Trial · NCT02758626

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

NCT02758626, Phase 2, Completed, NYU Langone Health; results first posted 2023-02-16

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome. Trials, record NCT02758626. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02758626

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT number: NCT02758626
Status: Completed
Phases: Phase 2
Enrollment: 15 participants, actual
Lead sponsor: NYU Langone Health
Conditions: Epilepsy
Interventions: ataluren, Placebo
First posted: 2016-05-02
Last updated: 2023-02-16
Results first posted: 2023-02-16

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Ataluren Treatment Period	Baseline, Week 12 of Ataluren Treatment (Up to Week 28)	Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed, multiplied by 28. Percent change from Baseline will be defined as the frequency of seizures per 28 days during the Ataluren Treatment Period minus frequency of seizures per 28 days at Baseline, divided by the frequency of seizures per 28 days at Baseline, multiplied by 100. Negative percent change from Baseline indicates improvement.
Primary	Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Placebo Treatment Period	Baseline, Week 12 of Placebo Treatment (Up to Week 28)	Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed, multiplied by 28. Percent change from Baseline will be defined as the frequency of seizures per 28 days during the Placebo Treatment Period minus frequency of seizures per 28 days at Baseline, divided by the frequency of seizures per 28 days at Baseline, multiplied by 100. Negative percent change from Baseline indicates improvement.

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Ataluren Treatment Period	Baseline, Week 12 of Ataluren Treatment (Up to Week 28)		percent change	MEAN
PRIMARY	Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Placebo Treatment Period	Baseline, Week 12 of Placebo Treatment (Up to Week 28)		percent change	MEAN

Source ClinicalTrials.gov

Results by group2

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Ataluren Treatment Period	Ataluren		723.17	Standard Deviation 279.912	percent change
Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Placebo Treatment Period	Placebo		0.72	Standard Deviation 222.82	percent change

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-Blind Phase (Week 0 - Week 28)	STARTED	Ataluren Followed By Placebo	9
Double-Blind Phase (Week 0 - Week 28)	STARTED	Placebo Followed by Ataluren	6
Double-Blind Phase (Week 0 - Week 28)	COMPLETED	Ataluren Followed By Placebo	8
Double-Blind Phase (Week 0 - Week 28)	COMPLETED	Placebo Followed by Ataluren	6
Double-Blind Phase (Week 0 - Week 28)	NOT COMPLETED	Ataluren Followed By Placebo	1
Double-Blind Phase (Week 0 - Week 28)	NOT COMPLETED	Placebo Followed by Ataluren	0
Open-Label Extension (Week 28-Week 124)	STARTED	Ataluren Followed By Placebo	8
Open-Label Extension (Week 28-Week 124)	STARTED	Placebo Followed by Ataluren	6
Open-Label Extension (Week 28-Week 124)	COMPLETED	Ataluren Followed By Placebo	6
Open-Label Extension (Week 28-Week 124)	COMPLETED	Placebo Followed by Ataluren	3

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events130

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Viral upper respiratory tract infection	Infections and infestations	Ataluren	9	15
other	Viral upper respiratory tract infection	Infections and infestations	Placebo	2	14
other	Camphylobacter gastroenteritis	Infections and infestations	Ataluren	1	15
other	Camphylobacter gastroenteritis	Infections and infestations	Placebo	0	14
other	Gastroenteriritis viral	Infections and infestations	Ataluren	2	15
other	Gastroenteriritis viral	Infections and infestations	Placebo	0	14
other	Influenza	Infections and infestations	Ataluren	5	15
other	Influenza	Infections and infestations	Placebo	2	14
other	Viral infection	Infections and infestations	Ataluren	1	15
other	Viral infection	Infections and infestations	Placebo	1	14

1 to 10 of 130Source ClinicalTrials.gov

See all 130 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ataluren	0	15
Total, all-cause mortality	Placebo	0	14
Total, other adverse events	Ataluren	13	15
Total, other adverse events	Placebo	5	14
Total, serious adverse events	Ataluren	7	15
Total, serious adverse events	Placebo	1	14

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33538404	RESULT

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ataluren vs Placebo: deaths from any cause	Trial comparisons
Ataluren vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Source

Release: trials-2026-11-12-0.4.0
Release manifest SHA-256: 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
Registry capture: 2026-11-12
Results copy, AACT: 2026-03-23
NIH clinical studies file: 2026-11-13

Source ClinicalTrials.gov

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNone stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02758626

GET https://conductscience.org/api/trials/v1/NCT02758626/events?page=2

API reference For AI assistants