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

• Register: ClinicalTrials.gov
• Last refreshed on: 11 January 2021
• Main ID: NCT04031066
• Date of registration: 17/07/2019
• Prospective Registration: Yes
• Primary sponsor: Chiesi Farmaceutici S.p.A.
• Public title: Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis SHAMAN
• Scientific 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
• Date of first enrolment: January 11, 2021
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT04031066
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Contacts

• Name: Paul Harmatz, MD
• Affiliation: UCSF Benioff Children's Hospital Oakland

Key inclusion & exclusion 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).



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Alpha-Mannosidosis

Intervention(s)

Drug: Placebo

Drug: Velmanase Alfa

Primary Outcome(s)

Change in serum level of total immunoglobulin (Ig)G level [Time Frame: 24 weeks (end of study)]

Change in concentration of serum oligosaccharides [Time Frame: 24 weeks (end of study)]

Secondary Outcome(s)

Assessment of PK parameter Maximum plasma Concentration [Cmax] [Time Frame: 12 weeks]

Change in serum IgG Subclasses [Time Frame: 24 weeks (end of study)]

Assessment of PK parameter Elimination half-life [t1/2] [Time Frame: 12 weeks]

Change in Intracellular level of oligosaccharides in peripheral blood leukocytes [Time Frame: 24 weeks (end of study)]

Assessment of PK parameter trough concentration (Ctrough) [Time Frame: 12 weeks]

Assessment of PK parameter Elimination half-life [t1/2] [Time Frame: 24 weeks (end of study)]

Incidence of Infections [Time Frame: 24 weeks (end of study)]

Assessment of PK parameter Area Under the Curve [AUC] [Time Frame: 12 weeks]

Assessment of PK parameter Maximum plasma Concentration [Cmax] [Time Frame: 24 weeks (end of study)]

Assessment of PK parameter Area Under the Curve [AUC] [Time Frame: 24 weeks (end of study)]

Assessment of PK parameter trough concentration (Ctrough) [Time Frame: 24 weeks (end of study)]

Secondary ID(s)

CLI-LMZYMAA2-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors