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Study of Ataluren (PTC124) in Cystic Fibrosis

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PTC Therapeutics

Status and phase

Terminated
Phase 3

Conditions

Cystic Fibrosis

Treatments

Drug: Ataluren

Study type

Interventional

Funder types

Industry

Identifiers

NCT02107859
PTC124-GD-023-CF
2013-005449-35 (EudraCT Number)

Details and patient eligibility

About

The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).

Enrollment

61 patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

  • Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
  • Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
  • Body weight greater than or equal to (≥) 16 kilograms (kg).
  • Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
  • Confirmed laboratory values within the central laboratory ranges at screening.
  • In male and female 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 60-day follow-up period.
  • Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions.

Key Exclusion Criteria:

  • Chronic use of systemic tobramycin within 4 weeks prior to screening.
  • Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
  • Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
  • Known hypersensitivity to any of the ingredients or excipients of the study drug.
  • Exposure to another investigational drug within 4 weeks prior to screening.
  • Treatment with intravenous antibiotics within 3 weeks prior to screening.
  • History of solid organ or hematological transplantation.
  • Ongoing immunosuppressive therapy (other than corticosteroids).
  • Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
  • Known portal hypertension.
  • Pregnancy or breast-feeding.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

61 participants in 1 patient group

Ataluren
Experimental group
Description:
Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
Treatment:
Drug: Ataluren

Trial documents
2

Trial contacts and locations

17

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Data sourced from clinicaltrials.gov

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