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

Glycomine

Status and phase: Invitation-only; Phase 2

Conditions: Pmm2-CDG; Phosphomannomutase 2 Deficiency

Treatments: Drug: GLM101

Study type: Interventional

Funder types: Industry

Identifiers

NCT06657859

GLM101-007

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 2 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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40 participants in 3 patient groups

10 mg/kg GLM101

Experimental group

Description:

GLM101 IV infusions, given weekly

Treatment:

Drug: GLM101

20 mg/kg GLM101

Experimental group

Description:

GLM101 IV infusions, given weekly

Treatment:

Drug: GLM101

30 mg/kg GLM101

Experimental group

Description:

GLM101 IV infusions, given weekly

Treatment:

Drug: GLM101