Trial · NCT00458341

A Study of Ataluren in Pediatric Participants With Cystic Fibrosis

NCT00458341, Phase 2, Completed, PTC Therapeutics; results first posted 2020-03-06

3 sourcestrials-2026-11-12-0.4.0

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ConductScience Library. A Study of Ataluren in Pediatric Participants With Cystic Fibrosis. Trials, record NCT00458341. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00458341

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Trial

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

Title: A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT number: NCT00458341
Status: Completed
Phases: Phase 2
Enrollment: 30 participants, actual
Lead sponsor: PTC Therapeutics
Conditions: Cystic Fibrosis
Interventions: Ataluren
First posted: 2007-04-10
Last updated: 2020-03-06
Results first posted: 2020-03-06

Source ClinicalTrials.gov

Registered outcomes17

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

List	Outcome	Time frame	Description
Primary	Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	Nasal transepithelial potential difference (TEPD) was assessed in each participant using standardized techniques. Warmed solutions of Ringer's solution, amiloride, chloride-free gluconate, isoproterenol, and adenosine triphosphate (ATP) were perfused for ≥3-minute sequentially through a nasal catheter while a voltage tracing was recorded. Total chloride transport was computed for each nostril. The total chloride transport values were calculated by subtracting the voltages at the end of a perfusion from the voltage at the end of an earlier perfusion (isoproterenol - amiloride). The average of the values for each nostril was computed. If the assessment was available in only 1 nostril, this value was used as if it were the average of both nostrils. Baseline data for Cycle 1 and Cycle 2 and change from Baseline data at Day 14 of Cycles 1 and 2 are presented.
Primary	Number of Participants With a Chloride Transport Response at Day 14 of Cycles 1 and 2	Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	Nasal TEPD was assessed in each participant using standardized techniques. Warmed solutions of Ringer's solution, amiloride, chloride-free gluconate, isoproterenol, and ATP were perfused for ≥3-minute sequentially through a nasal catheter while a voltage tracing was recorded. Total chloride transport was computed for each nostril. The total chloride transport values were calculated by subtracting the voltages at the end of a perfusion from the voltage at the end of an earlier perfusion (isoproterenol - amiloride). The average of the values for each nostril was computed. If the assessment was available in only 1 nostril, this value was used as if it were the average of both nostrils. Response to study treatment defined as an increase in total chloride transport as indicated by a change of at least -5 mV in nasal TEPD.
Primary	Number of Participants With Normalization of Chloride Transport Between Baseline and Day 14 of Cycles 1 and 2	Overall Baseline and Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	Nasal TEPD was assessed in each participant using standardized techniques. Warmed solutions of Ringer's solution, amiloride, chloride-free gluconate, isoproterenol, and ATP were perfused for ≥3-minute sequentially through a nasal catheter while a voltage tracing was recorded. Total chloride transport was computed for each nostril. The total chloride transport values were calculated by subtracting the voltages at the end of a perfusion from the voltage at the end of an earlier perfusion (isoproterenol - amiloride). The average of the values for each nostril was computed. If the assessment was available in only 1 nostril, this value was used as if it were the average of both nostrils. Normalization of chloride transport (normal range \[NR\]) was defined as nasal TEPD that was at least as electrically negative as -5 mV. Normalization in chloride transport can also be referred to as hyperpolarization.
Secondary	Change From Baseline in Parameters of Transepithelial Difference at Day 14 of Cycles 1 and 2	Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	To assess TEPD, warmed solutions of Ringer's solution, amiloride, chloride-free gluconate, isoproterenol and ATP were perfused for ≥3-minutes sequentially through a nasal catheter while a voltage tracing was recorded. Total chloride transport was computed per nostril. Totals were calculated by subtracting voltages at end of perfusion from voltage at end of earlier perfusion for: sodium transport (amiloride-Ringer's solution), intrinsic chloride transport (chloride-free gluconate-amiloride), stimulated chloride transport (isoproterenol-chloride-free gluconate), total potential difference (isoproterenol-Ringer's solution), and ATP-mediated chloride transport (ATP-isoproterenol). Basal potential difference voltage was obtained at end of Ringer's solution perfusion. Average values per nostril were computed. If assessment was available in only 1 nostril, the value was used as if it's the average of both nostrils. Baseline data for Cycles 1 and 2 and change from Baseline data are presented.
Secondary	Change From Baseline in CFTR Protein in Nasal Mucosa as Determined by Immunofluorescence at Overall Day 56	Overall Baseline, Overall Day 56	The immunofluorescence staining of normal epithelial cells (for example, from nasal mucosal curettage) reveals the presence of cystic fibrosis transmembrane regulator (CFTR) protein at the apical surface. Cells were stained with antibodies that recognized an epitope in the C-terminal portion of the CFTR protein, and the cells were imaged microscopically. The percentage of epithelial cells that showed apical CFTR staining was determined by 2 expert readers who were blinded to the timepoint at which the samples were obtained. The scores of the reviewers were averaged to determine the final percentage of cells with apical CFTR. Overall Baseline data for the study and change from overall Baseline data at overall Day 56 are presented.
Secondary	Change From Baseline in Nonsense Mutation CFTR mRNA in Nasal Mucosa as Determined by Quantitative Real-Time Polymerase Chain Reaction (RT-PCR) Assay at Overall Day 42	Overall Baseline, Overall Day 42	The collection and processing of the nasal mucosal curettage from each nostril of each participant for measurement of CFTR protein by immunofluorescence and for quantification of CFTR messenger ribonucleic acid (mRNA) was performed using standardized techniques. The slides were processed and immunostained for detection of CFTR protein. Microscopic images were to be captured photographically for analysis. Because the nasal brushing used to collect nasal mucosal epithelial cells did not result in collection of sufficient cells for RT-PCR to be performed, an insufficient number of paired baseline and follow-up samples were available for analysis. As a result, no data were available to evaluate the effects of ataluren on CFTR mRNA.
Secondary	Change From Baseline in Pulmonary Function as Measured by Spirometry at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	Pulmonary function tests, including forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and forced expiratory flow25-75 (FEF25-75), were measured using standard spirometry techniques. Overall Baseline data for the study and change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.
Secondary	Change From Baseline in Sputum Markers of Inflammation (Free Elastase) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	The inflammatory marker free elastase was measured in induced sputum from each participant. Hypertonic saline (3%) inhalation was used to induce the sputum (with efforts made to avoid oropharyngeal contamination). The sputum sample was divided into 4 aliquots (1 aliquot each for determination of cell count, IL-8 level, and elastase activity and 1 aliquot for potential future viscosity measurements). Change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.
Secondary	Change From Baseline in Sputum Markers of Inflammation (Matrix Metalloproteinase 9 [MMP-9] Active) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	The inflammatory marker MMP-9 active was measured in induced sputum from each participant. Hypertonic saline (3%) inhalation was used to induce the sputum (with efforts made to avoid oropharyngeal contamination). The sputum sample was divided into 4 aliquots (1 aliquot each for determination of cell count, IL-8 level, and elastase activity and 1 aliquot for potential future viscosity measurements). Change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.
Secondary	Change From Baseline in Sputum Markers of Inflammation (Tumor Necrosis Factor-Alpha [TNF-α], Interleukin-8 [IL-8], Transforming Growth Factor Beta 1 [TGF-β1]) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	The inflammatory markers TNF-α, IL-8, and TGF-β1 were measured in induced sputum from each participant. Hypertonic saline (3%) inhalation was used to induce the sputum (with efforts made to avoid oropharyngeal contamination). The sputum sample was divided into 4 aliquots (1 aliquot each for determination of cell count, IL-8 level, and elastase activity and 1 aliquot for potential future viscosity measurements). Change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes17

