Ignite Creation Date:
2025-12-24 @ 7:43 PM
Ignite Modification Date:
2025-12-25 @ 5:21 PM
Study NCT ID:
NCT06184503
Status:
RECRUITING
Last Update Posted:
2025-05-16
First Post:
2023-12-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008363', 'term': 'alpha-Mannosidosis'}], 'ancestors': [{'id': 'D044904', 'term': 'Mannosidase Deficiency Diseases'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 5}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-02-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2029-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-15', 'studyFirstSubmitDate': '2023-12-14', 'studyFirstSubmitQcDate': '2023-12-14', 'lastUpdatePostDateStruct': {'date': '2025-05-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-12-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Development of anti drug antibody to velmanase alfa', 'timeFrame': '52 weeks of treatment', 'description': 'Assessment of the impact of anti drug antibody on pharmacodinamic'}, {'measure': 'Treatment-emergent adverse events', 'timeFrame': '52 weeks', 'description': 'Number of undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment'}], 'primaryOutcomes': [{'measure': 'Pharmacodynamic Response to velmanase alfa', 'timeFrame': '52 weeks of treatment', 'description': 'Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Alpha-Mannosidosis']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.chiesiclinicalstudies.com/trials/CLI-LMZYMAA2-04', 'label': 'Chiesi site web study information'}]}, 'descriptionModule': {'briefSummary': "The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.\n\nThe main questions it aims to answer are:\n\n* study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy\n* explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.\n\nAdditional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '3 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Paediatric patients with a confirmed diagnosis of alpha mannosidosis via MAN2B1 genetic testing and/or alpha-mannosidase activity in leukocytes or fibroblasts \\< 10% of normal activity, with data for at least one pre- and one post-Lamzede treatment sample obtained when \\< 3 YOA, after at least 6 weeks of treatment (primary analyses).', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects with the provision of informed consent from their legal guardians (LAR)\n* Have a confirmed diagnosis of alpha mannosidosis\n* Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age\n* Have information on the disease marker GlcNAc(Man)2 obtained:\n\nbefore velmanase treatment initiation (ideally max 6 month before), and at least one post-treatment sample, collected following at least six weeks of treatment.\n\n\\- Participants treated with Lamzede, 1 mg/kg body weight, via weekly intravenous infusions.\n\nExclusion Criteria:\n\nParticipants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).'}, 'identificationModule': {'nctId': 'NCT06184503', 'acronym': 'LAMPO(0-3)', 'briefTitle': "Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3", 'organization': {'class': 'INDUSTRY', 'fullName': 'Chiesi Farmaceutici S.p.A.'}, 'officialTitle': 'A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age', 'orgStudyIdInfo': {'id': 'CLI-LMZYMAA2-04'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age', 'description': 'Paediatric patients with a confirmed diagnosis of alpha-mannosidosis with data for at least one pre- and one post-Lamzede treatment sample obtained when \\< 3 YOA.', 'interventionNames': ['Drug: Velmanase Alfa']}], 'interventions': [{'name': 'Velmanase Alfa', 'type': 'DRUG', 'otherNames': ['Lamzede'], 'description': 'Lamzede® (velmanase alfa, henceforth referred to as Lamzede) is a recombinant human lysosomal alpha-mannosidase product developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-mannosidosis.', 'armGroupLabels': ['Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Parul Jayakar', 'role': 'CONTACT', 'email': 'Parul.Jayakar@Nicklaushealth.org', 'phone': '786-624-4717'}], 'facility': "Nicklaus Children's Hospital", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '29605', 'city': 'Greenwood', 'state': 'South Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Laura Buch', 'role': 'CONTACT', 'email': 'lbuch@ggc.org', 'phone': '864-250-7944'}], 'facility': 'Greenwood Genetic Center', 'geoPoint': {'lat': 34.1954, 'lon': -82.16179}}], 'centralContacts': [{'name': 'Chiesi Clinical trials', 'role': 'CONTACT', 'email': 'clinicaltrials_info@chiesi.com', 'phone': '+39.0521 2791'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Chiesi Farmaceutici S.p.A.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}