ASCEND

• Research type

  Research Study

• Full title

  A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency

• IRAS ID

  174587

• Contact name

  Robin Lachmann

• Contact email

  robin.lachmann@uclh.nhs.uk

• Sponsor organisation

  Genzyme Corporation

• Eudract number

  2015-000371-26

• Clinicaltrials.gov Identifier

  NCT02004691

• Duration of Study in the UK

  6 years, 11 months, 2 days

• Research summary

  Niemann Pick B disease (NPB) is a serious and life-threatening disorder for which there is no safe effective treatment to improve symptoms or reverse the disease course. In this disorder, a genetic defect results in reduced enzyme acid sphinogomyelinase (ASM) activity and thus the 50 fold increase in sphingomyelin levels which accumulates in organs such as the liver, spleen, lung and bone marrow leading to dysfunction.

  This study is a Phase 2/3 trial to evaluate the possible risks and efficacy of repeated doses of an experimental study drug-Olipudase alfa, in patients with Niemann Pick disease due to Acid Sphingomyelinase Deficiency (ASMD).
  Patients who have ASMD do not have enough activity of the enzyme acid sphingomyelinase (ASM), which normally helps the body break down sphingomyelin (an important building block fat for the cell). As time goes by, cells become
  overloaded with sphingomyelin and are injured, thereby affecting major organs such as the spleen and liver and leading to easy bruising, difficulty breathing, slow growth in childhood, delayed puberty, fatigue, pain and possibly other symptoms as well. Although the study doctor will discuss other available treatment options with patients prior to consenting to entry into the study, as already mentioned above, there is no specific treatment, to date, for ASMD. This study will therefore seek to determine the safe and effective dose of Olipudase alfa (a form of enzyme replacement therapy) as given intravenously to patients. The Patient Information Sheet will acknowledge that whilst it is possible that Olipudase alfa may help to improve some aspects of the disease (including decreases in liver and spleen size, increases in haemoglobin and platelet counts, reduction in bleeding and bruising, and improvements in lung disease, breathing and exercise capacity), Olipudase alfa is still experimental and thus no benefit can be guaranteed at this stage.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  15/LO/1417

• Date of REC Opinion

  27 Oct 2015

• REC opinion

  Further Information Favourable Opinion