Pragmatic Clinical Trials in Scleroderma (PCTS)

U

University of West London

Status

Unknown

Conditions

Scleroderma, Systemic
Sclerosis, Systemic

Treatments

Other: Skin involvement algorithm
Other: Raynaud's phenomenon algorithm
Other: Pulmonary arterial hypertension algorithm
Other: Interstitial lung disease induction algorithm
Other: Constipation algorithm
Other: Gastroesophageal reflux algorithm
Other: Pain algorithm
Other: Inflammatory arthritis algorithm
Other: Bacterial overgrowth algorithm
Other: Digital ulcer algorithm

Study type

Interventional

Funder types

Other

Identifiers

NCT03610217
111419

Details and patient eligibility

About

Systemic Sclerosis (SSc) is an autoimmune connective tissue disease characterized by autoantibodies, fibrosis and microvascular injury and endothelial cell activation that results in vascular damage. Vascular injury induces both innate and acquired immune responses resulting in fibroblast activation and organ fibrosis. SSc may target multiple organs, including: skin, lungs, heart, vascularization, kidneys, the gastrointestinal tract and musculoskeletal structures. Mortality among scleroderma patients is significant, with a 3.5 standardized mortality ratio (SMR) in studies of prevalent cases. This mortality may be increased in the early years of the disease, reaching a SMR of 4 in a multinational inception cohort. In general, treatment strategies target involved organs as early as possible to avoid damage. Many treatment options are available for each manifestation, but evidence with respect to the order of treatment is scarce. Financial costs, the lack of proper outcome measures, difficulty to recruit patients as a rare disease, all prevent the development of new big clinical trials, oppositely to other common diseases such as stroke or cancer. The heterogeneous features of SSc may make trials challenging. The current guidelines available are the British guidelines (2017) , and the updated European League Against Rheumatism (EULAR) guidelines, published in 2017. Management guidelines have some gaps regarding second-line treatment, combinations and there are no proposed algorithms. With the pragmatic trials, the investigators intend to fill the gap between the complicated randomized clinical trials and the observational studies. Using the treatments that have already been proved useful in SSc, in an open-label randomized way and based on some refined expert-made algorithms, will allow the investigators to establish the order in how to use them. Patients will be offered to participate with the collection of their clinical data and, if they give their consent, they will be randomized according to the algorithms. There will be an optional part of the study consisting in the collection of blood samples and skin samples for future research.

Enrollment

400 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Any patient with an age >18 years meeting the 2013 SSc classification criteria managed at the Rheumatology division, St. Joseph's Healthcare London.
  • Patients who refuse to be randomized for treatments but wish to provide their data for the registry will also be included, after signing the informed consent form.

Exclusion criteria

  • Refusal to participate or to sign an informed consent form.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

400 participants in 10 patient groups

Interstitial lung disease induction
Experimental group
Treatment:
Other: Interstitial lung disease induction algorithm
Pulmonary arterial hypertension
Experimental group
Treatment:
Other: Pulmonary arterial hypertension algorithm
Raynaud's phenomenon
Experimental group
Treatment:
Other: Raynaud's phenomenon algorithm
Digital ulcers
Experimental group
Treatment:
Other: Digital ulcer algorithm
Inflammatory arthritis
Experimental group
Treatment:
Other: Inflammatory arthritis algorithm
Gastroesophageal reflux
Experimental group
Treatment:
Other: Gastroesophageal reflux algorithm
Bacterial overgrowth
Experimental group
Treatment:
Other: Bacterial overgrowth algorithm
Constipation
Experimental group
Treatment:
Other: Constipation algorithm
Skin involvement
Experimental group
Treatment:
Other: Skin involvement algorithm
Pain
Experimental group
Treatment:
Other: Pain algorithm

Trial contacts and locations

1

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Central trial contact

Andreu Fernandez-Codina, MD, MSc; Janet E Pope, MD, MPH, FRCPSC

Data sourced from clinicaltrials.gov

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