Mesenchymal stem cells as treatment for non healing wounds on the fingers in patients with systemic sclerosis

• Register: EUCTR
• Last refreshed on: 5 June 2017
• Main ID: EUCTR2015-000168-32-NL
• Date of registration: 09/07/2015
• Prospective Registration: Yes
• Primary sponsor: University Medical Center Utrecht
• Public title: Mesenchymal stem cells as treatment for non healing wounds on the fingers in patients with systemic sclerosis
• Scientific title: Mesenchymal stem cells for Angiogenesis and Neovascularization in digital Ulcers of Systemic sclerosis - MANUS Trial
• Date of first enrolment: 13/07/2016
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000168-32
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Contacts

• Name: Principal investigator
• Affiliation: University Medical Center Utrecht

• Name: Principal investigator
• Affiliation: University Medical Center Utrecht

Key inclusion & exclusion 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:
- 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.





Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

systemic sclerosis
MedDRA version: 18.0 Level: PT Classification code 10042953 Term: Systemic sclerosis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

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
Other descriptive name: EX VIVO CULTURED HUMAN MESENCHYMAL STEM CELLS
Concentration unit: CFU/ml colony forming unit(s)/millilitre
Concentration type: up to
Concentration number: 50000000-
Pharmaceutical form of the placebo: Suspension and solvent for suspension for injection
Route of administration of the placebo: Intramuscular use

Primary Outcome(s)

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

Main Objective: Determining the safety and efficacy of intramuscularly administered mesenchymal stem cells for the treatment of digital ucers in systemic sclerosis.

Secondary Outcome(s)

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

Secondary ID(s)

NL51705.000.15

Source(s) of Monetary Support

ZonMW