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	All randomized participants who received at least 1 dose of study drug and had evaluable chloride transport data.	millivolts (mV)	MEAN
PRIMARY	Number of Participants With a Chloride Transport Response at Day 14 of Cycles 1 and 2	Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	All randomized participants who received at least 1 dose of study drug and had evaluable chloride transport response data.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Normalization of Chloride Transport Between Baseline and Day 14 of Cycles 1 and 2	Overall Baseline and Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	All randomized participants who received at least 1 dose of study drug and had evaluable normalization of chloride transport data.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline in Parameters of Transepithelial Difference at Day 14 of Cycles 1 and 2	Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)	All randomized participants who received at least 1 dose of study drug and had evaluable transepithelial difference data.	mV	MEAN
SECONDARY	Change From Baseline in CFTR Protein in Nasal Mucosa as Determined by Immunofluorescence at Overall Day 56	Overall Baseline, Overall Day 56	All randomized participants who received at least 1 dose of study drug and had evaluable apical cell data.	percentage of apical cells	MEAN
SECONDARY	Change From Baseline in Nonsense Mutation CFTR mRNA in Nasal Mucosa as Determined by Quantitative Real-Time Polymerase Chain Reaction (RT-PCR) Assay at Overall Day 42	Overall Baseline, Overall Day 42	All randomized participants who received at least 1 dose of study drug and had evaluable CFTR mRNA data.
SECONDARY	Change From Baseline in Pulmonary Function as Measured by Spirometry at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	All randomized participants who received at least 1 dose of study drug and had evaluable pulmonary function data.	percent of predicted	MEAN
SECONDARY	Change From Baseline in Sputum Markers of Inflammation (Free Elastase) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	All randomized participants who received at least 1 dose of study drug and had evaluable sputum markers of inflammation data.	micrograms/milliliters (μg/mL)	MEAN
SECONDARY	Change From Baseline in Sputum Markers of Inflammation (Matrix Metalloproteinase 9 [MMP-9] Active) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	All randomized participants who received at least 1 dose of study drug and had evaluable sputum markers of inflammation data.	nanograms/milliliter (ng/mL)	MEAN
SECONDARY	Change From Baseline in Sputum Markers of Inflammation (Tumor Necrosis Factor-Alpha [TNF-α], Interleukin-8 [IL-8], Transforming Growth Factor Beta 1 [TGF-β1]) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56	Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56	All randomized participants who received at least 1 dose of study drug and had evaluable sputum markers of inflammation data.	picograms/milliliter (pg/mL)	MEAN

