Glycomine, Inc.
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101. Participants will complete weekly infusions of GLM101 at the same dose level received in previous trials.
This is a phase 2 open-label clinical study of GLM101 in patients with PMM2-CDG who have previously participated in a study of GLM101. This study is designed to monitor long-term safety and treatment effect of GLM101 and provide continued access to study treatment. Participants will receive 30 mg/kg. Dose levels may be adjusted to lower doses or higher doses based on available data that demonstrates a change to be safe and favorable. Among other assessments, participants will be asked to complete questionnaires to evaluate changes in ataxia and quality of life.
Inclusion Criteria: 1. Is willing and able to provide informed consent/assent, directly or through a legally authorized representative. 2. Has successfully completed the Treatment Period with GLM101 in a previous clinical study. 3. At least 2 years of age, at the time of signing the informed consent form (ICF). 4. Molecularly confirmed 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 bi-allelic variants AND phosphomannomutase-2 (PMM2) enzyme activity consistent with a…
GLM101 IV infusion