Topic: Medicines for Children Research Network
Conditions
Interventions
Infusion of Lamazym 1 mg/kg given weekly for 6 months
Sponsors
Royal Manchester Children's Hospital (UK)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The subject must have participated in the phase 1 trial (EudraCT number: 201002208436) and phase 2a trial (EudraCT number: 201002208526) 2. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial related activities (trial related activities are any procedures that would not have been performed during normal management of the subject) 3. The subject and his/her guardian(s) must have the ability to comply with the protocol
Exclusion criteria
Exclusion criteria: 1. The subject cannot walk without support 2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alphaMannosidosis 3. History of bone marrow transplantation 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. Pregnancy 7. Psychosis within the last 3 months 8. Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial 9. Participation in other interventional trials testing IMP except for studies with Lamazym
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Reduction of Oligosaccharides in blood serum and cerebrospinal fluid (CSF) and an improvement in the 3 minute stair climb | — |
Secondary
| Measure | Time frame |
|---|---|
| No secondary outcome measures | — |
Countries
Belgium, Denmark, France, Germany, Poland, Spain, United Kingdom
Outcome results
None listed