NCT00458341 is a Phase 2 clinical trial of Ataluren in Cystic Fibrosis, sponsored by PTC Therapeutics.

Who is sponsoring NCT00458341?

NCT00458341 is sponsored by PTC Therapeutics.

What drugs are being tested in NCT00458341?

Interventions in NCT00458341: Ataluren.

What condition does NCT00458341 study?

NCT00458341 studies Cystic Fibrosis.

Is NCT00458341 still recruiting?

NCT00458341 is currently completed. Last updated 6 March 2020.

Are results published for NCT00458341?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2020-03-06 · How we verify

NCT00458341

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Study of Ataluren in Pediatric Participants With Cystic Fibrosis

Completed Phase 2 Results posted Last updated 6 March 2020

What this trial tests

Phase 2 trial testing Ataluren in Cystic Fibrosis in 30 participants. Completed in 29 February 2008.

Timeline

23 March 2007

Primary endpoint
29 February 2008

29 February 2008

Quick facts

Lead sponsor	PTC Therapeutics
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	none
Primary purpose	treatment
Enrollment	30
Start date	23 March 2007
Primary completion	29 February 2008
Estimated completion	29 February 2008
Sites	3 locations across Belgium, France

Drugs / interventions tested

Ataluren (ATALUREN) — full drug profile →

Conditions studied

Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

PTC Therapeutics — full company profile →

Who can join

Adults 6 to 18, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2 Primary · 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 t

Baseline of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	1.45	± 5.671
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	0.66	± 4.563

Change at Day 14 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-2.81	± 6.543
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-2.69	± 7.289

Baseline of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.92	± 4.474
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-3.76	± 6.729

Change at Day 14 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-2.39	± 5.426
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	1.10	± 7.179

Number of Participants With a Chloride Transport Response at Day 14 of Cycles 1 and 2 Primary · 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 a

Day 14 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	4
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	4

Day 14 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	4
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	2

Number of Participants With Normalization of Chloride Transport Between Baseline and Day 14 of Cycles 1 and 2 Primary · Overall Baseline and Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)

Within NR at Baseline of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	0
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	0

Within NR at Day 14 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	1
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	5

Outside NR at Baseline/in NR at Day 14 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	1
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	5

Within NR at Baseline of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	2
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	4

Within NR at Day 14 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	6
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	3

Outside NR at Baseline/in NR at Day 14 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	6
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	2

Change From Baseline in Parameters of Transepithelial Difference at Day 14 of Cycles 1 and 2 Secondary · 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 (isopr

Basal nasal TEPD, Baseline of Cycle (C)1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-53.91	± 18.81
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-56.51	± 20.68

Basal nasal TEPD, Change at Day (D)14 of C1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	9.31	± 17.72
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-3.70	± 13.33

Basal nasal TEPD, Baseline of C2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-46.28	± 14.46
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-50.02	± 17.09

Basal nasal TEPD, Change at D14 of C2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-6.31	± 19.33
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-5.22	± 24.48

Sodium transport, Baseline of C1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	34.38	± 17.72
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	36.40	± 18.44

Sodium transport, Change at D14 of C1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-4.97	± 15.30
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	1.55	± 18.39

Sodium transport, Baseline of C2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	28.85	± 14.31
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	32.15	± 17.72

Sodium transport, Change at D14 of C2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	6.38	± 19.81
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	5.50	± 20.69

Change From Baseline in CFTR Protein in Nasal Mucosa as Determined by Immunofluorescence at Overall Day 56 Secondary · 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 perce

Overall Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	15.95	± 16.797
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	10.47	± 9.270

Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	10.45	± 24.337
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	22.89	± 29.389

Change From Baseline in Pulmonary Function as Measured by Spirometry at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56 Secondary · 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.

FEV1, Overall Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	92.68	± 22.645
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	85.14	± 27.850

FEV1, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	2.73	± 5.848
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-4.60	± 7.579

FEV1, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.46	± 11.031
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	0.02	± 8.801

FEV1, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.36	± 12.936
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-0.39	± 8.191

FVC, Overall Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	101.83	± 13.294
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	95.34	± 22.515

FVC, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	2.52	± 7.216
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-2.33	± 6.643

FVC, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.44	± 7.433
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-0.44	± 7.433

FVC, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.65	± 9.117
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	1.02	± 6.796

Change From Baseline in Sputum Markers of Inflammation (Free Elastase) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56 Secondary · 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.

