A double-blind placebo-controlled pilot study of safety and tolerability of AIMSPRO® in established diffuse cutaneous systemic sclerosis

ISRCTN	ISRCTN54813778
DOI	https://doi.org/10.1186/ISRCTN54813778
ClinicalTrials.gov number	NCT00769028
Secondary identifying numbers	DISS01

Submission date: 07/10/2008
Registration date: 24/10/2008
Last edited: 27/09/2013

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Musculoskeletal Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Prof Christopher Denton
Scientific

Centre for Rheumatology and Connective Tissue Diseases
Royal Free Hospital
Pond Street
London
NW3 2QG
United Kingdom

Phone	+44 (0)20 7317 7544
Email	niamhq@gmail.com

Study information

Study design	Treatment, parallel-assignment, double-blind (subject, caregiver, investigator, outcomes assessor), randomised, placebo-controlled, safety/efficacy trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request a patient information sheet
Scientific title
Study objectives	That the hyperimmune caprine serum medication AIMSPRO® will be a safe and well tolerated therapy for patients with established diffuse cutaneous systemic sclerosis. As of 18/08/2011 the anticipated end date of this trial has been refined, and the following end dates are now accurate: Double-blind phase end date: September 2011 Open-label phase end date: October 2011
Ethics approval(s)	This study was approved by the National Research Ethics Committee, Royal Free Hospital NHS Trust on 30/07/2008 (ref: 08/H0720/63).
Health condition(s) or problem(s) studied	Diffuse cutaneous systemic sclerosis
Intervention	AIMSPRO® (manufactured by Sypharma Pty Ltd, Australia) 1.0 ml twice weekly for 6 months, injected sub-cutaneously vs placebo.
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Not Applicable
Drug / device / biological / vaccine name(s)	AIMSPRO®
Primary outcome measure	Modified Rodnan Skin Score at 0, 6 and 26 weeks.
Secondary outcome measures	The following will be assessed at 0, 6 and 26 weeks: 1. Scleroderma Health Assessment Questionnaire 2. Scleroderma UK functional score 3. Patient and Physician Global Assessment 4. SF-36 Health Survey 5. Medical Research Concil (MRC) Sum Score
Overall study start date	21/10/2008
Completion date	01/08/2011

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	Both
Target number of participants	20
Key inclusion criteria	1. Males and females aged 18 years and older 2. Must fulfil 1980 preliminary classification criteria for systemic sclerosis of the American Rheumatism Association 3. Diffuse cutaneous systemic sclerosis, as evidenced by skin sclerosis proximal to the elbows or knees and absence of the anti-centromere autoantibody 4. Three years must have elapsed since the first non-Raynaud's manifestation 5. Men and women of chilbdearing potential must use adequate birth control measures for the duration of the study and should continue such precautions for six months after receiving the last injection of AIMSPRO® 6. Hb >8.5 g/dL 7. White blood cell (WBC) >3.5 x 10^9/L 8. Platelets >100 x 10^9/L 9. Serum glutamic-oxaloacetic transaminase (SGOT)/aspartate aminotransferase (AST) and alkaline phosphatatse levels must be within twice the upper limit of normal range
Key exclusion criteria	1. Woman who are pregnant, nursing or planning pregnancy within one and a half years after screening 2. Use of any Investigational Medicinal Product (IMP) within one month prior to screening or within five half-lives of the IMP, whichever is longer 3. Use of a putative disease modifying drug within one month of screening 4. Treatment with any medication targeted at reducing tumour necrotising factor (TNF) within three months of screening 5. Previous administration of AIMSPRO® 6. History of known allergy to animal proteins 7. Serious infections in the last three months 8. Active Hepatitis B or C 9. Active tuberculosis 10. Opportunistic infections within the last six months 11. History or suggestive signs of lymphoproliferative disease 12. Known recent substance abuse 13. Poor tolerability of venesection or poor access 14. Presence of a transplanted organ (other than a corneal transplant of >3 months duration) 15. Immunosuppressive therapy within one month of screening 16. Malignancy within the past five years 17. Signs or symptoms of severe, progressive or uncontrolled renal, hepatic, haematologic, gastrointestinal, endocrine, pulmonary, cardiac or neurological disease 18. Myocardial infarction, uncontrolled cardiac failure, unstable angina or uncontrolled systemic hypotension or hypertension within the past three months 19. Screening values which deviate 20% or more from the limits of normal which are considered to be clinically significant by the investigator
Date of first enrolment	21/10/2008
Date of final enrolment	01/08/2011

Locations

Countries of recruitment

England
United Kingdom

Study participating centre

Centre for Rheumatology and Connective Tissue Diseases

London
NW3 2QG
United Kingdom

Sponsor information

Daval International Ltd (UK)
Industry

4a Gildredge Street
Eastbourne
BN21 4RL
United Kingdom

Phone	+44 (0)845 130 3014
Email	bryanyoul@gmail.com
Website	http://www.davalinternational.com
"ROR"	https://ror.org/056p0fy66

Funders

Funder type

Industry

Daval International Ltd (UK)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/01/2014		Yes	No