The Abatacept Systemic SclErosis Trial (ASSET)
Research type
Research Study
Full title
A phase II study to evaluate subcutaneous abatacept vs. placebo in diffuse cutaneous systemic sclerosis—a double-blind, placebo-controlled, randomized controlled trial
IRAS ID
174615
Contact name
Chris Denton
Contact email
Eudract number
2014-005323-27
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
NCT02161406, US NCT Number
Duration of Study in the UK
1 years, 6 months, 4 days
Research summary
This is a phase II study to asses how safe and effective treatment of diffuse systemic scleroderma (SSc) with abatacept 125mg injected subcutaneously (under the skin) is. The effectiveness will be assessed primarily using the modified Rodnan Skin Score which measures skin tightness in Scleroderma patients.
Diffuse systemic scleroderma is a long term condition that causes thickening and hardening of the skin as well as other body parts i.e. (muscle, joints and blood vessels)
Several observations support the role of activated T cells in the disease development of SSc.A recent pilot study evaluated the effect of blockade of T cell co-stimulation with intravenous abatacept in patients withSSc was effective and there were no serious or unanticipated adverse events.This study was limited by a small sample size and skewing of disease duration between those randomized to placebo vs abatacept.Despite these limitations, our preliminary data support a potential role for abatacept in the treatment of cutaneous sclerosis in SSc. Therefore, we aim to perform a phase 2, multi-center double-blind randomized controlled trial to build on this pilot study.The study will consist of 2 phases: A double blind phase lasting 12 months and an open label phase lasting 6 months. inclusion criteria:
1. Signed written informed consent
2. Diagnosis of SSc, as defined using the 2013 American College of Rheumatology/ European Union League Against Rheumatism classification of SSc
3. dcSSc as defined by LeRoy and Medsger
4. Disease duration of ≤ 36 months (defined as time from the first non−Raynaud phenomenon manifestation)
5. Age ≥ 18 years at the screening visit
6. If female of childbearing potential, the patient must have a negative pregnancy test at screening and baseline visits
7. Oral corticosteroids and NSAIDs are permitted if the patient is on a stable dose regimen for ≥ 2 weeks prior to the baseline visit.
Patients will be seen at the Royal Free Scleroderma clinicREC name
London - Harrow Research Ethics Committee
REC reference
15/LO/1027
Date of REC Opinion
16 Sep 2015
REC opinion
Further Information Favourable Opinion