Astellas voluntarily halts patients dosing in AT132 trial in X-linked Myotubular Myopathy

Sep. 01, 2021 3:25 AM ETAstellas Pharma Inc. (ALPMF)ALPMYBy: Mamta Mayani, SA News Editor
  • Astellas Pharma (OTCPK:ALPMF) has voluntarily paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy (XLMTM).
  • This decision follows the reporting of a recent serious adverse event (SAE) in a study participant due to abnormal liver function tests observed in the weeks following dosing of the AT132 investigational gene therapy product at a lower dose (1.3x1014 vg/kg).
  • The company voluntarily halted screening and dosing, reported the SAE to regulatory agencies, and is engaged in dialogue with regulators.
  • At this time, an IND clinical hold has not been issued.
  • The participant has a history of intermittent cholestasis. However, prior to dosing, liver ultrasound was normal; and the participant's LFTs, reflecting normal bilirubin levels, were within eligibility criteria.
  • In December of 2020 the clinical hold was lifted after the FDA reviewed the modifications to the ASPIRO trial protocol, which included a reduction of dosing to the 1.3x1014 vg/kg dose level. The participant associated with this current SAE was dosed in the summer of 2021, after the original clinical hold was lifted.
  • XLMTM is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure and early death.

Recommended For You

Comments

To ensure this doesn’t happen in the future, please enable Javascript and cookies in your browser.
Is this happening to you frequently? Please report it on our feedback forum.
If you have an ad-blocker enabled you may be blocked from proceeding. Please disable your ad-blocker and refresh.