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TREAT-NMD: Trials in SMA
Dubowitz Neuromuscular Centre
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Latest Clinical Trial News 16/09/2016:  
​Statement from ENDEAR Principle Investigators in London and Newcastle, UK

You may have seen the recent announcements from pharmaceutical companies Biogen and Ionis about the interim results of the clinical trial called ENDEAR Phase 3. This involved infants with SMA Type 1. The drug being trialed was Nusinersen. 

Investigators from our two specialist centres were involved in this important study and are delighted with its positive outcome.

Biogen is now working to open an Expanded Access Program (EAP) in the autumn of 2016. Such a programme is designed to provide eligible others outside a trial with access to the successfully trialed treatment prior to potential regulatory approval.

Since the announcement made by Biogen about a proposed EAP for Nusinersen, we have received many requests from families with an infant with SMA Type 1, both from the UK and other countries, who, understandably, wish their children to participate. We are therefore wanting to make sure everyone making enquiries has access to the same, correct information.

The first step for Biogen will be to ensure that all the children from the current ENDEAR study have access to Nusinersen. This is via what is called their extension studies.

Next, Biogen needs to ensure that the proposed EAP can in fact take place in the UK according to UK laws and regulations. A third party called Idis/Clingen will help with the regulatory issues and practical steps that should allow the EAP to start in the UK. It is anticipated that the approval process is likely to take several months.

Because of our experience in administering Nusinersen, initially only our two existing clinical trial sites could potentially be able to participate in the proposed EAP. However, the expectation is that other sites could also be opened in the future.

It is clear that there is an urgent need to agree how to prioritise the requests of families with infants with SMA Type 1 whose child could potentially be enrolled in the EAP for Nusinersen. With this in mind, we are currently in discussion with other European and US sites to agree on the most rational and equitable approach. This discussion will take into consideration all the available information on the potential risks and benefits of Nusinersen for infants with SMA Type 1.

At this time, we are therefore unable to predict if and when we will be able to start the enrolment of infants with SMA Type 1 in an EAP.  We do though know that children who are not eligible to use the UK National Health Service (NHS), will be unable to access the proposed EAP in the UK.

As for any EAP, this proposal would anyway be a limited interim arrangement while full regulatory approval for Nusinersen is applied for in the UK.

We understand how frustrating and distressing this process can be for families. We are doing all we can to move things forward and will update you as soon as more information is available from Biogen, Ionis and the regulatory authorities.

Families can send a message to the following email address to obtain more information about Nusinersen Expanded Access Program (EAP):  Patientcenter@biogen.com
 
Physicians can send a message or call the number below.  medinfo@biogen.com
(+1) 866-633-4636
Francesco Muntoni                                                     Volker Straub
London ENDEAR Principal Investigator                      Newcastle ENDEAR Principal Investigator

For further information about the EAP please see: http://media.biogen.com/press-release/investor-relations/biogen-and-ionis-pharmaceuticals-report-Nusinersen-meets-primary-en) 

Information on the Trophos study
Trohphos Study: A Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients
The study was conducted in UK at GOSH London, Royal Victoria Hospital Newcastle and the Birmingham Heartland Hospital. The study was sponsored by TROPHOS (a biopharmaceutical company based in France). The aim was to assess efficacy, futility, safety and tolerability of a new drug called olesoxime (TRO 19622). Olesoxime displayed an excellent safety profile and has been well tolerated in phase I clinical trials.

In recent news, Roche has agreed to acquire the French biotechnology company Trophos, which is developing the investigational compound olesoxime for spinal muscular atrophy (SMA). The transaction is subject to customary closing conditions, including approval by the French Foreign Investment Clearance. The transaction is anticipated to close in the first quarter of 2015.

Olesoxime is an advanced drug candidate for SMA having completed a pivotal phase II trial in 2013. Roche will initiate the manufacturing of olesoxime and associated regulatory activities to allow an open-label extension study for patients who participated in the pivotal Phase 2 study. Roche are committed to bringing this treatment to patients as quickly as possible.

ISIS CS3 A Phase III Trial in Type-1 SMA
A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy

Roche Moonfish Study
A Multicentre, Randomized, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Investigate the Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Ro6885247 Following 12 Weeks of Treatment In Adult and Paediatric Patients with Spinal Muscular Atrophy
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