male participant must agree to use contraception as detailed in Section 10.4 during the
treatment period and for at least 90 days after the final dose of study intervention and
refrain from donating sperm during this period.
− A female participant is eligible to participate if she is not pregnant (see Section 10.4), not
breastfeeding, and at least 1 of the following conditions applies:
◦ Not a WOCBP as defined in Section 10.4
OR
A WOCBP who agrees to follow the contraceptive guidance in Section 10.4 during
the Intervention Period and for at least 90 days after the final dose of study
intervention.
Weight
10. Participant must have a bodyweight of ≥12kg.
Other inclusions
11. Participant has a consistent caregiver who is ≥18 years of age at the Screening Visit. The
caregiver needs to be able to complete the caregiver assessments defined for the entire study.
Every attempt should be made to have the same evaluator complete the assessments for the
duration of the study.
