Clinical trial NCT02339441: Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis
Official title: Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Early Diffuse Cutaneous Systemic Sclerosis
Keywords: Systemic sclerosis
Lead sponsor: University of Manchester
Sponsor class: OTHER
First posted: 2015-01-15
Enrollment: 320
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Eligibility:

Age \> 18 Skin thickening \< 3 years Immunosuppressant use \< 4 months Proximal skin involvement to face/neck, elbow or knee

Brief summary: The aim of the study is to compare the effectiveness of commonly used immunosuppressant treatments for early diffuse cutaneous systemic sclerosis (SSc).

Detailed description: Diffuse cutaneous systemic sclerosis (SSc) is a rare autoimmune disease characterised by excessive connective tissue in the skin (causing skin thickening called 'scleroderma'), muscle, joints and internal organs. A number of different drugs with effects on the immune system (known as 'immunosuppressants') are currently being used by clinicians in the treatment of early diffuse cutaneous SSc, but all can have significant side effects. We want to know whether any one is definitely effective and, if so, which is the most effective.

Eligibility criteria: Inclusion Criteria:

Age \> 18 Skin thickening \< 3 years Immunosuppressant use \< 4 months Proximal skin involvement to face/neck, elbow or knee

Exclusion Criteria:

Previous use of more than 4 months of methotrexate, mycophenolate mofetil, cyclophosphamide or other immunosuppressive treatments

Previous use of immunosuppressant therapy other than methotrexate, mycophenolate mofetil or cyclophosphamide within previous months

Previous stem cell transplantation therapy

Primary outcomes:
measure: Skin score as measured by modified rodnan skin score
timeFrame: 24 months
Secondary outcomes:
measure: Pulmonary function as measured by right ventricular systolic pressure (RVSP) or pulmonary artery pressure (PAP)
timeFrame: 24 months
measure: Echocardiographic findings
timeFrame: 24 months
measure: Cochin hand function
timeFrame: 24 months
measure: Fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT) scale
timeFrame: 24 months
measure: Short form 36 questionnaire (SF-36)
timeFrame: 24 months
measure: Scleroderma Health Assessment Questionnaire
timeFrame: 24 months
measure: Haemoglobin
timeFrame: 24 months
measure: Erythrocyte sedimentation rate
timeFrame: 24 months
measure: Estimated glomerular filtration rate (eGFR)
timeFrame: 24 months
Other outcomes:
Locations:
Study officials:
affiliation: University of Manchester
name: Ariane Herrick, MD, phD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Herrick AL, Peytrignet S, Lunt M, Pan X, Hesselstrand R, Mouthon L, Silman AJ, Dinsdale G, Brown E, Czirjak L, Distler JHW, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk MC, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jobanputra P, Jordan AC, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov NS, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, MacGregor AJ, McHugh N, Muller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, McLaren JS, van Laar JM, Pathare S, Proudman SM, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad GS, Denton CP. Patterns and predictors of skin score change in early diffuse systemic sclerosis from the European Scleroderma Observational Study. Ann Rheum Dis. 2018 Apr;77(4):563-570. doi: 10.1136/annrheumdis-2017-211912. Epub 2018 Jan 6.
citation: Herrick AL, Pan X, Peytrignet S, Lunt M, Hesselstrand R, Mouthon L, Silman A, Brown E, Czirjak L, Distler JHW, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk M, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jordan AC, Jobanputra P, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov N, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, MacGregor AJ, McHugh N, Muller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, McLaren JS, van Laar JM, Pathare S, Proudman S, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad GS, Denton CP. Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Ann Rheum Dis. 2017 Jul;76(7):1207-1218. doi: 10.1136/annrheumdis-2016-210503. Epub 2017 Feb 10.