Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients

Phase 2

• Conditions: Pmm2-CDG; Phosphomannomutase 2 Deficiency
• Interventions: Drug: GLM101

• Registration Number: NCT06657859
• Lead Sponsor: Glycomine, Inc.

Brief 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. It will also learn about the long term safety of GLM101. Participants will complete weekly infusions of GLM101 at the same dose level received in previous trials.

Detailed Description

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 provided continued access to study treatment. Participants will continue weekly infusions of GLM101 at the same dose level they received in the previous study. Dose levels may be adjusted to lower doses or higher doses based on available data that demonstrates a change to be safe and tolerable. Participants will be asked to complete questionnaires to evaluate changes in ataxia and quality of life.

Study Timeline

• Study Start: 2024-09-30
• Status Verified: 2024-10
• Study First Submitted: 2024-10-22
• Study First Submitted QC: 2024-10-23
• Last Update Submitted: 2024-10-23
• Study First Posted: 2024-10-26
• Last Update Posted: 2024-10-26
• Primary Completion: 2029-09
• Study Completion: 2029-10

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Willing and able to provide informed consent/assent and/or their legally authorized representative
• Completed previous clinical study with GLM101
• If female of childbearing potential, must not be pregnant and use a medically accepted method of contraception
• If male and sexually active must agree to use a medically acceptable method of contraception
• Willing ad able to adhere to study requirements described in the protocol

Exclusion Criteria

• Has a condition that would compromise safety or compliance of participant or preclude participant completion of study
• Has positive serum pregnancy test
• Unwilling or unable to comply with scheduled visits, study drug administration plan, laboratory tests, other study procedures, and study restrictions.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
10 mg/kg GLM101	GLM101	GLM101 IV infusions, given weekly
20 mg/kg GLM101	GLM101	GLM101 IV infusions, given weekly
30 mg/kg GLM101	GLM101	GLM101 IV infusions, given weekly

Primary Outcome Measures

Name	Time	Method
Evaluate long-term safety	From enrollment to end of treatment up to 4 years	Number of participants with treatment related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Secondary Outcome Measures

Name	Time	Method
Evaluate changes in ataxia using International Cooperative Ataxia Rating Scale (ICARS)	From enrollment, at 3 months, 6 months and annually to end of treatment up to 4 years	The scale is scored out of 100 with 19 items and 4 subscales of postural and gait disturbances, limb ataxia, dysarthria, and oculomotor disorders. Higher scores indicate higher levels of impairment.
Maximum observed plasma concentration (Cmax)	From enrollment to end of treatment up to 4 years	Assessment of the pharmacokinetics (PK) of GLM101
Time to maximum observed plasma concentration (Tmax)	From enrollment to end of treatment up to 4 years	Assessment pharmacokinetics (PK) of GLM101
Area under the plasma concentration vs. time curve (AUC)	From enrollment to end of treatment up to 4 years	Assessment of the pharmacokinetics (PK) of GLM101

Trial Locations

Locations (1)

Hospital Sant Joan de Déu; 🇪🇸 Barcelona, Spain