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Gaucher Disease Type 3 Clinical Trial

November 2017

April 2017

Clinical Trial ALERT!!!

Genzyme’s oral therapy in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3 (LEAP)

In the 2016 we reported on the impending clinical trial for adults (18 years and older) with Type 3 Gaucher disease with Genzyme’s small molecule GZ/SAR402671. We are delighted to report that this 52-week two-part study phase 2 clinical trial is now recruiting with sites open in the UK and US.

Recruitment onto this trial has been challenging for our nGD patients in the UK as despite this trial being for Type 3 patients there was a mandatory eligibility requirement of lung function (the ability of the lungs to transfer gas from inhaled air to the red blood cells in pulmonary capillaries) and that had excluded all the UK GD 3 community, meaning that with three sites open in the UK 0 patients have been enrolled in this study.

However following communication with the Gauchers Association and the Principle Investigators demonstrating that there were no eligible patients in the UK, Genzyme have removed this mandatory criteria from the trial, the UK Centre’s will now invite those patients that wish to be considered for this trial to visit their Centre for assessment.

Below is an outline of what the study objectives are and further details, including the eligibility and exclusion criteria can be found online at; http://bit.ly/2ns323X

The Primary Objective of the study:

Part 1:

• Evaluate central nervous system (CNS) biomarkers in adult Gaucher disease (GD) type 3 (GD3) patients that distinguish GD3 from Gaucher disease type 1 (GD1).

• Screen adult GD3 patients who qualify for treatment with GZ/ SAR402671 in Part 2.

Part 2:

• Evaluate the safety and tolerability of GZ/SAR402671 in adult GD3 patients.

• Evaluate the change in cerebrospinal fluid (CSF) central nervous system (CNS) biomarkers from adult GD3 patients receiving GZ/SAR402671.

And Secondary Objectives:

• Evaluate the pharmacokinetics of GZ/SAR402671 in adult GD3 patients.

• Explore the efficacy of GZ/SAR402671 in infiltrative lung disease (IDL) in adult GD3 patients.

• Explore the efficacy of GZ/SAR402671 in systemic disease in adult GD3 patients.

• Explore the efficacy of GZ/SAR402671 in neurological function and on exploratory CSF biomarkers in adult GD3 patients

PLEASE NOTE: To be eligible for this study all participants MUST be on Cerezyme for at least 6 months prior to enrollment, at a stable monthly dose and must continue at the same monthly dose during the study.

July 2016

At our 7th nGD Family conference in Manchester in November last year we were extremely grateful to Dr Derralynn Hughes and Dr Anupam Chakrapani who led a small closed session for the patients and parents of our GD3 community to inform them of a clinical trial that would be starting in 2016 and that 3 of our adult centres in the UK would be designated as trial sites.

It was extremely important to be able to have these discussions with the patients and their family members so that they could ask questions and then take that information away with them to decide if they wanted to put themselves forward for the trial once it opened in mid-2016.

This phase II trial will be opened to adult patients aged 18-40 years old with a confirmed diagnosis for Type 3 Gaucher disease. The trial drug is an oral therapy which will see patients take the new oral trial drug for 6 months in addition to their ERT, with a view to then continuing for the remainder of the trial, 52 weeks in total with the oral drug ONLY which is believed to cross the blood brain barrier.