Phase 2/3 Study of Arimoclomol in Inclusion Body Myositis (IBM)
Research type
Research Study
Full title
Phase 2/3 Study of Arimoclomol in Inclusion Body Myositis (IBM) A Randomized, Double-blind, Placebo-Controlled Trial
IRAS ID
245087
Contact name
Michael Hanna
Contact email
Sponsor organisation
Orphazyme A/S
Eudract number
2017-004903-33
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
IND Number, 076773
Duration of Study in the UK
3 years, 3 months, 1 days
Research summary
Inclusion Body Myositis (IBM) is a chronic disorder in which muscles become inflamed and can cause muscle weakening. Weakness comes on slowly (over months or years) and progresses steadily and usually leads to severe weakness and wasting of arm and leg muscles. Patients may become unable to perform activities of daily living and most require assistive devices within 5 to 10 years of symptom onset.
The cause of IMB is unknown but there is a new evidence which suggests that the pathology of IBM results from cellular changes caused by a variety of stressful events and diseases.
Normally, in response to these stressful events, the body increases levels of Heat Shock Proteins (HSP) to help counteract and stop cellular changes. In patients with IBM increase of HSP is not sufficient enough and the toxic cellular changes cannot be reversed.
Currently there are no effective treatments for IBM.
The purpose of this study is to evaluate the safety and efficacy of potential new medicine for treatment of IMB, called Arimoclomol. The efficacy of this investigational medicine will be assessed by comparing its effects to a placebo. Arimoclomol is a medicine which prompts the human body to produce more HSP, which may help people with IMB to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
Patients’ participation in the study is expected to last approximately 22 months. At the beginning of the study patients will be randomly assigned to 1 of 2 treatment groups (Arimoclomol or Placebo), which means that patients will have 50% chance to receive the study medicine.
Patients will receive study medication in the form of capsules, orally, for 20 months and will be required to attend 8 clinic visits. Patients will also have 7 phone visits including the follow-up call 30 days after the end of treatment.
During the study patients will undergo physical examinations, complete questionnaires, have blood and urine tests and tests to measure muscle strength, and ECG (electrocardiogram).
Patient safety will be monitored throughout the study.
At the end of the study patients will return to their standard of care treatment.REC name
London - Surrey Borders Research Ethics Committee
REC reference
18/LO/0696
Date of REC Opinion
13 Jun 2018
REC opinion
Further Information Favourable Opinion