Post authorisation observational study of Cerdelga in Gaucher patients
Research type
Research Study
Full title
A prospective multicenter observational post authorization safety sub-registry to characterize the long-term safety profile of commercial use of eliglustat (Cerdelga®) in adult patients with Gaucher disease
IRAS ID
246007
Contact name
Ana Jovanovic
Contact email
Sponsor organisation
Sanofi Genzyme
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
5 years, 3 months, 26 days
Research summary
The ELISAFE (OBS14099) study is conducted in patients with Gaucher disease (GD) who are already participating in the International Collaborative Gaucher Group (ICGG) Gaucher registry (DIREGC07009).
The study is designed to follow patients on Cerdelga and Cerezyme and in particular assess the long term safety profile of Cerdelga and descriptively compare with Cerezyme by collecting real-world safety data as part of a post approval commitment.
There is no set duration for the study and patients can remain on the study as long as it is open and they remain willing to participate. Data collection will cease once the 100th patient being treated with Cerdelga has completed 4 years of follow-up. The number of patients on Cedelga who continue their observation in EliSafe will be evaluated annually to determine the need to replace patients who dropped out of the study for any reason prior to completion of their 4-year observation period.
There will be no procedures mandated for the study and patients will visit their Health Care Practitioner (HCP) at the same frequency as expected for the usual care of their Gaucher disease. If however, the interval between their last visit and their next visit exceeds 6 months, the research team will arrange a phone call with the patient to inquire about their health and the side effects that they may have experienced.
Lay summary of study results: This study looked at how safe Cerdelga is when used in everyday clinical practice. The results showed that Cerdelga has a good safety profile, similar to what was seen in earlier clinical trials and in real-world use since it became available.
The most common side effects were stomach-related issues (about 35% of participants) and muscle or joint problems (about 34%). A small number of participants (12%) had serious side effects, mostly due to infections, but most of these people recovered by the end of the study. Importantly, there were no deaths or cases where treatment had to be permanently stopped because of side effects linked to Cerdelga.
Cerdelga was used according to its approved guidelines: only in people with Gaucher disease type 1 (GD1), and most participants were tested to make sure they had the right genetic profile for safe use. Overall, Cerdelga was both safe and used appropriately.REC name
London - Bloomsbury Research Ethics Committee
REC reference
18/LO/0874
Date of REC Opinion
22 May 2018
REC opinion
Favourable Opinion