Free Elastase, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	108.85	± 107.617
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	60.92	± 56.170

Free Elastase, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-37.78	± 95.154
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	74.66	± 83.258

Free Elastase, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-24.40	± 93.804
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	37.33	± 65.240

Free Elastase, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	12.47	± 84.427
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	57.18	± 139.211

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 Secondary · 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.

MMP-9 Active, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	69431.28	± 109894.966
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	20908.56	± 28441.612

MMP-9 Active, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-44527.45	± 92493.455
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	71639.89	± 150716.037

MMP-9 Active, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-51330.37	± 108057.265
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	14947.54	± 69337.080

MMP-9 Active, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-49644.49	± 86334.812
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	56884.39	± 158498.793

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 Secondary · 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.

IL-8, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	352539.57	± 331865.227
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	121879.75	± 83684.594

IL-8, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-171764.67	± 296343.195
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	98483.55	± 154432.714

IL-8, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-71206.95	± 289379.557
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-4087.25	± 127038.632

IL-8, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-121626.94	± 263731.747
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	52880.35	± 176223.116

TGF-b1, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	80.84	± 280.044
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	322.56	± 612.153

TGF-b1, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	0	± 0
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-322.56	± 612.15

TGF-b1, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-97.01	± 306.773
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-168.91	± 774.276

TGF-b1, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-61.32	± 367.788
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-392.21	± 771.006

Change From Baseline in Sputum Markers of Inflammation (Uridine-5'-Triphosphate [UTP]) at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56 Secondary · Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56

The inflammatory marker UTP 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.

UTP, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	800.27	± 509.794
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	483.31	± 222.102

UTP, Change at Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-202.85	± 427.324
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	411.50	± 359.669

UTP, Change at Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-200.94	± 549.730
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	198.03	± 436.888

UTP, Change at Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-220.16	± 414.258
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-15.86	± 242.686

Change From Baseline in Clinically Significant Neutrophil Levels in Blood at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56 Secondary · Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56

To assess inflammatory markers in the blood, neutrophil levels in the blood were measured. Higher levels of neutrophils are indicative of more inflammation. Change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.

Neutrophils - Absolute, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	3.555	± 1.2328
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	4.709	± 3.0884

Neutrophils - Absolute, Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.332	± 1.3416
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-0.330	± 2.7710

Neutrophils - Absolute, Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.290	± 1.4881
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-0.748	± 2.2473

Neutrophils - Absolute, Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.114	± 1.4724
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	-0.931	± 2.7452

Change From Baseline in Clinically Significant Serum Levels of C-Reactive Protein at Day 14 or 15 of Cycles 1 and 2 and Overall Day 56 Secondary · Overall Baseline, Day 14 or 15 of Cycle 1 and Cycle 2 (1 cycle=28 days), and Overall Day 56

To assess inflammatory markers in the blood, serum levels of C-reactive protein were measured. Higher levels of C-reactive protein are indicative of more inflammation. Change from overall Baseline data at Day 14 or 15 of Cycles 1 and 2 and at overall Day 56 are presented.

C-Reactive Protein, Baseline

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	6.432	± 4.9133
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	8.273	± 7.8838

C-Reactive Protein, Day 14 or 15 of Cycle 1

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.150	± 1.6337
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	7.059	± 16.9563

C-Reactive Protein, Day 14 or 15 of Cycle 2

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	-0.233	± 0.7761
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	2.933	± 12.0167

C-Reactive Protein, Overall Day 56

Group	Value	95% CI
Ataluren 4, 4, and 8 mg/kg, Then Ataluren 10, 10, and 20 mg/kg	2.361	± 7.4765
Ataluren 10, 10, and 20 mg/kg, Then Ataluren 4, 4, and 8 mg/kg	1.240	± 7.7170

Adverse events — posted to ClinicalTrials.gov

Time frame: Overall Baseline up to Overall Day 56. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ataluren 4, 4, and 8 mg/kg

Serious: 1/30 (3%)

Deaths: —

Ataluren 10, 10, and 20 mg/kg

Serious: 1/30 (3%)

Deaths: —

Serious adverse events (2 terms)

Reaction	System	Ataluren 4, 4, and 8 mg/kg	Ataluren 10, 10, and 20 mg…
Cystic fibrosis pulmonary exacerbation	Respiratory, thoracic and mediastinal disorders	—	—
Haematemesis	Gastrointestinal disorders	—	—

Other adverse events (29 terms — click to expand)

