Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00007251

Trial Description

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Title

An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124®)

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

This is a Phase 3, multicenter, open-label, safety and efficacy study of ataluren in
patients with nonsense mutation cystic fibrosis who participated in the previous Phase 3
study with ataluren (PTC124-GD-009-CF; Study 009).

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Brief Summary in Scientific Language

This is a Phase 3, multicenter, open-label, safety and efficacy study of ataluren in
patients with nonsense mutation cystic fibrosis who participated in the previous Phase 3
study with ataluren (PTC124-GD-009-CF; Study 009). The primary objective of this study is to
determine the long-term safety and tolerability of ataluren in patients with nmCF, 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 FEV1 and pulmonary
exacerbation rate, and change from baseline in other safety parameters (eg, 12-lead ECG
measurements, vital signs). Study subjects will be enrolled at approximately 19 sites in the
United States, Europe and Israel that previously participated in Study PTC124-GD-009-CF.
All study subjects will receive 10-, 10-, and 20-mg/kg ataluren (PTC124®) 3 times per day
(morning, midday and evening, respectively) for 48 weeks. Subjects will be evaluated at
clinic visits every 16 weeks and supplemented with interim phone calls in between onsite
visits. A final 4-week post-treatment follow-up visit will also be conducted.

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Organizational Data

  •   DRKS00007251
  •   2015/04/22
  •   2014/04/04
  •   no
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Secondary IDs

  •   2013-005449-35 
  •   NCT02107859  (ClinicalTrials.gov)
  •   PTC124-GD-023-CF  (PTC Therapeutics)
  •   2013-005449-35 
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Health Condition or Problem studied

  •   Cystic Fibrosis
  •   E84 -  Cystic fibrosis
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Interventions/Observational Groups

  •   Drug: Ataluren (PTC124®)
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Characteristics

  •   Interventional
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  •   Single arm study
  •   Open (masking not used)
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  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   III
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Primary Outcome

- Adverse events and laboratory abnormalities; time frame: 48 weeks

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Secondary Outcome

- Forced expiratory volume in 1 second (FEV1); time frame: 48 weeks
- Pulmonary exacerbation rate; time frame: 48 weeks
- 12 Lead ECG; time frame: 48 Weeks
- Vital Signs; time frame: 48 Weeks
- Physical Examination; time frame: 48 Weeks

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Countries of Recruitment

  •   United States
  •   Belgium
  •   France
  •   Germany
  •   Israel
  •   Italy
  •   Spain
  •   Sweden
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Locations of Recruitment

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Recruitment

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  •   2014/05/31
  •   80
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   6   Years
  •   no maximum age
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Additional Inclusion Criteria

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

- Evidence of completed participation in the double-blind study, PTC124-GD-009-CF
(Study 009)

- Performance of a valid, reproducible spirometry test using the study-specific
spirometer during the screening period.

- Confirmed screening laboratory values within the specified central laboratory ranges
(hepatic, immune, adrenal, renal, serum electrolytes, and reproductive parameters)

- In male and female subjects 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

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Exclusion Criteria

- Treatment with inhaled aminoglycoside antibiotics within 4 months prior to screening

- 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.

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

- Exposure to another investigational drug within 4 weeks prior to screening or ongoing
participation in any other therapeutic clinical trial

- 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 HIV test

- Known portal hypertension

- Pregnancy or breast-feeding

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Addresses

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    • PTC Therapeutics
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    • PTC Therapeutics, Inc.
    • Temitayo Ajayi, MD 
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    • PTC Therapeutics, Inc.
    • Temitayo Ajayi, MD 
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Sources of Monetary or Material Support

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    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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    •   [---]*
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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .
  •   1
  •   2014/11/05
* This entry means the parameter is not applicable or has not been set.