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CompletedNCT02998879rhLaman-08Updated Oct 26, 2021

Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis

A Phase 2 interventional study of Velmanase Alfa (e.g. Lamazym) in Alpha-Mannosidosis, sponsored by Chiesi Farmaceutici S.p.A.. Completed at 6 sites in 5 countries. Open to participants aged Up to 6 Years. Per ClinicalTrials.gov, last updated 2021-10-26.

Sponsored by Chiesi Farmaceutici S.p.A. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
5
Allocation
Not applicable
Ages
Up to 6 Years
Sex
All
01

Study summary

The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years

Read the detailed description

The Primary endpoints of the study include:

  • Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis)
  • Detection of anti-velmanase alfa antibodies and neutralizing/inhibitory antibodies

The Secondary endpoints include changes from baseline to 24 months for the following parameters. Efficacy outcomes:

  • Serum oligosaccharides
  • Functional capacity: Peabody Developmental Motor Scale - 2nd edition (PDMS-2) scores, Mullen's Scale of Early Learning (MSEL) scores, Bruininks-Oseretsky Test Of Motor Proficiency-2nd Edition (BOT-2), when applicable by age (from 4 years) or upon the judgment of the physician
  • Endurance: 3-Minute Stair Climb Test (3MSCT) and 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician, 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age, or when applicable according to the judgment of the physician
  • Hearing evaluation: Otoacoustic Emissions (OAE) testing, Automatic Auditory Brainstem Response (A-ABR) audiometry
  • Immunological profile, when applicable upon the judgment of the physician:
  • CSF biomarkers: Tau protein (Tau), Neurofilament Protein Light (NFL), Glial Fibrillary Acidic Protein (GFAp), Oligosaccharides
  • Assessment of quality of life via Questionnaire to parents
  • Assessment of mannose-rich oligosaccharides in brain tissue, MRI
  • Pharmacokinetic parameters
02

Conditions studied

  • Alpha-Mannosidosis

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03

In context

alpha-Mannosidosis

26 studies on the registry are indexed under alpha-Mannosidosis; 7 are open to participants now.

This study's enrollment of 5 is below the median of 18 across 15 interventional studies indexed under alpha-Mannosidosis.

Browse alpha-Mannosidosis studies →

Lead sponsor

Chiesi Farmaceutici S.p.A. is the lead sponsor of 185 studies on the registry; 22 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 11 (65%) have results posted.

Browse Chiesi Farmaceutici S.p.A. studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 6 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Patient's custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities
  2. The subject's custodial parent(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 in leukocytes or fibroblasts \< 10% of normal activity (historical data)
  4. The subject must have an age at the time of screening \< 6 years.

Exclusion criteria

Exclusion Criteria:

  1. The subject's diagnosis cannot be confirmed by alpha-mannosidase activity \< 10% of normal activity
  2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
  3. History of BMT
  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. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
  6. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
  7. Participation in other interventional trials testing the IMP within the last 3 months.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
5 participants (actual)

Study arms

  • Experimental
    Velmanase Alfa

    velmanase alfa 1mg/kg body weight infusion

    Drug: Velmanase Alfa (e.g. Lamazym)

Interventions

06

What researchers measure

Primary outcomes

  1. Safety and tolerability of velmanase alfa as per Adverse events

    Safety and tolerability assessed as per AEs including infusion-related reactions \[IRRs\]

    Time frame: From baseline throughout study completion, at least of 2 years

  2. Safety and tolerability of velmanase alfa as per vital signs

    Time frame: From baseline throughout study completion, at least of 2 years

  3. Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per hematology

    Time frame: From baseline throughout study completion, at least of 2 years

  4. Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per blood biochemistry

    Time frame: From baseline throughout study completion, at least of 2 years

  5. Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per urinalysis

    Time frame: From baseline throughout study completion, at least of 2 years

  6. Detection of anti-velmanase alfa-IgG antibodies (ADA) and neutralizing/inhibitory antibodies

    Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained

    Time frame: From baseline throughout study completion, at least of 2 years

Secondary outcomes

  1. Evaluation of levels of Serum oligosaccharides

    Assessment of change from baseline in levels of Serum oligosaccharides

    Time frame: From baseline throughout study completion, at least for 2 years

  2. Functional capacity: The Peabody Developmental Motor Scale test (PDMS-2)

    Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained

    Time frame: From baseline throughout study completion, at least for 2 years

  3. Functional capacity: Bruininks-Oseretsky test of Motor Proficiency (BOT-2) when applicable by age (from 4 years) or upon the judgment of the physician

