Treatment of diffuse systemic sclerosis with hyperimmune caprine serum (AIMSPRO):
a phase II double-blind placebo-controlled trial.
Author(s): Quillinan NP(1), McIntosh D, Vernes J, Haq S, Denton CP.
Affiliation(s): Author information:
(1)Centre for Rheumatology, UCL Medical School, Royal Free Campus, , London, UK.
Publication date & source: 2014, Ann Rheum Dis. , 73(1):56-61
OBJECTIVE: The primary objective of the study was to explore safety and
tolerability of hyperimmune caprine serum (AIMSPRO) in established diffuse
cutaneous systemic sclerosis (SSc). Secondary objectives included assessment of
potential efficacy and biological activity and exploration of candidate
biomarkers.
METHODS: This was a double-blind parallel group randomised placebo-controlled
clinical trial. After informed consent 20 patients with established diffuse
cutaneous SSc of greater than 3 years duration not receiving immunosuppressive
therapy were randomised to receive either active (n=10) or placebo formulation
(n=10) by subcutaneous twice weekly injection over 26 weeks. Clinical assessments
were evaluated over 26 weeks.
RESULTS: There were no safety concerns during this study. Frequency of adverse
events was not different between active and placebo groups. Mean modified Rodnan
Skin Score (mRSS) fell by 1.4±4.7 units with active treatment but increased by
2.1±6.4 units on placebo when baseline values were compared with 26 weeks and
responder analysis showed clinically meaningful improvement in mRSS at 26 weeks
in 5 (50%) of actively treated patients compared with 1 (10%) in the control
group (p=0.062). PIIINP (µg/L) showed a comparatively larger increase in the
treatment group compared with the placebo group, (p=0.0118).
CONCLUSIONS: These results confirm tolerability and safety of this novel
biological agent in established diffuse SSc. The value of a placebo treated
control group in small clinical trials evaluating skin disease in SSc is
confirmed. Potential improvement in mRSS and changes in PIIINP in cases receiving
active therapy suggest that this intervention may be of clinical benefit and
warrants further evaluation.
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