Have you or your loved ones been diagnosed with alpha-mannosidosis?

You may be eligible to participate in a alpha-mannosidosis clinical trial.

Have you or your loved ones been diagnosed with alpha-mannosidosis? You may be eligible to participate in a alpha-mannosidosis clinical trial.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Alpha-Mannosidosis Clinical Trial

NCT02998879 | Phase 2 | Interventional

Have you or your loved ones been diagnosed with alpha-mannosidosis?

You may be eligible to participate in a alpha-mannosidosis clinical trial.

Have you or your loved ones been diagnosed with alpha-mannosidosis? You may be eligible to participate in a alpha-mannosidosis clinical trial.

Completed

Male & Female

Up to 6

Years old

This study has recruited 5 Participants

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

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Details for the study

Brief Title

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

Official Title

A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis

Brief Summary

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

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

Treatments and/or Procedures

Velmanase alfa e g lamazym

iv infusion treatment

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

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

From baseline throughout study completion, at least of 2 years

Primary

Safety and tolerability of velmanase alfa as per Adverse events

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

From baseline throughout study completion, at least of 2 years

Secondary

Evaluation of levels of Serum oligosaccharides

Assessment of change from baseline in levels of Serum oligosaccharides

From baseline throughout study completion, at least for 2 years

Secondary

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

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

From baseline throughout study completion, at least for 2 years

Study Criteria

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:

  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.