A long-term safety trial of repeated Velmanase Alfa treatment of subjects with Alpha-Mannosidosis

A multi-center, un-controlled, open-labeled trial of the long-term safety of Velmanase Alfa aftercare treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase Alfa - trials - Phase IIIb

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-000321-31-DK

Enrollment

Registered

2013-06-07

Start date

2013-06-07

Completion date

Unknown

Last updated

2023-01-09

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

Conditions

Treatement of Alpha-Mannosidosis MedDRA version: 20.0 Level: LLT Classification code 10032658 Term: Other specified disorders of carbohydrate transport and metabolism System Organ Class: 100000004850

Interventions

Product Name: Velmanase Alfa Product Code: rhLAMAN Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: velmanase alfa CAS Number: 1492823-75-2 Current Sponsor code: rhLAMAN Othe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. The subject must have participated in previous Velmanase- Alfa -trials: phase 2b: 2011-004355-40, phase 3: 2012-000979-17 or phase 2 2016-001988-36 2. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities 3. The subject and his/her guardian(s) must have the ability to comply with the protocol Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. 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 2. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial 3. Pregnancy: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and evaluate with the patients and/or parents whether or not there is a need for contraception. 4. Psychosis; any psychotic disease, also in remission, is an exclusion criteria 5. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial.

Design outcomes

Primary

Measure	Time frame
Main Objective: The overall objective of this trial is to provide aftercare treatment with Velmanase- Alfa and to evaluate the safety and efficacy of repeated Velmanase- Alfa i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase- Alfa -trial. This protocol only concern subjects, where compassionate use program was not accepted (OR NO PHYSICIAN COULD BE IDENTIFIED) Efficacy will be evaluated once yearly to follow the subject’s progress in clinical parameters and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.;Secondary Objective: Not applicable;Primary end point(s): All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial). Safety Endpoints: •Adverse events (AEs) •Vital signs and change in physical examination •Development of Velmanase- Alfa antibodies and neutralizing/inhibitory antibodies •Clinical laboratory parameters (hematology, biochemistry, IgG, IgA, IgM and urinalysis) ;Timepoint(s) of evaluation of this end point: Safety will be assessed at every visit. Development of antibodies are measured every 12th week Serum Oligosaccarides will be evaluated every 6th months.	—

Measure

Time frame

Main Objective: The overall objective of this trial is to provide aftercare treatment with Velmanase- Alfa and to evaluate the safety and efficacy of repeated Velmanase- Alfa i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase- Alfa -trial. This protocol only concern subjects, where compassionate use program was not accepted (OR NO PHYSICIAN COULD BE IDENTIFIED) Efficacy will be evaluated once yearly to follow the subject’s progress in clinical parameters and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.;Secondary Objective: Not applicable;Primary end point(s): All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial). Safety Endpoints: •Adverse events (AEs) •Vital signs and change in physical examination •Development of Velmanase- Alfa antibodies and neutralizing/inhibitory antibodies •Clinical laboratory parameters (hematology, biochemistry, IgG, IgA, IgM and urinalysis) ;Timepoint(s) of evaluation of this end point: Safety will be assessed at every visit. Development of antibodies are measured every 12th week Serum Oligosaccarides will be evaluated every 6th months.

—

Secondary

Measure	Time frame
Secondary end point(s): All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial). The Efficacy endpoints of the trial are evaluation of: o Oligosaccharides in serum o Motor proficiency (BOT-2) o Three minute stair climb test (3MSCT) o Leiter R o Forced Vital Capacity (FVC) o Six minute walk test (6MWT) o Pure tone Audiometry o Questionnaires CNS parameters (visit 2500 only): o Assessment of mannose-rich oligosaccharides in brain tissue as measured by MRS visual score (for patients who previously participated in rhLAMAN-02), MRI diffusion coefficient in white matter, gray matter and centrum semi oval o Cerebrospinal fluid neuro-degeneration biomarker (Tau, NFL, GFAp) o Oligosaccharides in CSF Other laboratory parameters (visit 2500 only): o Plasma PK profile o Oligosaccharides in 24-h urine collection o Measurement of in vivo biological activity;Timepoint(s) of evaluation of this end point: Efficacy will be assessed yearly, counted from the latest evaluation in the subject's previous Velmanase- Alfa trial, and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.	—

Measure

Time frame

Secondary end point(s): All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial). The Efficacy endpoints of the trial are evaluation of: o Oligosaccharides in serum o Motor proficiency (BOT-2) o Three minute stair climb test (3MSCT) o Leiter R o Forced Vital Capacity (FVC) o Six minute walk test (6MWT) o Pure tone Audiometry o Questionnaires CNS parameters (visit 2500 only): o Assessment of mannose-rich oligosaccharides in brain tissue as measured by MRS visual score (for patients who previously participated in rhLAMAN-02), MRI diffusion coefficient in white matter, gray matter and centrum semi oval o Cerebrospinal fluid neuro-degeneration biomarker (Tau, NFL, GFAp) o Oligosaccharides in CSF Other laboratory parameters (visit 2500 only): o Plasma PK profile o Oligosaccharides in 24-h urine collection o Measurement of in vivo biological activity;Timepoint(s) of evaluation of this end point: Efficacy will be assessed yearly, counted from the latest evaluation in the subject's previous Velmanase- Alfa trial, and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.

—

Countries

Denmark, Poland

Contacts

Public ContactClinical Project Manager

Chiesi Farmaceutici S.p.A

clinicaltrials_info@chiesi.com003905211689176

Outcome results

None listed

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