Mesenchymal Stromal Cells as Treatment for Digital Ulcers in Systemic Sclerosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorUMC Utrecht
About this trial
The MANUS Trial aims to examine the safety, feasibility and potential efficacy of intramuscularly injected allogeneic mesenchymal stromal cells as treatment for digital ulcers of systemic sclerosis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Established diagnosis of SSc according to the 2013 ACR/EULAR criteria
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) within 1 month after prostacyclins iv
Disqualifiers
Ulcer with underlying calcinosis (ruled out by X-ray prior to screening/inclusion)
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
Trial design
Design model
Parallel
Treatments tested in this trial
Mesenchymal stromal cells
Drug8 intramuscular injections at designated sites in the hand/forearm muscles of the most affected side. Blinded syringes will be used. Injections will be administered by an experienced clinician (plastic surgeon or hand surgeon).
Placebo
Other intervention8 intramuscular injections at designated sites in the hand/forearm muscles of the most affected side. Blinded syringes will be used. Injections will be administered by an experienced clinician (plastic surgeon or hand surgeon).
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: MSC injectionsExperimental treatment 1 intervention
Group B: Placebo injectionsPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Toxicity of the treatment
Toxicity of the treatment is defined as 1. Local toxicity, including signs of local inflammation (swelling, warmth, impairment of function), worsening of ulcers or new ulcers or hematomas after MSC administration 2. Other adverse events, graded according to the Common Terminology Criteria for Adverse Events version 4.0, expressed as maximum grade toxicity per organ system.
Time frame
12 weeks after MSC administrationSecondary outcomes
1
Serious adverse events
Any treatment-related serious adverse events (SAE) defined as events leading to hospitalization, death, or persistent or significant disability. To establish the presence or absence of a causal relationship, the World Health Organisation guidelines for pharmacovigilance will be followed.
Time frame
48 hours, 2, 4, 8, 12, 24 weeks and 52 weeks after MSC administration2
Change in perceived pain based on the Numerical Rating Scale
Change in pain as assessed using the Numerical Rating Scale,
Time frame
48 hours, 2, 4, 8, 12, 24 weeks and 52 weeks after MSC administration3
Change in perceived pain based on the digital ulcer visual analogue scale (part of the S-HAQ)
Change in pain as assessed using the digital ulcer visual analogue scale (part of the S-HAQ).
Time frame
48 hours, 2, 4, 8, 12, 24 weeks and 52 weeks after MSC administration4
Change in perceived pain based on the pain VAS ( part of the S-HAQ)
Change in pain as assessed using the pain VAS (S-HAQ), use of analgesics.
Time frame
48 hours, 2, 4, 8, 12, 24 weeks and 52 weeks after MSC administrationSponsors and contacts
Click on the lead sponsor to view all of their trials.
UMC Utrecht
Lead sponsor
ZonMw: The Netherlands Organisation for Health Research and Development
Collaborator
Source ClinicalTrials.gov