Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients (NCT06657859) | Clinical Trial Compass
By InvitationPhase 2
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
Spain, United Kingdom90 participantsStarted 2024-09-30
Plain-language summary
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.
Who can participate
Age range2 Years
SexALL
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Inclusion criteria
✓. Is willing and able to provide informed consent/assent, directly or through a legally authorized representative.
✓. Has successfully completed the Treatment Period with GLM101 in a previous clinical study.
✓. At least 2 years of age, at the time of signing the informed consent form (ICF).
✓. 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 diagnosis of PMM2-CDG. Historical diagnosis including from a prior parent trial is permitted;
✓. Male or female participant has appropriate measures in place to prevent pregnancy:
✓. If the participant is male, he must agree to refrain from donating sperm during the study and 50 days after the last infusion of GLM101.
✓. Is willing and able to comply with this protocol.
Exclusion criteria
✕. Has any other condition that would, in the opinion of the Investigator, potentially compromise the safety or compliance of the participant or preclude the participant's successful completion of the study.
✕. Diagnosis of congenital disorder of glycosylation (CDG) other than PMM2; 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 the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG.
What they're measuring
1
Evaluate long-term safety
Timeframe: From enrollment to end of treatment up to 4 years
✕. If not enrolling directly from a parent study (i.e., more than 28 days from Final Treatment visit in a parent study to date of consent), has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
✕. ALT or AST \>3× ULN OR total bilirubin \>2× ULN or INR \>1.5 (if no anti-coagulation treatment) or INR \> 4 (if participant on anti-coagulation treatment) considered clinically significant;
✕. Has a history of liver transplant;
✕. Has a history of drug or alcohol use disorder within the 12 months prior to Screening;
✕. If not enrolling directly from a parent study (i.e., if more than 28 days from Final Treatment visit in a parent study to date of consent), has had a major surgical procedure within 30 days prior to Screening;
✕. Has laboratory value(s) outside the laboratory reference range considered clinically significant and not related to PMM2-CDG;