CompletedPhase 1/2

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

United States6 participantsStarted 2022-03-10

Plain-language summary

This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of oral miglustat (100 mg once daily \[QD\] to 200 mg 3 times daily \[TID\]) in subjects ≥ 17 years of age with CLN3 disease over a period of 104 weeks.

Who can participate

Age range17 Years

SexALL

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Inclusion criteria

✓. Have provided informed consents (TCH and NIH) by subject or parent/legal guardian/legally authorized representative (as appropriate).
✓. Are males or females ≥ 17 years of age at the time of screening
✓. Have genetically confirmed diagnosis of syndromic CLN3 disease with
✓. Male and female participants must use a highly effective method of contraception and must continue for the duration of the trial (and for 30 days after the end of treatment).
✓. Are able to complete study assessments (subject or caregiver) and return to the clinic as scheduled

Exclusion criteria

✕. Have a medical condition that in the opinion of the PI would interfere with the safety assessments or increase the subject's risk of adverse events (AEs)
✕. Use of any therapy (approved, off-label, or unapproved) intended to modify the course of any neuronal ceroid lipofuscinosis disease, including but not limited to flupirtine or flupirtine derivatives, cerliponase alfa (Brineura)
✕. Have, in the opinion of the PI, a clinically significant abnormality in their clinical laboratory values (hematology, chemistry, or urinalysis) at screening that would preclude their participation in the study

What they're measuring

Number of Treatment-emergent Adverse Events.

Timeframe: 78 weeks

Trial details

NCT IDNCT05174039

SponsorBeyond Batten Disease Foundation

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-05-30

Results submitted2025-07-04

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