Clinical trial information:
SHAMAN study

The SHAMAN study is a clinical trial.1 Clinical trials are clinical studies performed in humans, designed and carried out by scientists, pharmaceutical companies and healthcare professionals to ensure that new medications are safe for use and effective at treating the condition for which they were designed.2,3 There are many regulations around clinical trials to ensure the safety of the participants.3
Visit this page for more information on clinical trials.


The goal of the SHAMAN study is to establish the safety and efficacy of a new drug, velmanase alfa, compared to placebo in patients with Alpha Mannosidosis1 — a condition in which the enzyme alpha-mannosidase does not function properly, causing cell damage throughout the body.4


Velmanase alfa is an enzyme replacement therapy (ERT) that is still in the investigational stage in the USA and Canada. ERT provides replacement alpha-mannosidase to patients with the working enzyme through direct infusion into the blood stream (intravenous infusion — IV).
The enzyme will be taken up by the cells to replace the malfunctioning enzyme.5


In the study, there will be two treatment groups of participants: one will receive velmanase alfa and the second will receive placebo once weekly for 24 weeks. Visit this page to learn about placebos. The group you will join will be decided randomly (like flipping a coin).
A variety of tests will be performed in order to establish a full picture of how well the treatment works, from drug absorption over time to quality of life, and how safe it is.1 The clinical trial is double-blinded, which means that the participant and the research staff will not know which treatment is being administered to each participant, but the study team can find out in case of an emergency.1,3

Who can take part in the study?1

Patients of all sexes and ages who have a confirmed diagnosis of Alpha Mannosidosis can take part in the trial.
Children can take part in the trial with the authorization of their legal guardian.
Participants must not have received hematopoietic stem cell transplantation (HSCT) before, or have failed previous HSCT treatment.

A description of the SHAMAN clinical study is available on, the US clinical study database, as required by US Law and available at the following link: The current clinical study is identified by the study code: NCT04031066.

  1. Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis (SHAMAN). Available at: Last accessed: January 21, 2020.
  2. Learn About Clinical Studies. Available at: Last accessed: January 21, 2020.
  3. National Institutes of Health. NIH Clinical Research Trials and You. Available at: Last accessed: January 21, 2020.
  4. Borgwardt L, Stensland HM, Olsen KJ, et al. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation. Orphanet J Rare Dis. 2015;10:70.
  5. European Medicines Agency. New enzyme replacement therapy to treat rare genetic disorder alpha-mannosidosis in children and adults. Available at: Last accessed: January 21, 2020.
  6. Chiesi. Clinical Trial Transparency and Data Sharing. Available at: Last accessed: January 21, 2020.
  7. Malm D, Nilssen Ø. Alpha-mannosidosis. Orphanet J Rare Dis. 2008;3:21.
  8. Bavisetty S, Grody WW, Yazdani S. Emergence of pediatric rare diseases: Review of present policies and opportunities for improvement. Rare Dis. 2013;1:e23579.