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

July 30, 2020 updated by: Zymenex A/S

A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 1

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 20 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Lamazym 6.25
Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Names:
  • rhLAMAN
Experimental: Lamazym 12.5
Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Names:
  • rhLAMAN
Experimental: Lamazym 25
Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Names:
  • rhLAMAN
Experimental: Lamazym 50
Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Names:
  • rhLAMAN
Experimental: Lamazym 100
Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Names:
  • rhLAMAN

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To evaluate the safety profile of rhLAMAN (Lamazym)
Time Frame: 1-5 weeks
1-5 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Zymenex A/S

Collaborators

European Commission

Investigators

  • Study Chair: Jens Fogh, Zymenex A/S
  • Principal Investigator: Allan M. Lund, MD, Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2010

Primary Completion (Actual)

January 1, 2011

Study Completion (Actual)

January 1, 2011

Study Registration Dates

First Submitted

December 29, 2010

First Submitted That Met QC Criteria

December 29, 2010

First Posted (Estimate)

December 30, 2010

Study Record Updates

Last Update Posted (Actual)

August 3, 2020

Last Update Submitted That Met QC Criteria

July 30, 2020

Last Verified

July 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • rhLAMAN-02
  • 2010-022084-36 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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