Status:
COMPLETED
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
Lead Sponsor:
PTC Therapeutics
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
6-18 years
Phase:
PHASE2
Brief Summary
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. A...
Detailed Description
In this study, participants with CF due to a nonsense mutation will be treated with a new investigational drug called ataluren. Evaluation procedures (history, physical examination, blood and urine te...
Eligibility Criteria
Inclusion
- Participants must meet all of the following conditions to be eligible for enrollment into the study:
- Diagnosis of CF based on conclusively abnormal sweat test (sweat chloride \>35 milliequivalents \[mEq\]/liter).
- Abnormal nasal epithelial TEPD total chloride conductance (a more electrically negative value than 5 mV for Δchloride-free+isoproterenol).
- Presence of a mutation in both alleles.
- Documentation that a blood sample has been drawn for reconfirmation of the presence of a nonsense mutation in the CFTR gene.
- Age ≥6 years.
- Body weight ≥25 kg.
- FEV1 ≥40% of predicted for age, gender, and height.
- Oxygen saturation ≥92% on room air.
- Willingness of male and female participants, if not surgically sterile, to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and follow-up periods.
- Negative pregnancy test (for females of childbearing potential).
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures (including TEPD measurements, clinical laboratory tests, pulmonary function tests, and PK sampling), and study restrictions.
- Ability to provide written informed consent and/or assent.
- Evidence of signed and dated informed consent document (by the participant or a legal guardian) indicating that the participant and/or the legal guardian has been informed of all pertinent aspects of the trial.
Exclusion
- The presence of any of the following conditions will exclude a participant from enrollment in the study:
- Prior exposure to ataluren.
- Prior or ongoing medical condition (for example, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG 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.
- Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before start of study treatment.
- History of major complications of lung disease (including recent massive hemoptysis or pneumothorax) within 2 months prior to start of study treatment.
- Abnormalities on screening chest x-ray suggesting clinically significant active pulmonary disease other than CF, or new, significant abnormalities such as atelectasis or pleural effusion which may be indicative of clinically significant active pulmonary involvement secondary to CF.
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test.
- Hemoglobin \<10 grams/deciliter (g/dL).
- Serum albumin \<2.5 g/dL.
- Abnormal liver function (serum total bilirubin \> the upper limit of normal, or serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) \>2.0 times the upper limit of normal).
- Abnormal renal function (serum creatinine \>1.5 times upper limit of normal).
- Pregnancy or breast-feeding.
- History of solid organ or hematological transplantation.
- Exposure to another investigational drug within 14 days prior to start of study treatment.
- Ongoing participation in any other therapeutic clinical trial.
- Ongoing use of thiazolidinedione peroxisome proliferator-activated receptor gamma (PPAR γ) agonists, for example, rosiglitazone (Avandia® or equivalent) or pioglitazone (Actos® or equivalent).
- Change in intranasal medications (including use of corticosteroids, cromolyn, ipratropium bromide, phenylephrine, or oxymetazoline) within 14 days prior to start of study treatment.
- Change in treatment with systemic or inhaled corticosteroids within 14 days prior to start of study treatment.
- Use of or requirement for inhaled gentamicin or amikacin within 14 days prior to start of study treatment or during study treatment.
- Requirement for systemic aminoglycoside antibiotics within 14 days prior to start of study treatment.
Key Trial Info
Start Date :
March 23 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 29 2008
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00458341
Start Date
March 23 2007
End Date
February 29 2008
Last Update
March 6 2020
Active Locations (3)
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1
Reine Fabiola Hospital
Brussels, Belgium, 1020
2
UZ Gasthuisberg Leuven
Leuven, Belgium, 3000
3
Hopital Necker Enfants Malades
Paris, France, 75015