VBP15-004 study is a phase 2b, double blind, placebo and prednisolone controlled study to evaluate safety and efficacy of vamorolone in ambulant boys with Duchenne muscular dystrophy over a 6-month period.
The study will compare the effect and the side effect profile of vamorolone versus placebo and daily prednisolone to determine whether vamorolone improves muscle strength and function compared to placebo and whether it has less side effects compared to prednisolone.
The study is expected to start recruitment in January 2018.
The study will recruit boys with a confirmed diagnosis of Duchenne muscular dystrophy (defined as total absence of dystrophin on muscle biopsy or genetic confirmation of an out-of-frame mutation in the dystrophin gene), who has not previously receive corticosteroids (prednisolone or deflazacort), older than 4 and younger than 7 years of age. Participants must be able to walk and to get up from the floor without assistance. Any condition which would preclude the prescription of corticosteroids would represent an exclusion criteria from the study. Current or previous (within 3 months) participation in other clinical trials with investigational drugs and the use of idebenone will represent exclusion criteria. Other specific inclusion and exclusion criteria will be in place to ensure to reach the primary endpoints within the study period.
This is a randomized, double blind study. This means that decision on whether a subject will receive vamorolone, placebo or prednisolone will be made randomly (like flipping a coin) by a computer and neither the participants, the families nor the study doctors will know which treatment the subject is receiving during the study. This is to guard against both experimenter bias and placebo effects.
The study will recruit 100 boys with DMD across approximately 30 sites in Europe, the US, Canada, Israel and Australia.
In order to participate in the study, the boys and the families will need to sign a consent form and full information about the details of the study will be provided to allow them to make an informed decision.
During the study, participants will take the study drug for 6 months. Study drugs (vamorolone, prednisolone and placebo) will be administered orally daily, in the morning after breakfast. Study drugs will be administered in clinic during the study visits and at home in between study visits. Parents will be asked to keep a diary to record when the participant takes the drug, any missed dose or if he felt unwell while participating in the study.
Participants will be asked to regularly attend the clinic for study assessments. The frequency of these assessments will be higher at the beginning of the study (during screening period and when study drugs is started). At each study visit, the doctor and the study nurse will ensure that the participants are well and have not experienced side effects from the study drug. To do so, several procedures will be performed, including physical examination, blood and urine tests, questionnaires and vital signs. At some visits, participants will undergo a comprehensive physiotherapy and functional assessment to evaluate the effect of the study drugs on muscle strength: these will include the North Star Ambulatory Assessment, functional testing (time to rise from the floor, time to climb stairs, time to run 10 meters), 6 minute walking test (6MWT), muscle MRI, measures of the respiratory and heart functions.
At the end of the study (6 month treatment period) participants will be offered the possibility to continue receiving vamorolone in an extension study or to move into standard care (i.e. discontinuing study drug and receiving treatment according to care recommendations).
The study will be conducted in accordance with the highest standards of care currently available for the management of DMD.
The boys and the families will be able to withdraw from the study at any time and this will not affect the level of care they receive.