Evaluation of Long-term Efficacy of Treatment With Lamazym

NCT02478840 | Completed Phase 3

• 1 other identifier: rhLAMAN-10
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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.

Conditions

Alpha-Mannosidosis

Outcome Measures

Primary Outcomes (2)

• Change from baseline in reduction of oligosaccharides in serum

  Primary Endpoint evaluation as change

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Change from baseline in 3 Minutes Stair Climb Test (3MSCT)

  Primary Endpoint evaluation as change

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

Secondary Outcomes (16)

• 6 Minute Walk Test (6MWT)

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Pulmonary function: Forced Vital Capacity (FVC)

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Pulmonary function: Forced Expiratory Volume during first second (FEV1)

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Pulmonary function: Peak Expiratory Flow Rate (PEF)

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Functional capacity according to Bruininks-Oseretsky test of Motor Proficiency (BOT-2)

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

Other Outcomes (9)

• Quality of life based on questionnaires

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Quality of life based on questionnaires

  Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

• Development of adverse events

  1 week

Study Arms (1)

Lamazym

EXPERIMENTAL

1 mg Lamazym/kg Body weight

Drug: Lamazym

Interventions

Lamazym DRUG

recombinant human alpha-mannosidase

Also known as: rhLAMAN

Lamazym

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• The subject must still be receiving weekly intravenous infusions of Lamazym according to the AfterCare Program
• The Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
• The subject and his/her guardian(s) must have the ability to comply with the protocol

You may not qualify if:

• 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. 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
• Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial
• 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.
• 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
• Pause of the IMP for 2 consecutive weeks during the last month. Subjects are allowed to be re-screened

Sponsors & Collaborators

• Zymenex A/S: lead

Study Sites (1)

Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9

Copenhagen, DK-2100, Denmark

Location

Related Publications (4)

• Phillips D, Hennermann JB, Tylki-Szymanska A, Borgwardt L, Gil-Campos M, Guffon N, Amraoui Y, Geraci S, Ardigo D, Cattaneo F, Lund AM. Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis. Mol Genet Metab Rep. 2020 Apr 8;23:100586. doi: 10.1016/j.ymgmr.2020.100586. eCollection 2020 Jun.

  PMID: 32292699 BACKGROUND

• Borgwardt L, Guffon N, Amraoui Y, Jones SA, De Meirleir L, Lund AM, Gil-Campos M, Van den Hout JMP, Tylki-Szymanska A, Geraci S, Ardigò D, Cattaneo F, Harmatz P, Phillips D. Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis: Long-Term Data of Enzyme Replacement Therapy With Velmanase Alfa (Human Recombinant Alpha Mannosidase). Journal of Inborn Errors of Metabolism & Screening 2018, Volume 6: 1-12

  BACKGROUND

• Lund AM, Borgwardt L, Cattaneo F, Ardigo D, Geraci S, Gil-Campos M, De Meirleir L, Laroche C, Dolhem P, Cole D, Tylki-Szymanska A, Lopez-Rodriguez M, Guillen-Navarro E, Dali CI, Heron B, Fogh J, Muschol N, Phillips D, Van den Hout JMH, Jones SA, Amraoui Y, Harmatz P, Guffon N. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018 Nov;41(6):1225-1233. doi: 10.1007/s10545-018-0175-2. Epub 2018 May 3.

  PMID: 29725868 RESULT

• Harmatz P, Cattaneo F, Ardigo D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis. Mol Genet Metab. 2018 Jun;124(2):152-160. doi: 10.1016/j.ymgme.2018.04.003. Epub 2018 Apr 18.

  PMID: 29716835 RESULT

Related Links

• Study Record on EU Clinical Trials Register including results

MeSH Terms

Conditions

alpha-Mannosidosis

Condition Hierarchy (Ancestors)

Mannosidase Deficiency Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Allan M Lund, MD

  Copenhagen University Hospital, Center for Metabolic Diseases, Department of Clinical Genetics

  PRINCIPAL INVESTIGATOR

• Jens M Fogh, DVM

  Zymenex A/S

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2015
• First Posted: June 23, 2015
• Study Start: February 1, 2015
• Primary Completion: June 1, 2015
• Study Completion: June 1, 2015
• Last Updated: November 20, 2020

Record last verified: 2020-11

Locations

DK (1)