A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis
- Conditions
- Treatment of Alpha-MannosidosisTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2012-000979-17-ES
- Lead Sponsor
- Zymenex A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 25
1. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
2. The subject and his/her guardian(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 < 10% of normal activity (historical data)
4. The subject must have an age at the time of screening ? 5 years and ? 35 years
5. The subject must have the ability to physically and mentally cooperate in the tests
6. The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
Are the trial subjects under 18? yes
Number of subjects for this age range: 13
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. The subjects diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity
2. The subject cannot walk without support
3. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
4. History of BMT
5. 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
6. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
7. Pregnancy: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
8. Psychosis any psychotic disease, also in remission, is an exclusion criteria
9. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
10. Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
11. Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
12. Total IgE >800 IU/ml
13. Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method