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Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

NCT02758626 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2023-02-16

Study results available
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Summary

This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant epilepsy. Patients will receive 12 weeks of ataluren or placebo during each treatment period. Treatment Period 1 will be followed by a 4-week Washout Period. Based on ataluren PK and pharmacodynamic data, the 4-week washout period is deemed an appropriate length of time to eliminate any ataluren drug effects. Following the Washout Period, patients will crossover to receive the opposite treatment during Treatment Period 2 as follows: Patients receiving ataluren during Treatment Period 1 will receive placebo during Treatment Period 2. Patients receiving placebo during Treatment Period 1 will receive ataluren during Treatment Period 2.

Conditions

Interventions

DRUG

ataluren

Powder formulation

DRUG

Placebo

Powder formulation

Sponsors & Collaborators

Principal Investigators

  • Orrin Devinsky, MD · NYU Langone Health

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-11-30
Primary Completion
2021-02-27
Completion
2021-02-27
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02758626 on ClinicalTrials.gov