Study of Ataluren (PTC124™) in Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00803205
• Date of registration: 04/12/2008
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: Study of Ataluren (PTC124™) in Cystic Fibrosis
• Scientific title: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
• Date of first enrolment: September 8, 2009
• Target sample size: 238
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00803205
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Temitayo Ajayi, MD
• Affiliation: PTC Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Ability to provide written informed consent (parental/guardian consent and participant
assent if <18 years of age)

- Age =6 years

- Body weight =16 kg

- Abnormal nasal transepithelial potential difference (TEPD) total chloride conductance
(a less electrically negative value than -5 millivolts (mV) for total chloride
conductance [?chloride-free+isoproterenol])

- Sweat chloride >40 milliequivalents/liter (mEq/L)

- Documentation of the simultaneous presence of a nonsense mutation in at least 1 allele
of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by
gene sequencing from a laboratory certified by the College of American Pathologists
(CAP), under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an
equivalent organization

- Verification that a blood sample has been drawn for confirmation of the presence of a
nonsense mutation in the CFTR gene

- Ability to perform a valid, reproducible spirometry test using the study-specific
spirometer with demonstration of an FEV1 =40% and =90% of predicted for age, gender,
and height

- Resting oxygen saturation (as measured by pulse oximetry) =92% on room air

- Documentation by VivoMetrics that the participant has satisfactorily completed a
24-hour LifeShirt® cough frequency assessment

- Confirmed screening laboratory values within the central laboratory ranges (hepatic,
adrenal, renal, serum electrolytes, and reproduction [women only] parameters)

- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during the study
drug administration and 4-week follow-up period

- Willingness and ability to comply with scheduled visits, drug administration plan,
study restrictions, and study procedures

Exclusion Criteria:

- Known hypersensitivity to any of the ingredients or excipients of the study drug

- Any change (initiation, change in type of drug, dose modification, schedule
modification, interruption, discontinuation, or reinitiation) in a chronic
treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior
to start of study treatment

- Exposure to another investigational drug within 4 weeks prior to start of study
treatment

- Treatment with systemic aminoglycoside antibiotics at the time of the Baseline TEPD
assessment

- Treatment with intravenous antibiotics within 3 weeks prior to start of study
treatment

- History of solid organ or hematological transplantation

- Ongoing immunosuppressive therapy (other than corticosteroids)

- Ongoing warfarin, phenytoin, or tolbutamide therapy

- Ongoing participation in any other therapeutic clinical trial

- Major complications of lung disease (including massive hemoptysis, pneumothorax, or
pleural effusion) within 8 weeks prior to start of study treatment

- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection
(including viral illnesses) within 3 weeks prior to randomization

- Known portal hypertension

- Positive hepatitis B surface antigen, hepatitis C antibody test, or human
immunodeficiency virus (HIV) test

- Pregnancy or breast-feeding

- Current smoker or a smoking history of =10 pack-years (number of cigarette packs/day *
number of years smoked)

- Prior or ongoing medical condition (for example, concomitant illness, alcoholism, drug
abuse, psychiatric condition), medical history, physical findings, electrocardiography
findings, or laboratory abnormality that, in the Investigator's opinion, could
adversely affect the safety of the participant, makes it unlikely that the course of
treatment or follow-up would be completed, or could impair the assessment of study
results.



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: Ataluren

Primary Outcome(s)

Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline [Time Frame: Baseline (Week 1)]

Percentage Change From Baseline in Percent-Predicted of FEV1 at Week 48 [Time Frame: End of Treatment (EOT) (Week 48)]

Secondary Outcome(s)

Change From Baseline in Awake Cough Hourly Rate at Week 48 [Time Frame: Baseline, EOT (Week 48)]

Change From Baseline in the Respiratory Domain Score of the Revised Cystic Fibrosis Questionnaire (CFQ-R) at Week 48 [Time Frame: Baseline, EOT (Week 48)]

Percentage Change From Baseline in Percent-Predicted of FVC at Week 48 [Time Frame: EOT (Week 48)]

Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks [Time Frame: Baseline to EOT (Week 48)]

Percent-Predicted of Forced Vital Capacity (FVC) at Baseline [Time Frame: Baseline (Week 1)]

Secondary ID(s)

2008-003924-52

Orphan Product Grant #FD003715

PTC124-GD-009-CF

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation

Results

• Results available: Yes
• Date Posted: 14/05/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00803205