Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

Brief Title

Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

Official Title

A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 3 Study to Evaluate the Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

Brief Summary

      Randomized, double-blind, placebo-controlled, parallel group study where subjects will
      receive velmanase alfa or placebo for 24 weeks.

      Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and
      functional assessments. Study treatment is administered weekly through i.v. infusions
    

Detailed Description

      A Screening visit (V1) will take place 7±3 days prior to randomization in order to give the
      subject enough time to consider their participation in the study, to plan the next visits
      including the long-stay visits at V2, V5 and V8 (long-stay visits as PK and certain tests are
      performed over more than one day), and to allow the clinic center to complete the evaluation
      of the eligibility criteria.

      Upon confirmation of eligibility, subjects will be randomized to receive weekly i.v.
      administration of either velmanase alfa 1 mg/kg or placebo.

      Thereafter, subjects will undergo weekly visits for administration of study treatment and
      safety data collection. Clinical, laboratory and functional assessments will be performed at
      the 4-weekly assessment visits with each subject undergoing a minimum of 8 assessment visits
      (V1 to V8).
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change in concentration of serum oligosaccharides

Secondary Outcome

 Change in Intracellular level of oligosaccharides in peripheral blood leukocytes

Condition

Alpha-Mannosidosis

Intervention

Velmanase Alfa

Study Arms / Comparison Groups

 Velmanase alfa
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

0

Start Date

January 11, 2021

Completion Date

December 29, 2021

Primary Completion Date

October 27, 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Confirmed diagnosis of alpha-mannosidosis based on alpha mannosidase activity <10% of
             normal in leukocytes or fibroblasts or through genetic testing;

          -  Capability to comply with the protocol;

          -  Evidence of informed consent provided by subject or legally authorized guardian(s)
             prior to performance of any trial-related activities.

        Exclusion Criteria:

          -  Previous hematopoietic stem cells transplantation (HSCT) with positive outcome;

          -  Major surgery planned within 3 months prior to study entry or planned during the study
             that, in the opinion of the Investigator, would preclude participation in the trial;

          -  Known clinically significant cardiovascular, hepatic, pulmonary or renal disease or
             other medical condition that would preclude participation in the study in the
             Investigator's judgment;

          -  Pregnant (as evident by a positive urine hCG or serum-hCG test) or lactating women and
             all women physiologically capable of becoming pregnant (i.e. women of childbearing
             potential [WOCBP]) UNLESS they are willing to use highly effective birth control
             methods;

          -  Participation in other interventional trials testing investigational medicinal
             products (IMPs) within the last 6 months;

          -  Total IgE >800 IU/ml;

          -  Any hypersensitivity to velmanase alfa or its excipients that, in the judgment of the
             Investigator, places the subject at an increased risk for adverse reactions

          -  Clinically active infection and recent vaccinations (within the last month before
             screening).
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Paul Harmatz, MD, , 



Administrative Informations


NCT ID

NCT04031066

Organization ID

CLI-LMZYMAA2-01


Responsible Party

Sponsor

Study Sponsor

Chiesi Farmaceutici S.p.A.


Study Sponsor

Paul Harmatz, MD, Principal Investigator, UCSF Benioff Children's Hospital Oakland


Verification Date

December 2020