ong-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis
- Conditions
- Topic: Medicines for Children Research NetworkSubtopic: All DiagnosesDisease: All DiseasesNutritional, Metabolic, EndocrineDefects in glycoprotein degradation
- Registration Number
- ISRCTN96077718
- Lead Sponsor
- Royal Manchester Children's Hospital (UK)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 10
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method