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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-30

Study Completion Date

2029-10-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Pmm2-CDG Phosphomannomutase 2 Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

This is an open-label extension study. Participants will continue to receive the same dose level they received in the prior study.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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10 mg/kg GLM101

GLM101 IV infusions, given weekly

Group Type EXPERIMENTAL

GLM101

Intervention Type DRUG

GLM101 IV infusion

20 mg/kg GLM101

GLM101 IV infusions, given weekly

Group Type EXPERIMENTAL

GLM101

Intervention Type DRUG

GLM101 IV infusion

30 mg/kg GLM101

GLM101 IV infusions, given weekly

Group Type EXPERIMENTAL

GLM101

Intervention Type DRUG

GLM101 IV infusion

Interventions

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GLM101

GLM101 IV infusion

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No