Copenhagen, June 21, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that the first patient has received the first dose in a Phase II clinical trial for Gaucher disease.
Anders Hinsby, Chief Executive Officer, said: “With the enrollment of the first patient in the Gaucher disease trial, we reach another important milestone in the development of our pipeline this year. Glucocerebrosidase deficiency leads to severe neurological manifestations in many patients with Gaucher disease. With this trial we move a step closer to a treatment option for these patients.”
The trial takes place at clinical sites in India and will include approximately 40 patients. Patients will be randomized 1:1:1:1 into four treatment arms – active treatment at three different doses and placebo. The patients will receive arimoclomol or placebo-controlled treatment for six months. Following the placebo-controlled period, the placebo group will be re-randomized into one of the three active treatment groups for a six-month extension.
The primary endpoint in this trial is measurement of the enzyme chitotriosidase, which is a known and well-characterized marker of Gaucher disease. Measurements will be performed in samples from cerebrospinal fluid and blood to assess arimoclomol’s potential effect on both neurological and peripheral disease.
Results from the Phase II trial are expected in H1 2019.