Overview

This trial is active, not recruiting.

Condition alpha mannosidosis
Treatment lamazym
Phase phase 2
Sponsor Zymenex A/S
Collaborator European Commission
Start date January 2011
End date January 2012
Trial size 10 participants
Trial identifier NCT01285700, 2010-022085-26, rhLAMAN-03

Summary

This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Active Comparator)
25 U/kg
lamazym rhLAMAN
ERT, infusion weekly
(Active Comparator)
50 U/kg
lamazym rhLAMAN
ERT, infusion weekly

Primary Outcomes

Measure
Reduction of Oligosaccharides in urine
time frame: 3 months (interim evaluation) + 6 months

Secondary Outcomes

Measure
Reduction of Oligosaccharides in serum
time frame: 3 months (interim evaluation) + 6 months
Reduction of Oligosaccharides in CSF
time frame: 3 months (interim evaluation) + 6 months
The distance walked in 6 minutes
time frame: 3 months (interim evaluation) + 6 months
The number of steps climbed in 3 minutes
time frame: 3 months (interim evaluation) + 6 months
Pulmonary Function
time frame: 3 months (interim evaluation) + 6 months
Adverse events
time frame: 1 week
Development of clinically significant changes in vital signs and change in physical examination
time frame: 1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
time frame: 4 weeks
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
time frame: 2 weeks

Eligibility Criteria

Male or female participants from 5 years up to 21 years old.

Inclusion Criteria: 1. The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes 2. The patient must have an age at the time of screening ≥ 5 year and ≤ 21 years 3. The patient must have physical ability to perform 6-minutes walk test (6MWT), 3 minute-stair climb test (3MSCT) and pulmonary lung function test (spirometry, body plethysmography). 4. The patient must have the ability to mentally cooperate in the cognitive and motor function tests 5. The patient must have the ability to hear and follow a request. Hearing aids can be worn. 6. Patient or patient's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject) 7. The patient and his/her guardian(s) must have the ability to comply with the protocol Exclusion Criteria: 1. The patient cannot walk without support. 2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis 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. Presence of an ECHO with abnormalities within half a year 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 8. Psychosis within the last 3 months

Additional Information

Official title A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.
Principal investigator Allan M. Lund, MD
Trial information was received from ClinicalTrials.gov and was last updated in September 2012.
Information provided to ClinicalTrials.gov by Zymenex A/S.