A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG (NCT06892288) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
United States, Belgium50 participantsStarted 2025-07-09
Plain-language summary
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults. Researchers will compare participants receiving GLM101 to those receiving a placebo to see if GLM101 improves symptoms of PMM2-CDG.
The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B).
Who can participate
Age range4 Years
SexALL
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Inclusion criteria: Participant is eligible for participation in the study if all of the following apply:
* Participant is aged ≥ 4 years old at the time of signing the consent.
* Participant with molecular diagnosis of PMM2-CDG. Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of biallelic variants and PMM2 enzyme activity consistent with a diagnosis of PMM2-CDG. Diagnosis with laboratory report(s) on file is required.
* Participant is willing and capable of completing the ICARS in its entirety without any assessment deemed as "not evaluable".
* Participant screening total ICARS score is ≥ 20 and ≤ 80 .
* Male or female participant has appropriate measures in place to prevent pregnancy:
* If the participant is a woman of childbearing potential, i.e., fertile, following menarche and until becoming postmenopausal unless permanently sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy), she must not be pregnant (confirmed by a negative serum pregnancy test), is using a medically accepted method of contraception (abstinence, a hormonal contraceptive associated with inhibition of ovulation in conjunction with a barrier method, or use of an intrauterine device), and must agree to continue using this method for 50 days after the last infusion. Note: sexual abstinence is considered a highly effective method only if def…
What they're measuring
1
Evaluation of Ataxia Changes Using the International Cooperative Ataxia Rating Scale (ICARS) in PMM2-CDG Patients at 24 weeks