Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

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

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-01-31

Study Completion Date

2013-09-30

Brief Summary

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The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis

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Detailed Description

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Conditions

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Alpha-Mannosidosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lamazym

1 mg/kg body weight

Group Type EXPERIMENTAL

Lamazym

Intervention Type DRUG

ERT, i.v. infusions weekly

Interventions

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Lamazym

ERT, i.v. infusions weekly

Intervention Type DRUG

Other Intervention Names

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rhLAMAN recombinant human alpha-mannosidase

Eligibility Criteria

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Inclusion Criteria

* The subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
* Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
* The subject and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria

* The subject 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
* 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: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study
* Psychosis within the last 3 months
* Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
* Participation in other interventional trials testing IMP except for studies with Lamazym

Minimum Eligible Age

5 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No