Status:
COMPLETED
Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
Lead Sponsor:
PTC Therapeutics
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
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 to determine if a participant qualifies for the st...
Eligibility Criteria
Inclusion
- Diagnosis of CF based on documented evidence of a conclusively abnormal sweat test (sweat chloride \>60 milliequivalents/litre \[mEq/liter\]).
- Abnormal chloride secretion as measured by TEPD (a less than -5 mV TEPD assessment of chloride secretion with chloride-free amiloride and isoproterenol).
- Presence of a nonsense mutation in one of the alleles of the CFTR gene.
- Age ≥18 years.
- Body weight ≥40 kg.
- Forced expiratory volume in 1 second (FEV1) ≥40% of predicted for age, gender, and height (Knudson standards).
- Oxygen saturation (as measured by pulse oximetry) ≥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, and study restrictions.
- Ability to provide written informed consent.
Exclusion
- Prior or ongoing medical 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 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 that 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 per deciliter (g/dL).
- Serum albumin \<2.5 g/dL.
- Abnormal liver function (serum alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], gamma-glutamyl transferase \[GGT\], alkaline phosphatase, lactate dehydrogenase \[LDH\], or total bilirubin \> 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, eg, 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 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 :
November 30 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 31 2006
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT00237380
Start Date
November 30 2005
End Date
May 31 2006
Last Update
June 11 2020
Active Locations (1)
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1
Hadassah University Hospital - Mount Scopus
Jerusalem, Israel, 91240