Reaction	System	Ataluren 4, 4, and 8 mg/kg	Ataluren 10, 10, and 20 mg…
Abdominal pain	Gastrointestinal disorders	—	—
Rhinitis	Infections and infestations	—	—
Cystic fibrosis pulmonary exacerbation	Respiratory, thoracic and mediastinal disorders	—	—
Diarrhoea	Gastrointestinal disorders	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Productive cough	Respiratory, thoracic and mediastinal disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Pyrexia	General disorders	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Asthenia	General disorders	—	—
Headache	Nervous system disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Chest pain	General disorders	—	—
Nasopharyngitis	Infections and infestations	—	—
Stenotrophomonas infection	Infections and infestations	—	—
Haemoptysis	Respiratory, thoracic and mediastinal disorders	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—
Rash pruritic	Skin and subcutaneous tissue disorders	—	—
Ascites	Gastrointestinal disorders	—	—
Constipation	Gastrointestinal disorders	—	—
Diarrhoea haemorrhagic	Gastrointestinal disorders	—	—
Dysphagia	Gastrointestinal disorders	—	—
Fatigue	General disorders	—	—
Thirst	General disorders	—	—
Laryngitis	Infections and infestations	—	—
Blood creatinine increased	Investigations	—	—
Gamma-glutamyltransferase increased	Investigations	—	—
Tendonitis	Musculoskeletal and connective tissue disorders	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—

Most-reported serious reactions: Cystic fibrosis pulmonary exacerbation, Haematemesis.

Data from ClinicalTrials.gov NCT00458341 adverse events section.

Sponsor's own description

In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase functional CFTR protein in the cells of participants with CF due to a nonsense mutation.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.
Lopes-Pacheco M. · · 2016 · cited 108× · PMID 27656143 · DOI 10.3389/fphar.2016.00275
Nonsense suppression therapies in human genetic diseases.
Martins-Dias P, Romão L. · · 2021 · cited 70× · PMID 33751142 · DOI 10.1007/s00018-021-03809-7
Ataluren-Promising Therapeutic Premature Termination Codon Readthrough Frontrunner.
Michorowska S. · · 2021 · cited 35× · PMID 34451881 · DOI 10.3390/ph14080785
Gene-targeting pharmaceuticals for single-gene disorders.
Beaudet AL, Meng L. · · 2016 · cited 33× · PMID 26628634 · DOI 10.1093/hmg/ddv476
One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.
Ensinck MM, Carlon MS. · · 2022 · cited 24× · PMID 35740997 · DOI 10.3390/cells11121868
Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.
Bezzerri V, Api M, Allegri M, Fabrizzi B, et al · · 2020 · cited 8× · PMID 32630050 · DOI 10.3390/ijms21134672
Advancing Therapeutic Strategies for Nonsense-Related Diseases: From Small Molecules to Nucleic Acid-Based Innovations.
Ricci D, Cruciata I, Fiduccia I, Vitale E, et al · · 2025 · cited 7× · PMID 40420818 · DOI 10.1002/iub.70027

Verify or expand the search:

Other trials of Ataluren

Trials testing the same drug.

NCT04336826 — A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With N · Phase 2 · completed
NCT03796637 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have · Phase 2 · completed
NCT04117880 — A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia · Phase 2 · withdrawn
NCT03648827 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) · Phase 2 · completed
NCT03179631 — Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy · Phase 3 · completed

Other recruiting trials for Cystic Fibrosis

Currently open trials in the same condition.

NCT07437105 — Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants · Phase 1 · recruiting
NCT07283770 — Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants · Phase 1 · recruiting
NCT07274631 — A Cohort for Inflammatory Respiratory Diseases: From Phenotyping to Personalised Medicine · recruiting
NCT06810167 — Assessing Tenapanor as a Treatment of CF-related Constipation. · Phase 3 · recruiting
NCT06962852 — A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI · Phase 1, PHASE2 · active not recruiting

Other PTC Therapeutics trials

Trials by the same sponsor.

NCT06302348 — A Study of Sepiapterin in Participants With Phenylketonuria (PKU) · Phase 3 · recruiting
NCT05515536 — A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia · Phase 3 · active not recruiting
NCT05485987 — A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia · Phase 2 · completed
NCT05218655 — A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease · Phase 3 · completed
NCT05358717 — A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) · Phase 2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT00458341 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by PTC Therapeutics
Last refreshed: 6 March 2020

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00458341.

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