Ignite Creation Date:
2025-12-24 @ 2:33 PM
Ignite Modification Date:
2026-01-05 @ 7:55 AM
Study NCT ID:
NCT06657859
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-10-26
First Post:
2024-10-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535739', 'term': 'Congenital disorder of glycosylation type 1A'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'This is an open-label extension study. Participants will continue to receive the same dose level they received in the prior study.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2024-09-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2029-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-23', 'studyFirstSubmitDate': '2024-10-22', 'studyFirstSubmitQcDate': '2024-10-23', 'lastUpdatePostDateStruct': {'date': '2024-10-26', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-10-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluate long-term safety', 'timeFrame': 'From enrollment to end of treatment up to 4 years', 'description': 'Number of participants with treatment related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0'}], 'secondaryOutcomes': [{'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', 'description': '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.'}, {'measure': 'Maximum observed plasma concentration (Cmax)', 'timeFrame': 'From enrollment to end of treatment up to 4 years', 'description': 'Assessment of the pharmacokinetics (PK) of GLM101'}, {'measure': 'Time to maximum observed plasma concentration (Tmax)', 'timeFrame': 'From enrollment to end of treatment up to 4 years', 'description': 'Assessment pharmacokinetics (PK) of GLM101'}, {'measure': 'Area under the plasma concentration vs. time curve (AUC)', 'timeFrame': 'From enrollment to end of treatment up to 4 years', 'description': 'Assessment of the pharmacokinetics (PK) of GLM101'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['GLM101', 'Pmm2', 'CDG', 'CDG 1a'], 'conditions': ['Pmm2-CDG', 'Phosphomannomutase 2 Deficiency']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Willing and able to provide informed consent/assent and/or their legally authorized representative\n* Completed previous clinical study with GLM101\n* If female of childbearing potential, must not be pregnant and use a medically accepted method of contraception\n* If male and sexually active must agree to use a medically acceptable method of contraception\n* Willing ad able to adhere to study requirements described in the protocol\n\nExclusion Criteria:\n\n* Has a condition that would compromise safety or compliance of participant or preclude participant completion of study\n* Has positive serum pregnancy test\n* Unwilling or unable to comply with scheduled visits, study drug administration plan, laboratory tests, other study procedures, and study restrictions.'}, 'identificationModule': {'nctId': 'NCT06657859', 'briefTitle': 'Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Glycomine, Inc.'}, 'officialTitle': 'A Phase 2, Open-Label Extension Study to Assess the Safety and Efficacy of GLM101 Administered Intravenously to Participants With PMM2-CDG', 'orgStudyIdInfo': {'id': 'GLM101-007'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '10 mg/kg GLM101', 'description': 'GLM101 IV infusions, given weekly', 'interventionNames': ['Drug: GLM101']}, {'type': 'EXPERIMENTAL', 'label': '20 mg/kg GLM101', 'description': 'GLM101 IV infusions, given weekly', 'interventionNames': ['Drug: GLM101']}, {'type': 'EXPERIMENTAL', 'label': '30 mg/kg GLM101', 'description': 'GLM101 IV infusions, given weekly', 'interventionNames': ['Drug: GLM101']}], 'interventions': [{'name': 'GLM101', 'type': 'DRUG', 'description': 'GLM101 IV infusion', 'armGroupLabels': ['10 mg/kg GLM101', '20 mg/kg GLM101', '30 mg/kg GLM101']}]}, 'contactsLocationsModule': {'locations': [{'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}], 'overallOfficials': [{'name': 'Chief Medical Officer', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Glycomine, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Glycomine, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}