Condition category
Musculoskeletal Diseases
Date applied
02/02/2011
Date assigned
02/02/2011
Last edited
11/06/2015
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Ms Gisela Barreto

ORCID ID

Contact details

Centre for Neuromuscular Diseases
Queen Square
London
WC1N 3BG
United Kingdom
-
Gisela.Barreto@uclh.nhs.uk

Additional identifiers

EudraCT number

2008-008208-42

ClinicalTrials.gov number

NCT00769860

Protocol/serial number

7748

Study information

Scientific title

A randomised, double blinded, placebo-controlled pilot study assessing the safety and tolerability of arimoclomol in adult patients with inclusion body myositis

Acronym

Study hypothesis

Sporadic inclusion body myositis (IBM) is the commonest muscle disease acquired by those aged over 50 years. Nevertheless, despite being the subject of several clinical trials, it remains without any proven treatment. This study seeks to make the first assessment of a novel therapeutic compond, arimoclomol, in IBM. This is the first potential treatment of IBM not to target purely inflammation.

The primary objective of this proposed study is to assess the safety and tolerability of arimiclomol (100 mg three times a day [TDS]). The secondary objective is to determine whether arimoclomol has its anticipated pharmaceutical action to augment the concentration of key heat shock proteins (HSPs) in muscle tissue of IBM patients. The further objective is to evaluate a framework of clinical assessment, including measures of muscle strength, which can be used for subsequent practical and statistical planning of a larger future study of efficacy.

The study will include 12 patients with IBM, 8 of whom will receive arimoclomol and 4 a matching placebo over 4 months. The primary outcome measure will be adverse event reporting. Secondary outcome measures will include muscle strength testing (by manual muscle testing and myometry), muscle mass measure (DEXA), IBM functional rating scale, and muscle biopsies pre- and post-treatment to measure levels of HSP70, and to assess pathological changes in muscle fibres.

More details can be found here: http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=7748

Ethics approval

The Joint UCL/UCLH Committees on the Ethics of Human Research (committee A) - currently Central London REC 4 - approved (ref: 09/H0714/22)

Study design

Randomised multicentre interventional treatment trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Other

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Topic: Musculoskeletal; Subtopic: Musculoskeletal (all Subtopics); Disease: Musculoskeletal

Intervention

Twelve subjects will be randomised to one of two groups: placebo (4 patients) or Arimoclomol 100 mg three times daily (TDS) (8 patients). Participants will receive study medication for 4 months. During the treatment trial patients will be seen at screening, day 0 (baseline) and months 0.4, 1, 1.5, 2, 2.5, 3, 3.5 and 4. After the 4 month drug treatment trial, patients will be followed monthly for 8 months in order to obtain clinical endpoint measures for a total of 12 months. Muscle biopsies will be obtained at baseline (day 0) and month 4.

Follow-up length: 8 months
Study entry: registration and one or more randomisations

Intervention type

Drug

Phase

Phase II

Drug names

Arimiclomol

Primary outcome measures

Adverse events, measured throughout the study

Secondary outcome measures

1. Muscle strength testing (by manual muscle testing and myometry)
2. Muscle mass measures (by DEXA scans)
3. IBM functional rating scale
4. Pre- and post-treatment concentrations of HSP70
5. Pathological changes in muscle fibres

Overall trial start date

01/10/2010

Overall trial end date

01/10/2012

Reason abandoned

Eligibility

Participant inclusion criteria

1. Meet the diagnostic criteria for definite or probable inclusion body myositis (IBM) (Griggs 1995)
2. Muscle function adequate for quantitative muscle testing. At least 8 of the following 16 muscle groups have a Manual Muscle Test (MMT) Grade greater than 3, or greater on the modified Medical Research Council Scale
3. Aged greater than 50 years, either sex
4. Women must be post-menopausal (no menses in greater than 12 months) or status post-hysterectomy

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned sample size: 24; UK sample size: 12

Participant exclusion criteria

1. Presence of any one of the following medical conditions:
1.1. Diabetes mellitus or patients taking anti-diabetic medications
1.2. Chronic infection
1.3. Chronic renal insufficiency
1.4. Cancer other than skin cancer less than 5 years previously
1.5. Multiple sclerosis or prior episode of central nervous system demyelination
1.6. Other chronic serious medical illnesses
2. Presence of any of the following on routine blood screening:
2.1. White blood cell count (WBC) less than 300/cm3
2.2. Platelets less than 100,000/cm3
2.3. Haematocrit less than 30%
2.4. Urea greater than 10 mmol/l
2.5. Creatinine greater than 150 µmol/l
2.6. Symptomatic liver disease with serum albumin less than 30 g/l
2.7. Prothrombin time or activated partial thromboplastin greater than upper range of control values
3. Currently taking riluzole
4. Women who are pregnant or lactating
5. History of non-compliance with other therapies
6. Coexistence of other neuromuscular disease
7. Drug or alcohol abuse within last 3 months
8. Inability to give informed consent
9. Known bleeding disorder (e.g. haemophilia, Von Willebrand's Disease)
10. Use of potentially nephrotoxic drugs
11. Prior difficulties with local anaesthetic

Recruitment start date

01/10/2010

Recruitment end date

01/10/2012

Locations

Countries of recruitment

United Kingdom, United States of America

Trial participating centre

National Hospital for Neurology and Neurosurgery
London
WC1N 3BG
United Kingdom

Sponsor information

Organisation

University College London (UCL) (UK)

Sponsor details

UCL Biomedicine Research and Development Unit
Maple House
149 Tottenham Court Road
London
W1T 7NF
United Kingdom

Sponsor type

University/education

Website

http://www.ucl.ac.uk/

Funders

Funder type

Charity

Funder name

Arthritis Research UK

Alternative name(s)

Funding Body Type

private sector organisation

Funding Body Subtype

other non-profit

Location

United Kingdom

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes