Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

Phase 1

Completed

• Conditions: Alpha Mannosidosis
• Interventions: Drug: Lamazym

• Registration Number: NCT01268358
• Lead Sponsor: Zymenex A/S

Brief Summary

This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.

It is the hypothesis that Lamazym is safe to use.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-10
• Study First Submitted: 2010-12-29
• Study First Submitted QC: 2010-12-29
• Study First Posted: 2010-12-30
• Primary Completion: 2011-01
• Study Completion: 2011-01
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-30
• Last Update Posted: 2020-08-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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 ≤ 20 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Lamazym 6.25	Lamazym	-
Lamazym 12.5	Lamazym	-
Lamazym 25	Lamazym	-
Lamazym 50	Lamazym	-
Lamazym 100	Lamazym	-

Primary Outcome Measures

Name	Time	Method
To evaluate the safety profile of rhLAMAN (Lamazym)	1-5 weeks

Secondary Outcome Measures

Name	Time	Method
To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03)	1 week
To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma	1 dosis