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ataluren

Trade name: Translarna

Small moleculeexperimental

Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics. — Wikipedia

Clinical trial activity

33 trials · 14 clinical orgs · 0 marketing orgs

Phase 1
3
Phase 2
27
Phase 3
12
Phase 4
2

Earliest trial started Sep 1, 2005 (NCT00234663)

Timeline

2000s

  1. Jan 1, 2005

    Earliest Phase 2 Sponsor(trial)

  2. Jan 1, 2009

    Earliest Phase 3 Sponsor(trial)

2010s

  1. Jan 1, 2015

    Earliest Phase 1 Sponsor(trial)

Indications

Approved for

Studied for

Mechanism of action

Chemistry & pharmacology

Loading structure…

SMILES

O=C(O)c1cccc(-c2noc(-c3ccccc3F)n2)c1
Mol. weight
284.25 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Achiral Molecule
Inorganic
No
Polymer
No

Oral

No

Parenteral

No

Topical

No

Sources

Also known as

  • 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
  • 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ataluren
  • ec-000.2051
  • ec-000.2051
  • ptc124
  • ptc124
  • ptc124
  • ptc124
  • ptc124
  • ptc 124
  • ptc-124
  • translarna

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT06900049Early Phase 1 (Phase 1)Oct 24, 2024Jiao Tong University
NCT04336826Phase 2Jun 1, 2020PTC Therapeutics
NCT04014530Phase 1/Phase 2 (Phase 2)Aug 1, 2019Merck, PTC Therapeutics, University of Amsterdam
NCT03796637Phase 2Apr 11, 2019PTC Therapeutics
NCT04117880Phase 2Dec 31, 2018PTC Therapeutics
NCT03648827Phase 2Nov 30, 2018PTC Therapeutics
NCT03179631Phase 3Jun 1, 2017PTC Therapeutics
NCT03256799Phase 4Mar 17, 2017University of Alabama, Birmingham
NCT03256968Phase 4Jan 27, 2017University of Alabama, Birmingham
NCT02758626Phase 2Nov 1, 2016New York University, PTC Therapeutics
NCT02819557Phase 2Jun 1, 2016PTC Therapeutics
NCT02647359Phase 2Jan 1, 2016PTC Therapeutics
NCT02456103Phase 3Jul 1, 2015PTC Therapeutics
NCT02409004Phase 1Mar 1, 2015PTC Therapeutics
NCT02139306Phase 3Jun 1, 2014Cystic Fibrosis Foundation Therapeutics, European Cystic Fibrosis Society-Clinical Trial Network, PTC Therapeutics
NCT02107859Phase 3May 1, 2014PTC Therapeutics
NCT02090959Phase 3Mar 1, 2014PTC Therapeutics
NCT01826487Phase 3Mar 1, 2013PTC Therapeutics
NCT01557400Phase 3May 1, 2012PTC Therapeutics
NCT01247207Phase 3Nov 1, 2010PTC Therapeutics
Showing 20 of 33 trials
Page 1 / 2

Organizations

Research & Development (14)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
PTC TherapeuticsFor profit302832005
GenzymeFor profit5022008
Cystic Fibrosis Foundation TherapeuticsAcademic/Hospital4022005
University of Alabama, BirminghamAcademic/Hospital3222005
Hebrew UniversityAcademic/Hospital2012005
European Cystic Fibrosis Society-Clinical Trial NetworkAcademic/Hospital1012014
Food and Drug AdministrationGovernment1012005
Jiao Tong UniversityAcademic/Hospital1112024
MerckFor profit1012019
Muscular Dystrophy AssociationAcademic/Hospital1012005
New York UniversityAcademic/Hospital1112016
University of AmsterdamAcademic/Hospital1112019
University of ParisAcademic/Hospital1012007
University of PennsylvaniaAcademic/Hospital1012005
14 organizations
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