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

  • The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
  • The patient must have an age at the time of screening ≥ 5 year and ≤ 21 years
  • 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).
  • The patient must have the ability to mentally cooperate in the cognitive and motor function tests
  • The patient must have the ability to hear and follow a request. Hearing aids can be worn.
  • 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)
  • The patient and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria

  • The patient cannot walk without support.
  • Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
  • History of bone marrow transplantation
  • 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
  • Presence of an ECHO with abnormalities within half a year 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
  • 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.