BN29854 - Olesoxime in patients with SMA
Research type
Research Study
Full title
MULTICENTER, OPEN LABEL, SINGLE ARM STUDY TO EVALUATE LONG-TERM SAFETY, TOLERABILITY, AND EFFECTIVENESS OF 10MG/KG OLESOXIME IN PATIENTS WITH SMA.
IRAS ID
185946
Contact name
Volker Straub
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2015-001589-25
Duration of Study in the UK
4 years, 2 months, days
Research summary
Spinal muscular Atrophy (SMA) is an autosomal recessive neuromuscular disorder clinically characterized by progressive muscular weakness and atrophy. It is the most common genetic cause of infant mortality with an estimated incidence of 1 in 6000–10 000 live births.
SMA leads to predominantly proximal muscle atrophy and weakness and the potential for medical complications such as scoliosis, joint contractures and restrictive lung disease due to respiratory muscle weakness. Progressive respiratory failure and frequent pulmonary infections are common in SMA Type 1 and 2, so that in severe phenotypes non-invasive respiratory support is often needed.
There is no effective pharmacological treatment for SMA. Current management is focused on prevention and treatment of comorbidities, such as failure to thrive, surgical and non-surgical treatment for scoliosis and contractures, pulmonary hygiene, non-invasive ventilation, mobility and seating support, and physical and occupational therapy. Treatment is supportive with the goals being to improve the patient’s quality of life and to minimize disability.
Olesoxime is a cholesterol-like compound identified through its survival-promoting activity on trophic factor deprived motor neurons in culture. Olesoxime binds to proteins that have been implicated in the formation or modulation of the mitochondrial permeability transition pore complex. By binding to these proteins, olesoxime may preserve essential mitochondrial functions such as calcium buffering in stressed neurons, thereby reducing neuronal degeneration and death.
This open label study will provide an opportunity for SMA patients who participated in previously completed studies with olesoxime to benefit from continuous olesoxime treatment. The main objective of the study is to evaluate long-term safety and effcetiveness to support long term treatment with olesoxime in SMA patients.
In the UK it is anticipated there will be 18 patients across 3 participating sites.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
15/NE/0317
Date of REC Opinion
19 Nov 2015
REC opinion
Further Information Favourable Opinion