Ataluren for Nonsense Mutation Methylmalonic Acidemia
A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia
Sponsor: Genzyme, a Sanofi Company
A PHASE2 clinical study on Amino Acid Metabolism, Inborn Errors, this trial is terminated or withdrawn. The trial is conducted by Genzyme, a Sanofi Company and has accumulated 13 data snapshots since 2010. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.
Change History
13 versions recorded-
Sep 2025 — Present [monthly]
Terminated PHASE2
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Sep 2024 — Sep 2025 [monthly]
Terminated PHASE2
-
Jul 2024 — Sep 2024 [monthly]
Terminated PHASE2
-
Jan 2023 — Jul 2024 [monthly]
Terminated PHASE2
-
Dec 2022 — Jan 2023 [monthly]
Terminated PHASE2
▶ Show 8 earlier versions
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Dec 2021 — Dec 2022 [monthly]
Terminated PHASE2
-
Jan 2021 — Dec 2021 [monthly]
Terminated PHASE2
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Nov 2020 — Jan 2021 [monthly]
Terminated PHASE2
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Aug 2020 — Nov 2020 [monthly]
Terminated PHASE2
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Jun 2018 — Aug 2020 [monthly]
Terminated PHASE2
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Feb 2018 — Jun 2018 [monthly]
Terminated PHASE2
Status: Suspended → Terminated
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Feb 2017 — Feb 2018 [monthly]
Suspended PHASE2
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Jan 2017 — Feb 2017 [monthly]
Suspended PHASE2
First recorded
Jul 2010
Trial started
Per CT.gov start date — pre-dates our first snapshot
Eligibility Summary
No eligibility information available.
Contact Information
- Genzyme, a Sanofi Company
- PTC Therapeutics
For direct contact, visit the study record on ClinicalTrials.gov .
Study Locations
- • Antwerp, Belgium
- • Düsseldorf, Germany
- • London, United Kingdom
- • Lyon, France
- • Milan, Italy
- • Naples, Italy
- • Padua, Italy
- • Paris, France
- • Zurich, Switzerland