A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

Zymenex

Status and phase

Completed

Phase 3

Conditions

Alpha-Mannosidosis

Treatments

Drug: Lamazym

Drug: Placebo

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01681953

rhLAMAN-05

2012-000979-17 (EudraCT Number)

Details and patient eligibility

About

The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis

Enrollment

25 patients

Sex

All

Ages

5 to 35 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
The subject and his/her guardian(s) must have the ability to comply with the protocol
The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-Mannosidase activity < 10% of normal activity (historical data)
The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
The subject must have the ability to physically and mentally cooperate in the tests
The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial

Exclusion criteria

The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity < 10% of normal activity
The subject cannot walk without support
Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
History of BMT
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
Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
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
Psychosis; any psychotic disease, also in remission, is an exclusion criteria
Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
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
Total IgE >800 IU/ml
Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)