Clinical trial NCT06657859: Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
Official title: A Phase 2, Open-Label Extension Study to Assess the Safety and Efficacy of GLM101 Administered Intravenously to Participants With PMM2-CDG
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: Pmm2-CDG
Phosphomannomutase 2 Deficiency
Keywords: GLM101
Pmm2
CDG
CDG 1a
Lead sponsor: Glycomine, Inc.
Sponsor class: INDUSTRY
First posted: 2024-10-26
Enrollment: 40
Sex: ALL
Minimum age (years): 2
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is an open-label extension study. Participants will continue to receive the same dose level they received in the prior study.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluate long-term safety
timeFrame: From enrollment to end of treatment up to 4 years
Secondary outcomes:
measure: Evaluate changes in ataxia using International Cooperative Ataxia Rating Scale (ICARS)
timeFrame: From enrollment, at 3 months, 6 months and annually to end of treatment up to 4 years
measure: Maximum observed plasma concentration (Cmax)
timeFrame: From enrollment to end of treatment up to 4 years
measure: Time to maximum observed plasma concentration (Tmax)
timeFrame: From enrollment to end of treatment up to 4 years
measure: Area under the plasma concentration vs. time curve (AUC)
timeFrame: From enrollment to end of treatment up to 4 years
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu
Study officials:
affiliation: Glycomine, Inc.
name: Chief Medical Officer
role: STUDY_DIRECTOR
References: