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An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Miglustat for the Treatment of CLN3 Disease in Patients 17 Years of Age and Older

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Trial Profile

An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Miglustat for the Treatment of CLN3 Disease in Patients 17 Years of Age and Older

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 03 May 2024

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At a glance

  • Drugs BBDF 101 (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus Adverse reactions

    • Most Recent Events

    • 03 May 2024 Last checked against ClinicalTrials.gov: US National Institutes of Health record.
    • 25 Apr 2024 Planned End Date changed from 1 Oct 2024 to 15 Aug 2024.
    • 25 Apr 2024 Planned primary completion date changed from 1 Sep 2024 to 15 May 2024.

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