ATX-MTM-001 - Recensus Retrospective Study for Patients with XLMTM

• Research type

  Research Study

• Full title

  The RECENSUS Study: A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM), sponsored by Audentes Therapeutics

• IRAS ID

  163336

• Contact name

  Tristen Moors

• Contact email

  tmoors@audentestx.com

• Eudract number

  2014-003766-24

• Clinicaltrials.gov Identifier

  NCT02231697

• Duration of Study in the UK

  0 years, 3 months, 28 days

• Research summary

  This is a retrospective medical record review of approximately 50 deceased and 20 living patients with X-Linked Myotubular Myopathy (XLMTM). The objectives include 1) to characterize aspects of the disease and recorded medical management of XLMTM, 2) to identify potential prognostic variables of the disease, 3) to identify potential outcome measures for therapeutic intervention studies, and 4) to identify features of the disease that warrant particular monitoring during therapeutic intervention.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  15/LO/1144

• Date of REC Opinion

  13 Jul 2015

• REC opinion

  Favourable Opinion