    Time frame: From baseline throughout study completion, at least for 2 years

  4. Functional capacity: Mullen Scales of Early Learning (MSEL)

    Time frame: From baseline throughout study completion, at least for 2 years

  5. Endurance: 3-Minute Stair Climb Test (3MSCT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician

    Time frame: From baseline throughout study completion, at least for 2 years

  6. Endurance: 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age

    Time frame: From baseline throughout study completion, at least for 2 years

  7. Hearing evaluation: Otoacoustic Emissions (OAE) testing

    Time frame: From baseline throughout study completion, at least for 2 years

  8. Hearing evaluation: Automatic Auditory Brainstem Response (A-ABR) audiometry

    Time frame: From baseline throughout study completion, at least for 2 years

  9. Immunological profile when applicable upon the judgement of the physician (Serum IgG, IgA, IgM; in vitro synthesis of IgG; in vitro proliferative response and Immunophenotype)

    Time frame: From baseline throughout study completion, at least for 2 years

  10. CSF biomarkers: Tau protein (Tau) § Neurofilament Protein Light (NFL) § Glial Fibrillary Acidic Protein (GFAp) § Oligosaccharides

    Time frame: From baseline throughout study completion, at least for 2 years

  11. Assessment of quality of life via Questionnaire

    Time frame: From baseline throughout study completion, at least for 2 years

  12. Assessment of mannose-rich oligosaccharides in brain tissue, as measured by Magnetic Resonance Spectroscopy (MRS)

    Time frame: From baseline throughout study completion, at least for 2 years

  13. Magnetic Resonance Imaging (MRI) in white matter, gray matter and in centrum semi ovale, and diffusion-MRI of the brain,

    Time frame: From baseline throughout study completion, at least for 2 years

  14. Pharmacokinetic parameters to determine Cmax (Peak Concentration)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  15. Pharmacokinetic parameters to determine Ctrough (Trough Plasma Concentration)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  16. Pharmacokinetic parameters to determine Area Under Curve (AUC24)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  17. Pharmacokinetic parameters to determine AUClast (Area Under Curve After The Last Count)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  18. Pharmacokinetic parameters to determine AUCinf (Area Under Curve From Time Zero To Infinity)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  19. Pharmacokinetic parameters to determine tmax (Time To Peak Concentration)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  20. Pharmacokinetic parameters to determine CL (Clearance)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  21. Pharmacokinetic parameters to determine t1/2 (Elimination Half-Life)

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

  22. Pharmacokinetic parameters to determine Rac (Obs) Observed Accumulation Ratio

    Time frame: At first dose (visit 1) and after 6 months (visit 26)

07

Study locations

6 sites
  • Vienna, Austria
  • Copenhagen, Denmark
  • Lyon, France
  • Hamburg, Germany
  • Mainz, Germany
  • Trieste, Italy
08

References and documents

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Dec 2016, a primary completion of Jul 2020 and a completion of Jul 2020. It does not say whether these dates are actual or estimated.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 25, 2016
First posted
Dec 21, 2016
Start
Dec 2016
Primary completionActual
Jul 2020
CompletionActual
Jul 2020
Last update
Oct 26, 2021
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Oct 26, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02998879
Lead sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
Cromsource
Responsible party
Sponsor
First posted
Dec 21, 2016
Start date
Dec 2016
Primary completion
Jul 2020
Completion
Jul 2020
Last update
Oct 26, 2021

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Oct 2021. You cannot join it, but the record below documents what was studied.

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