Digital Ulcer, Raynaud Phenomenon, Scleroderma, Systemic Sclerosis
Conditions
Keywords
Rheopheresis, Raynaud Phenomenon, Systemic Sclerosis, Digital Ulcers, Therapeutic plasma exchange
Brief summary
In this feasibility study, we aim to explore therapeutic Rheopheresis (RheoP) as a novel treatment option for SSc-associated Raynaud's phenomenon and/or digital ulcers and compare it to the standard of care treatment (intravenous iloprost. RheoP has been used for RP/DU with some success in observational studies, nevertheless, the optimal treatment modality, duration, or frequency of RheoP (and PEX in general) in SSc has not been established as of yet.
Interventions
PROCEDURERheopheresis treatment
After obtaining venous access, anticoagulated blood is pumped through a plasmafilter. The plasma is then run through the Rheofilter and large plasma proteins are removed. Finally, cells are reinfused, and blood is returned to the patient.
DRUGIntravenous Infusion
Standard of care treatment consists of intravenous iloprost infusions at a dose of 0.5-2 ng/kg/min administered over at least 6 hours as per local standard
Sponsors
Peter Korsten
DiaMed GmbH
Study design
Allocation
RANDOMIZED
Intervention model
FACTORIAL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Adult patients fulfilling ACR/EULAR classification criteria for SSc 2. Presence of RP with or without DU 3. Failure of at least one standard of care treatment (CCB or iloprost) for at least three months 4. RCS \> 4 5. Possibility to obtain venous access (either through a peripherally or centrally inserted catheter)
Exclusion criteria
1. Significant anemia (\<8 g/dL) 2. Clinically relevant hemorrhagic diathesis or coagulopathy 3. Diabetes mellitus 4. Serious acute or chronic kidney (eGFR\<30 ml/min/1.73m2) or liver failure 5. Hypotension with systolic blood pressure \<100 mmHg 6. Chronic viral infections (HIV, Hepatitis B, C) 7. Epilepsia, psychosis, dementia, or other relevant neurologic condition precluding the conduct of plasmapheresis 8. Malignant disease or any other condition with life expectancy \<12 months 9. Known history of alcohol or drug abuse 10. Long-term serious tobacco abuse with documented severe vascular disease (Fontaine \>III). 11. Severe hyperlipoproteinemia, defined as a significant elevation of Lp(a) or LDL cholesterol despite standard doses of medical therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Raynaud Condition Score (RCS) | 24 weeks | changes of the Raynaud Condition after treatment, higher RCS denotes worse clinical findings |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to healing of existing digital ulcers | 24 weeks | Time to healing of existing digital ulcers |
| Scleroderma Health Assessment Questionnaire | 24 weeks | Changes in the SHAQ with treatment; higher scores mean better functional status |
| Development of new digital ulcers | 24 weeks | To assess the number of new digital ulcers with treatment |
| Quick DASH | 24 weeks | changes in the Quick DASH with treatment; lower scores mean better functional status |
| Nailfold video capillaroscopy changes | 24 weeks | changes in in NVC assessments with treatment |
| Whole blood viscosity | 24 weeks | changes in Whole blood viscosity with treatment |
| Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score | 24 weeks | changes in the FACIT-Fatigue score with treatment; higher scores indicate a better clinical status |
Other
| Measure | Time frame | Description |
|---|---|---|
| Exploratory objectives | 24 weeks | changes in miRNA |
Countries
Germany
Contacts
Primary ContactPeter Korsten, Dr. med.
Outcome results
None listed