1 to 10 of 17Source ClinicalTrials.gov

See all 17 outcomes

Results by group190

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

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	Baseline of Cycle 1	1.45	Standard Deviation 5.671	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	Baseline of Cycle 1	0.66	Standard Deviation 4.563	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	Change at Day 14 of Cycle 1	-2.81	Standard Deviation 6.543	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	Change at Day 14 of Cycle 1	-2.69	Standard Deviation 7.289	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	Baseline of Cycle 2	-0.92	Standard Deviation 4.474	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	Baseline of Cycle 2	-3.76	Standard Deviation 6.729	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	Change at Day 14 of Cycle 2	-2.39	Standard Deviation 5.426	millivolts (mV)
Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	Change at Day 14 of Cycle 2	1.10	Standard Deviation 7.179	millivolts (mV)
Number of Participants With a Chloride Transport Response at Day 14 of Cycles 1 and 2	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	Day 14 of Cycle 1	4		Participants
Number of Participants With a Chloride Transport Response at Day 14 of Cycles 1 and 2	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	Day 14 of Cycle 1	4		Participants

1 to 10 of 190Source ClinicalTrials.gov

See all 190 results by group

Participant flow16

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

Period	Milestone	Group	Participants
Cycle 1	STARTED	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	15
Cycle 1	STARTED	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	15
Cycle 1	Received at Least 1 Dose of Study Drug	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	15
Cycle 1	Received at Least 1 Dose of Study Drug	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	15
Cycle 1	COMPLETED	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	14
Cycle 1	COMPLETED	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	15
Cycle 1	NOT COMPLETED	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	1
Cycle 1	NOT COMPLETED	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	0
Cycle 2	STARTED	Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	15
Cycle 2	STARTED	Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	15

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events62

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	Gastrointestinal disorders	Ataluren 4, 4, and 8 mg/kg	6	30
other	Abdominal pain	Gastrointestinal disorders	Ataluren 10, 10, and 20 mg/kg	8	30
other	Diarrhoea	Gastrointestinal disorders	Ataluren 4, 4, and 8 mg/kg	3	30
other	Diarrhoea	Gastrointestinal disorders	Ataluren 10, 10, and 20 mg/kg	2	30
other	Nausea	Gastrointestinal disorders	Ataluren 4, 4, and 8 mg/kg	2	30
other	Nausea	Gastrointestinal disorders	Ataluren 10, 10, and 20 mg/kg	0	30
other	Vomiting	Gastrointestinal disorders	Ataluren 4, 4, and 8 mg/kg	1	30
other	Vomiting	Gastrointestinal disorders	Ataluren 10, 10, and 20 mg/kg	1	30
other	Chest pain	General disorders	Ataluren 4, 4, and 8 mg/kg	1	30
other	Chest pain	General disorders	Ataluren 10, 10, and 20 mg/kg	0	30

1 to 10 of 62Source ClinicalTrials.gov

See all 62 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ataluren 4, 4, and 8 mg/kg
Total, all-cause mortality	Ataluren 10, 10, and 20 mg/kg
Total, other adverse events	Ataluren 4, 4, and 8 mg/kg	19	30
Total, other adverse events	Ataluren 10, 10, and 20 mg/kg	24	30
Total, serious adverse events	Ataluren 4, 4, and 8 mg/kg	1	30
Total, serious adverse events	Ataluren 10, 10, and 20 mg/kg	1	30

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
20622033	DERIVED

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 comparisons1

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

Comparison
Ataluren 4, 4, and 8 mg/kg vs Ataluren 10, 10, and 20 mg/kg: serious adverse events

Source 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.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license 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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