Evaluation of Long-term Efficacy of Treatment With Lamazym

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Brief Title

Evaluation of Long-term Efficacy of Treatment With Lamazym

Official Title

A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials

Brief Summary

      The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in
      patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently
      receiving the treatment according to the AfterCare Program.

Detailed Description

      The primary objective of the trial is to evaluate the impact of the long-term treatment with
      Lamazym upon the level of biomarker oligosaccharides in serum and upon the endurance as
      measured by the change from baseline in the number of steps climbed in 3 minutes (3MSCT).

      As secondary objectives, the long term efficacy of Lamazym will be investigated upon
      endurance as measured by the change from baseline in the number of meters walked in six
      minutes (6MWT), upon pulmonary function, motor proficiency by BOT-2 and hearing capability by
      audiometry. In addition, cognitive development will be assessed by Leiter-R test. CNS
      involvement will be evaluated with MRI/MRS (for patients who previously participated in
      rhLAMAN-02 trial), CSF biomarkers (Tau, NFL, GFAp) and CSF biomarkers oligosaccharides.
      Clearance of oligosaccharides in urine will be measured.

      Long-term safety and Pharmaco-Kinetic (PK) profile after long-term treatment as measured by
      rhLAMAN levels in plasma will be assessed as well.

      Quality of life will be assessed by questionnaires (CHAQ and EQ-5D-5L).

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Change from baseline in reduction of oligosaccharides in serum

Secondary Outcome

 6 Minute Walk Test (6MWT)

Condition

Alpha-Mannosidosis

Intervention

Lamazym

Study Arms / Comparison Groups

 Lamazym
Description:  1 mg Lamazym/kg Body weight

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

18

Start Date

February 2015

Completion Date

June 2015

Primary Completion Date

June 2015

Eligibility Criteria

        Inclusion Criteria:

          1. The subject must have participated in the phase 1 trial (EudraCT number:
             2010-022084-36), phase 2a trial (EudraCT number: 2010-022085-26), phase 2b trial
             (EudraCT number: 2011-004355-40) or phase 3 trial (EudraCT number: 2012-000979-17)

          2. The subject must still be receiving weekly intravenous infusions of Lamazym according
             to the AfterCare Program

          3. The Subject or subjects legally authorized guardian(s) must provide signed, informed
             consent prior to performing any trial-related activities

          4. The subject and his/her guardian(s) must have the ability to comply with the protocol

        Exclusion Criteria:

          1. History of bone marrow transplantation

          2. 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. Subjects unable to perform the motor tests
             independently from support are permitted to participate in the trial and will be
             evaluated for the remnant non motor endpoints

          3. Any other medical condition or serious intercurrent illness, or extenuating
             circumstance that, in the opinion of the investigator, would preclude participation in
             the trial

          4. Pregnant and/or lactating women cannot participate in the trial. Concerning women of
             child bearing potential (WOCBP), 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.

          5. Participation in other interventional trials testing IMP, including rhLAMAN-07
             (EudraCT number: 2013-000336-97) and rhLAMAN-09 (EudraCT number: 2013-000321-31)
             trials with Lamazym

          6. Pause of the IMP for 2 consecutive weeks during the last month. Subjects are allowed
             to be re-screened

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Allan M Lund, MD, ,

Location Countries

Denmark

Location Countries

Denmark

Administrative Informations

NCT ID

NCT02478840

Organization ID

rhLAMAN-10

Responsible Party

Sponsor

Study Sponsor

Zymenex A/S

Study Sponsor

Allan M Lund, MD, Principal Investigator, Copenhagen University Hospital, Center for Metabolic Diseases, Department of Clinical Genetics

Verification Date

November 2020