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

IRB No: S25.329.11

Principal Investigator: Prakash, Vikram MD

Phase: N/A

Age Group: CHILDADULT

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

Share:

Related Clinical Trials

An Interventional Phase Ib/II Study to Evaluate the Safety and Efficacy of PF-08634404 Monotherapy and in Combination with Other Anticancer Agents in Adult Participants with Locally Advanced or Metastatic Renal Cell Carcinoma
Percutaneous or Endoscopic Gallbladder Drainage: The PEG Trial
A Phase III, Randomized, Open-Label Study Comparing Efficacy and Safety of Sacituzumab Tirumotecan (sac-TMT, MK-2870) as a Monotherapy and in Combination with Pembrolizumab (MK-3475) versus Treatment of Physicians Choice in Participants with Previously Untreated Locally Recurrent Unresectable or Metastatic Triple-Negative Breast Cancer Expressing PD L1 at CPS Less than 10 (TroFuse-11)
ColoSeal Intraluminal Colonic Diversion (ICD) System Anastomotic Protection Pivotal Study