Trial · NCT02647359

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359, Phase 2, Completed, PTC Therapeutics; results first posted 2022-05-27

3 sourcestrials-2026-11-12-0.4.0

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ConductScience Library. Study of Ataluren in Participants With Nonsense Mutation Aniridia. Trials, record NCT02647359. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02647359

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Trial

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

Title: Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT number: NCT02647359
Status: Completed
Phases: Phase 2
Enrollment: 39 participants, actual
Lead sponsor: PTC Therapeutics
Conditions: Aniridia
Interventions: Ataluren, Placebo
First posted: 2016-01-06
Last updated: 2022-05-27
Results first posted: 2022-05-27

Source ClinicalTrials.gov

Registered outcomes11

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

List	Outcome	Time frame	Description
Primary	Percent Change From Baseline in Maximum Reading Speed of Oculus Unitas (OU) (Both Eyes) at Week 48, as Measured Using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts	Baseline, Week 48	MNREAD Acuity Chart can only be used to assess participants ≥8 years old. MNREAD Acuity Chart measures reading speed as a function of print size in participants with normal and low vision. The test consists of short sentences with print size decreasing by 0.1 log unit steps from a maximum of 1.3 logarithm of the minimum angle of resolution (logMAR) (equivalent to 20/400 or 6/120 when viewed at 40 centimeters \[cm\]) to -0.5 logMAR (equivalent to 20/6 or 6/2). An MNREAD Acuity Chart curve of reading speed vs print size has a typical shape for normally sighted persons and many low-vision individuals. This curve is characterized by 3 summary values. At large print sizes, reading speed remains fairly constant, forming a plateau that represents the maximum reading speed. As the print size decreases, a critical print size (CPS) is reached at which reading speed begins to decline rapidly. Finally, the smallest print size that can be read is defined as the reading acuity (RA).
Secondary	Change From Baseline in Reading Accessibility Index of Both Eyes at Week 48	Baseline, Week 48	Reading Accessibility Index is defined as the mean reading speed in words per minute (wpm) across the 10 largest physical print sizes on the MNREAD Acuity Chart, normalized by the value for a group of normally sighted young adults. For a viewing distance of 40 cm, this range of print sizes corresponds to 0.4 to 1.3 logMAR. This range of print sizes was chosen for 2 reasons. First, it sustains the manifest refraction spherical equivalent (MRS) in normally sighted persons. Second, it covers most contemporary printed text found in everyday life. Because the Reading Accessibility Index is normalized by the value for a group of normally sighted young adults (aged 18 to 39 years), a Reading Accessibility Index of 1.0 represents normal performance for this age group. Values less than 1.0 mean reduced accessibility to printed text within the range of print size encountered in daily life. Missing data was imputed using last observation carried forward (LOCF) method.
Secondary	Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Baseline, Week 48	The BCVA was evaluated using the Early Treatment Diabetic Retinopathy Study (ETDRS) Method. Missing data was imputed using LOCF method.
Secondary	Percent Change From Baseline in Maximum Reading Speed of Oculus Dexter (OD) (Right Eye) and Oculus Sinister (OS) (Left Eye) at Week 48	Baseline, Week 48	Maximum Reading Speed was measured using the MNREAD Acuity Chart, which can only be used to assess participants ≥8 years old. The MNREAD Acuity Chart measures reading speed as a function of print size in participants with normal and low vision. The test consists of short sentences with print size decreasing by 0.1 log unit steps from a maximum of 1.3 logMAR (equivalent to 20/400 or 6/120 when viewed at 40 cm) to -0.5 logMAR (equivalent to 20/6 or 6/2). An MNREAD Acuity Chart curve of reading speed vs print size has a typical shape for normally sighted persons and many low-vision individuals. This curve is characterized by 3 summary values. At large print sizes, reading speed remains fairly constant, forming a plateau that represents the maximum reading speed. As the print size decreases, a CPS is reached at which reading speed begins to decline rapidly. Finally, the smallest print size that can be read is defined as the RA.
Secondary	Change From Baseline in Reading Accessibility Index of Right Eye and Left Eye at Week 48	Baseline, Week 48	Reading Accessibility Index is defined as the mean reading speed in wpm across the 10 largest physical print sizes on the MNREAD Acuity Chart, normalized by the value for a group of normally sighted young adults. For a viewing distance of 40 cm, this range of print sizes corresponds to 0.4 to 1.3 logMAR. This range of print sizes was chosen for 2 reasons. First, it sustains the MRS in normally sighted persons. Second, it covers most contemporary printed text found in everyday life. Because the Reading Accessibility Index is normalized by the value for a group of normally sighted young adults (aged 18 to 39 years), a Reading Accessibility Index of 1.0 represents normal performance for this age group. Values less than 1.0 mean reduced accessibility to printed text within the range of print size encountered in daily life. Missing data was imputed using LOCF method.
Secondary	Change From Baseline in Critical Print Size (CPS) of Both Eyes, Right Eye, and Left Eye at Week 48	Baseline, Week 48	The MNREAD Acuity Chart measures reading speed as a function of print size in participants with normal and low vision. The test consists of short sentences with print size decreasing by 0.1 log unit steps from a maximum of 1.3 logMAR (equivalent to 20/400 or 6/120 when viewed at 40 cm) to -0.5 logMAR (equivalent to 20/6 or 6/2). An MNREAD Acuity Chart curve of reading speed vs print size has a typical shape for normally sighted persons and many low-vision individuals. This curve is characterized by 3 summary values. At large print sizes, reading speed remains fairly constant, forming a plateau that represents the maximum reading speed. As the print size decreases, a CPS is reached at which reading speed begins to decline rapidly. Finally, the smallest print size that can be read is defined as the RA. Missing data was imputed using LOCF method.
Secondary	Change From Baseline in Reading Acuity (RA) of Both Eyes, Right Eye, and Left Eye at Week 48	Baseline, Week 48	The MNREAD Acuity Chart measures reading speed as a function of print size in participants with normal and low vision. The test consists of short sentences with print size decreasing by 0.1 log unit steps from a maximum of 1.3 logMAR (equivalent to 20/400 or 6/120 when viewed at 40 cm) to -0.5 logMAR (equivalent to 20/6 or 6/2). An MNREAD Acuity Chart curve of reading speed vs print size has a typical shape for normally sighted persons and many low-vision individuals. This curve is characterized by 3 summary values. At large print sizes, reading speed remains fairly constant, forming a plateau that represents the maximum reading speed. As the print size decreases, a CPS is reached at which reading speed begins to decline rapidly. Finally, the smallest print size that can be read is defined as the RA. Missing data was imputed using LOCF method.
Secondary	Number of Participants With Change From Baseline in Severity of Corneal Keratopathy at Week 48	Baseline to Week 48	The severity of corneal keratopathy was reported as worsened, not change, or improve. Missing data were imputed using LOCF.
Secondary	Change From Baseline in Iris Area at Week 48	Baseline, Week 48	Missing data were imputed using LOCF.
Secondary	Change From Baseline in BCVA at Week 240	Baseline, Week 240	The BCVA was evaluated using the ETDRS Method. Missing data were imputed using LOCF method.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

