A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis

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 - Phase III

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-000979-17-DE

Enrollment

Registered

2012-07-09

Start date

2012-12-17

Completion date

Unknown

Last updated

2015-11-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Treatement of Alpha-Mannosidosis

Interventions

Product Name: Lamazym Product Code: rhLAMAN Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: rhLAMAN Current Sponsor code: rhLAMAN Other descriptive name: recombinant human a

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities 2. The subject and his/her guardian(s) must have the ability to comply with the protocol 3. The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. The subjects diagnosis cannot be confirmed by alpha-mannosidase activity 800IU/ml 13. 13. Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)

Design outcomes

Primary

Measure	Time frame
Main Objective: 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;Secondary Objective: PK evaluation;Primary end point(s): Primary Efficacy Endpoints is change from baseline in the active treated group versus the placebo group: • The level of oligosaccharides in serum • 3-minute stair climb test (3MSCT);Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site at baseline (prior to first dose), as midterm evaluation (after 26±3 weeks, referred to as midterm evaluation or visit 26a), and as end evaluation (after 52±3 weeks, referred to as end evaluation or visit 52a).	—

Measure

Time frame

Main Objective: 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;Secondary Objective: PK evaluation;Primary end point(s): Primary Efficacy Endpoints is change from baseline in the active treated group versus the placebo group: • The level of oligosaccharides in serum • 3-minute stair climb test (3MSCT);Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site at baseline (prior to first dose), as midterm evaluation (after 26±3 weeks, referred to as midterm evaluation or visit 26a), and as end evaluation (after 52±3 weeks, referred to as end evaluation or visit 52a).

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Secondary

Measure	Time frame
Secondary end point(s): Prioritized Secondary Efficacy Endpoints: • Forced Vital Capacity (FVC) • 6 minute walk test (6MWT) Other Secondary Efficacy Endpoints: • Bruininks-Oseretsky test of Motor Proficiency (BOT2) • Leiter R • Cerebrospinal fluid biomarkers incl oligosaccharide and standard tests • Pulmonary Function Tests • Pure tone audiometry (PTA) • Questionnaires (CHAQ and EQ-5D) Safety Endpoints: • Adverse events (AEs) • Vital signs and change in physical examination • Clinical laboratory parameters (hematology, biochemistry and urinalysis) • Development of Lamazym antibodies and neutralizing/inhibitory antibodies Pharmacokinetic endpoints: • Pharmacokinetics (PK) ;Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site at baseline (prior to first dose), as midterm evaluation (after 26±23 weeks, referred to as midterm evaluation or visit 26a), and as end evaluation (after 52±3 weeks, referred to as end evaluation or visit 52a). Safety will be assessed at every visit. PK will be analyzed at visit 1.	—

Measure

Time frame

Secondary end point(s): Prioritized Secondary Efficacy Endpoints: • Forced Vital Capacity (FVC) • 6 minute walk test (6MWT) Other Secondary Efficacy Endpoints: • Bruininks-Oseretsky test of Motor Proficiency (BOT2) • Leiter R • Cerebrospinal fluid biomarkers incl oligosaccharide and standard tests • Pulmonary Function Tests • Pure tone audiometry (PTA) • Questionnaires (CHAQ and EQ-5D) Safety Endpoints: • Adverse events (AEs) • Vital signs and change in physical examination • Clinical laboratory parameters (hematology, biochemistry and urinalysis) • Development of Lamazym antibodies and neutralizing/inhibitory antibodies Pharmacokinetic endpoints: • Pharmacokinetics (PK) ;Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site at baseline (prior to first dose), as midterm evaluation (after 26±23 weeks, referred to as midterm evaluation or visit 26a), and as end evaluation (after 52±3 weeks, referred to as end evaluation or visit 52a). Safety will be assessed at every visit. PK will be analyzed at visit 1.

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Countries

Belgium, Denmark, Germany, Spain, Sweden

Contacts

Public ContactCEO

Zymenex A/S

zxmail@zymenex.com+4548250054

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026