systemic sclerosis MedDRA version: 18.0 Level: PT Classification code 10042953 Term: Systemic sclerosis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Conditions
Interventions
Product Name: Mesenchymal stem cells
Product Code: MSC
Pharmaceutical Form: Suspension and solvent for suspension for injection
INN or Proposed INN: EX VIVO CULTURED HUMAN MESENCHYMAL STEM CELLS
Othe
Sponsors
University Medical Center Utrecht
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age >18 years - Established diagnosis of SSc according to criteria of the American College of Rheumatology (2013) - At least one active digital ulcer (painful area, >2 mm in diameter with visible depth and loss of dermis) refractory to intravenous prostacyclins ------------‘Refractory to prostacyclins’ is defined as ? -Worsening of ulcer(s) at 1 month after prostacyclins iv ? -No improvement of ulcer(s) at 2 months after prostacyclins iv, as judged by the referring physician ? -Recurrence of exactly the same ulcer(s) (same location) at 3 months after prostacyclins iv - Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Ulcer with underlying calcinosis (ruled out by X-ray prior to referral) -History of neoplasm or malignancy in the past 10 years - Pregnancy or unwillingness to use adequate contraception during study - Serious known concomitant disease with life expectancy <1 year - Uncontrolled hypertension - Uncontrolled acute or chronic infection - Follow-up impossible.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Determining the safety and efficacy of intramuscularly administered mesenchymal stem cells for the treatment of digital ucers in systemic sclerosis.;Secondary Objective: To assess the accuracy of various parameters related to inflammation, endothelial activation and angiogenesis to predict therapeutic efficacy and/or serve as biomarkers ;Primary end point(s): Number of serious adverse events ;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety: - treatment related toxicity as assessed with the WHO parameters Efficacy: -ulcer count, ulcer area -ulcer VAS -quality of life as determined with the S-HAQ, SF-36, EQ5D, CHFS -capillaroscopy semi-quantitative score - change in biomarkers ;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Countries
Netherlands
Contacts
Public ContactPrincipal investigator
University Medical Center Utrecht
Outcome results
None listed