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 Maximum Reading Speed of Oculus Unitas (OU) (Both Eyes) at Week 48, as Measured Using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts	Baseline, Week 48	The intent-to-treat (ITT) population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.	percent change	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Reading Accessibility Index of Both Eyes at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.	units on a scale	MEAN
SECONDARY	Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	LogMAR	MEAN
SECONDARY	Percent Change From Baseline in Maximum Reading Speed of Oculus Dexter (OD) (Right Eye) and Oculus Sinister (OS) (Left Eye) at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	percent change	MEAN
SECONDARY	Change From Baseline in Reading Accessibility Index of Right Eye and Left Eye at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	units on a scale	MEAN
SECONDARY	Change From Baseline in Critical Print Size (CPS) of Both Eyes, Right Eye, and Left Eye at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	LogMAR	MEAN
SECONDARY	Change From Baseline in Reading Acuity (RA) of Both Eyes, Right Eye, and Left Eye at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	LogMAR	MEAN
SECONDARY	Number of Participants With Change From Baseline in Severity of Corneal Keratopathy at Week 48	Baseline to Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline in Iris Area at Week 48	Baseline, Week 48	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	millimeter square (mm^2)	MEAN
SECONDARY	Change From Baseline in BCVA at Week 240	Baseline, Week 240	The ITT population included all randomized participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.	logMAR	MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group49

