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

NCT ID: NCT01285700

Last Updated: 2012-09-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-01-31

Study Completion Date

2012-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Alpha Mannosidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lamazym 25

25 U/kg

Group Type ACTIVE_COMPARATOR

Lamazym

Intervention Type DRUG

ERT, infusion weekly

Lamazym 50

50 U/kg

Group Type ACTIVE_COMPARATOR

Lamazym

Intervention Type DRUG

ERT, infusion weekly

Interventions

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Lamazym

ERT, infusion weekly

Intervention Type DRUG

Other Intervention Names

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rhLAMAN

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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European Commission

OTHER

Sponsor Role collaborator

Zymenex A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Allan M. Lund, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark

Jens Fogh

Role: STUDY_CHAIR

Zymenex A/S

Locations

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Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9

Copenhagen, , Denmark

Site Status

Countries

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Denmark

Other Identifiers

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2010-022085-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

rhLAMAN-03

Identifier Type: -

Identifier Source: org_study_id