Avrupa, İsrail, Avustralya ve Kanada'da Daha Önce Tedavi Görmüş Anlamsız Mutasyonlu Duchenne/Becker Musküler Distrofili (nmDBMD) Katılımcılarda Ataluren Çalışması

Sade dilde özet

Bu çalışma, DMD geninde 'anlamsız' (nonsense) mutasyon taşıyan, daha önce tedavi görmüş Duchenne/Becker Musküler Distrofi (Duchenne/Becker muscular dystrophy) hastalarında Ataluren ilacını inceliyor. Bu tanıya sahip, uygun katılımcılar dahil edilmiştir. Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 3 (Phase 3)

Durum

Tamamlandı

Sponsor

PTC Therapeutics

Müdahale

Ataluren

Başlangıç

20.05.2012

Tahmini bitiş

19.01.2018

Ülkeler

Australia, Belgium, Canada, France, Germany, Israel, Italy, Spain, Sweden, United Kingdom

İlgili genler

Merkezler

• Parkville, Australia
• Westmead, Australia
• Leuven, Belgium
• Calgary, Canada
• Vancouver, Canada
• London, Canada
• Marseille, France
• Nantes, France
• Paris, France
• Essen, Germany
• Freiburg im Breisgau, Germany
• Jerusalem, Israel
• Milan, Italy
• Rome, Italy
• Rome, Italy
• Barcelona, Spain
• Valencia, Spain
• Gothenburg, Sweden
• Stockholm, Sweden
• London, United Kingdom
• Newcastle upon Tyne, United Kingdom

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

1. Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethics Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
2. History of exposure to ataluren in a prior PTC study in nmDBMD. Note: Participants are considered eligible only if they received ataluren during their participation in 1 or more prior PTC-sponsored studies of ataluren in nmDBMD. Note: Participants who have participated in a prior or ongoing PTC study with ataluren in nmDBMD at a trial site in the US or Canada, but reside outside of the US and Canada, may be eligible for this study (with the approval of the PTC Therapeutics Medical Monitor).
3. Male sex.
4. In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 6-week follow-up period.
5. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.

Exclusion Criteria:

1. Exposure to another investigational drug within 1 month prior to start of study treatment.
2. Eligibility for another ataluren clinical trial that is actively enrolling study participants.
3. Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse® UltraTM \[refined polydextrose\], polyethylene glycol 3350, Lutrol® micro F127 \[poloxamer 407\], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P \[colloidal silica\], magnesium stearate).
4. Ongoing use of the following medications:

   1. Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
   2. Systemic aminoglycoside therapy
5. Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant or make it unlikely that follow-up would be completed.