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 Maximum Reading Speed of Oculus Unitas (OU) (Both Eyes) at Week 48, as Measured Using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts	Ataluren		9.87	Standard Error 7.425	percent change
Percent Change From Baseline in Maximum Reading Speed of Oculus Unitas (OU) (Both Eyes) at Week 48, as Measured Using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts	Placebo		-0.89	Standard Error 11.809	percent change
Change From Baseline in Reading Accessibility Index of Both Eyes at Week 48	Ataluren		0.10	Standard Deviation 0.241	units on a scale
Change From Baseline in Reading Accessibility Index of Both Eyes at Week 48	Placebo		0.02	Standard Deviation 0.267	units on a scale
Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Ataluren	Left Eye	-0.00	Standard Deviation 0.076	LogMAR
Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Placebo	Left Eye	0.01	Standard Deviation 0.103	LogMAR
Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Ataluren	Right Eye	0.03	Standard Deviation 0.125	LogMAR
Change From Baseline in Best Corrected Visual Acuity (BCVA) at Week 48	Placebo	Right Eye	-0.04	Standard Deviation 0.138	LogMAR
Percent Change From Baseline in Maximum Reading Speed of Oculus Dexter (OD) (Right Eye) and Oculus Sinister (OS) (Left Eye) at Week 48	Ataluren	Left Eye	8.70	Standard Deviation 36.464	percent change
Percent Change From Baseline in Maximum Reading Speed of Oculus Dexter (OD) (Right Eye) and Oculus Sinister (OS) (Left Eye) at Week 48	Placebo	Left Eye	-4.11	Standard Deviation 38.380	percent change

1 to 10 of 49Source ClinicalTrials.gov

See all 49 results by group

Participant flow20

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

Period	Milestone	Group	Participants
Stage 1: Double-Masked Period (48 Weeks)	STARTED	Ataluren	26
Stage 1: Double-Masked Period (48 Weeks)	STARTED	Placebo	13
Stage 1: Double-Masked Period (48 Weeks)	Received at Least 1 Dose of Study Drug	Ataluren	26
Stage 1: Double-Masked Period (48 Weeks)	Received at Least 1 Dose of Study Drug	Placebo	13
Stage 1: Double-Masked Period (48 Weeks)	COMPLETED	Ataluren	22
Stage 1: Double-Masked Period (48 Weeks)	COMPLETED	Placebo	12
Stage 1: Double-Masked Period (48 Weeks)	NOT COMPLETED	Ataluren	4
Stage 1: Double-Masked Period (48 Weeks)	NOT COMPLETED	Placebo	1
Stage 2: Open-Label Extension (96 Weeks)	STARTED	Ataluren	22
Stage 2: Open-Label Extension (96 Weeks)	STARTED	Placebo	11

1 to 10 of 20Source ClinicalTrials.gov

See all 20 participant flow

Adverse events333

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	Abdominal pain upper	Gastrointestinal disorders	Stage 1: Ataluren	5	26
other	Abdominal pain upper	Gastrointestinal disorders	Stage 1: Placebo	3	13
other	Abdominal pain upper	Gastrointestinal disorders	Overall Ataluren Exposure	9	37
other	Vomiting	Gastrointestinal disorders	Stage 1: Ataluren	5	26
other	Vomiting	Gastrointestinal disorders	Stage 1: Placebo	3	13
other	Vomiting	Gastrointestinal disorders	Overall Ataluren Exposure	8	37
other	Nausea	Gastrointestinal disorders	Stage 1: Ataluren	4	26
other	Nausea	Gastrointestinal disorders	Stage 1: Placebo	2	13
other	Nausea	Gastrointestinal disorders	Overall Ataluren Exposure	4	37
other	Flatulence	Gastrointestinal disorders	Stage 1: Ataluren	3	26

1 to 10 of 333Source ClinicalTrials.gov

See all 333 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Stage 1: Ataluren	0	26
Total, all-cause mortality	Stage 1: Placebo	0	13
Total, all-cause mortality	Overall Ataluren Exposure	0	37
Total, other adverse events	Stage 1: Ataluren	22	26
Total, other adverse events	Stage 1: Placebo	10	13
Total, other adverse events	Overall Ataluren Exposure	35	37
Total, serious adverse events	Stage 1: Ataluren	1	26
Total, serious adverse events	Stage 1: Placebo	0	13
Total, serious adverse events	Overall Ataluren Exposure	1	37

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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 comparisons0

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

None in this release.

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.

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