Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
NCT ID: NCT02998879
Last Updated: 2021-10-26
Study Results
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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COMPLETED
PHASE2
5 participants
INTERVENTIONAL
2016-12-31
2020-07-31
Brief Summary
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Detailed Description
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* Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis)
* Detection of anti-velmanase alfa antibodies and neutralizing/inhibitory antibodies
The Secondary endpoints include changes from baseline to 24 months for the following parameters. Efficacy outcomes:
* Serum oligosaccharides
* Functional capacity: Peabody Developmental Motor Scale - 2nd edition (PDMS-2) scores, Mullen's Scale of Early Learning (MSEL) scores, Bruininks-Oseretsky Test Of Motor Proficiency-2nd Edition (BOT-2), when applicable by age (from 4 years) or upon the judgment of the physician
* Endurance: 3-Minute Stair Climb Test (3MSCT) and 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician, 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age, or when applicable according to the judgment of the physician
* Hearing evaluation: Otoacoustic Emissions (OAE) testing, Automatic Auditory Brainstem Response (A-ABR) audiometry
* Immunological profile, when applicable upon the judgment of the physician:
* CSF biomarkers: Tau protein (Tau), Neurofilament Protein Light (NFL), Glial Fibrillary Acidic Protein (GFAp), Oligosaccharides
* Assessment of quality of life via Questionnaire to parents
* Assessment of mannose-rich oligosaccharides in brain tissue, MRI
* Pharmacokinetic parameters
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Velmanase Alfa
velmanase alfa 1mg/kg body weight infusion
Velmanase Alfa (e.g. Lamazym)
iv infusion treatment
Interventions
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Velmanase Alfa (e.g. Lamazym)
iv infusion treatment
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. The subject's custodial parent(s) must have the ability to comply with the protocol
3. The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity in leukocytes or fibroblasts \< 10% of normal activity (historical data)
4. The subject must have an age at the time of screening \< 6 years.
Exclusion Criteria
2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
3. History of BMT
4. Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
5. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
6. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
7. Participation in other interventional trials testing the IMP within the last 3 months.
6 Years
ALL
No
Sponsors
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Cromsource
INDUSTRY
Chiesi Farmaceutici S.p.A.
INDUSTRY
Responsible Party
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Locations
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Vienna, , Austria
Copenhagen, , Denmark
Lyon, , France
Hamburg, , Germany
Mainz, , Germany
Trieste, , Italy
Countries
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Related Links
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Study Record on EU Clinical Trials Register including results
Other Identifiers
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2016-001988-36
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CCD-LMZYMAA1-08
Identifier Type: -
Identifier Source: org_study_id