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Completed

NCT Number: NCT01681953

A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis

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Key information

Conditions

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

Age range

5 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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
  • The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-Mannosidase activity < 10% of normal activity (historical data)
  • The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
  • The subject must have the ability to physically and mentally cooperate in the tests
  • The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial

Exclusion criteria

  • The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity < 10% of normal activity
  • The subject cannot walk without support
  • Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
  • History of BMT
  • 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: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
  • Psychosis; any psychotic disease, also in remission, is an exclusion criteria
  • Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
  • Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
  • Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
  • Total IgE >800 IU/ml
  • Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)

Treatment and study plan

Lamazym

Drug

ERT, i.v. infusions weekly

Other names: rhLAMAN, recombinant human alpha-mannosidase

Placebo

Drug

Infusions weekly

Primary outcomes

  1. Reduction of oligosaccharides in serum

    Time frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks

    Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group

  2. The number of steps climbed in 3 minutes (3-minute stair climb test)

    Time frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks

    Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group

Secondary outcomes

  1. Forced Vital Capacity

    Time frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks

    Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group

  2. The distance walked in 6 minutes (6-minute walk test)

    Time frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks

    Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group

  3. Adverse Events

    Time frame: 1 week

    Safety endpoint assessed weekly throughout the trial

  4. Development of clinically significant changes in vital signs and change in physical examination

    Time frame: 1 week

    Safety endpoints assessed weekly throughout the trial

  5. Clinical laboratory parameters (hematology, biochemistry and urinalysis)

    Time frame: 1 week

    Safety endpoints assessed weekly throughout the trial

  6. Development of Lamazym antibodies and neutralizing/inhibitory antibodies

    Time frame: 1 week

    Safety endpoints assessed weekly throughout the trial

Other outcomes

  1. Quantitative determination of rhLAMAN in plasma

    Time frame: 10 min, 60 min, 2 hours, 24 hours, 3 days, 7 days

    Pharmacokinetic (PK) assessments. Blood samples are drawn pre-treatment and at various times post-treatment (see time frame above)

Sponsors and collaborators

Lead sponsor

Zymenex A/S

Industry

Collaborators

  • European Commission

Registry information

Official study title

A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis.

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Sep 10, 2012
Registry last updated
Aug 3, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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