HUMAN MESENCHYMAL STEM CELLS for Systemic sclerosis | Diffuse systemic sclerosis

Inclusion criteria

• {"criterion_text":"- Age >18 years\n- Established diagnosis of SSc according to criteria of the American College of Rheumatology (2013)\n- At least one active digital ulcer (painful area, >2 mm in diameter with visible depth and loss of dermis) refractory to 5 days intravenous prostacyclines\n- informed consent"}

Exclusion criteria

• {"criterion_text":"- Ulcer with underlying calcinosis (ruled out by X-ray prior to screening/inclusion)\n- History of neoplasm or malignancy in the past 10 years\n- Pregnancy or unwillingness to use adequate contraception during study\n- Serious known concomitant disease with life expectancy <1 year\n- Uncontrolled hypertension\n- Uncontrolled acute or chronic infection\n- follow-up impossible"}

Primary endpoints

• {"endpoint_text":"- The primary outcome is the toxicity of the treatment at 12 weeks after MSC administration, defined as -1. Local toxicity, including signs of local inflammation (swelling, warmth, impairment of function), worsening of ulcers or new ulcers or hematomes after MSC administration - 2. Other adverse events, graded according to the Common Terminology Criteria for Adverse Events.","definition_or_measurement_approach":"Toxicity assessed at 12 weeks after MSC administration and defined as: 1) Local toxicity including signs of local inflammation (swelling, warmth, impairment of function), worsening of ulcers or new ulcers or hematomes after MSC administration; 2) Other adverse events graded according to the Common Terminology Criteria for Adverse Events (CTCAE)."}

Secondary endpoints

• {"endpoint_text":"- A secondary outcome measure for safety is the incidence (at 12 weeks post treatment) of any treatment-related serious adverse events (SAE) defined as events leading to hospitalization, death, or persistent or significant disability","definition_or_measurement_approach":"Incidence at 12 weeks post treatment of treatment-related SAEs defined as events leading to hospitalization, death, or persistent/significant disability."}
• {"endpoint_text":"- Change in pain as assessed using the Numerical Rating Scale, the digital ulcer visual analogue scale (part of the S-HAQ), pain VAS (S-HAQ), use of analgesics.","definition_or_measurement_approach":"Change in pain measured by Numerical Rating Scale, digital ulcer visual analogue scale (part of S-HAQ), pain VAS (S-HAQ), and analgesic use."}
• {"endpoint_text":"- Quality of life and disability as assessed with the HAQ-disability index, and the SF-36, EuroQol (EQ-5D) questionnaires","definition_or_measurement_approach":"Quality of life and disability assessed via HAQ-disability index, SF-36, and EuroQol (EQ-5D) questionnaires."}
• {"endpoint_text":"- Hand function as assessed with the Cochin Hand Function Scale.","definition_or_measurement_approach":"Hand function measured using the Cochin Hand Function Scale."}
• {"endpoint_text":"- Number of digital ulcers 12 weeks post treatment","definition_or_measurement_approach":"Count of digital ulcers at 12 weeks post treatment."}
• {"endpoint_text":"- Change in the number of active tip digital ulcers on the volar aspect","definition_or_measurement_approach":"Change in number of active tip digital ulcers on the volar aspect (count comparison)."}
• {"endpoint_text":"- Need to alter medication regime as determined by the patient’s own rheumatologist","definition_or_measurement_approach":"Recorded need to alter medication regimen as determined by the patient's treating rheumatologist."}
• {"endpoint_text":"- The severity of scleroderma as assessed with the Modified Rodnan skin score 79 and the Scleroderma Health Assessment Questionnaire (S-HAQ)","definition_or_measurement_approach":"Severity assessed with Modified Rodnan Skin Score (MRSS) and Scleroderma Health Assessment Questionnaire (S-HAQ)."}
• {"endpoint_text":"- Number and severity of Raynaud’s symptoms, as assessed using the Raynaud Condition Score","definition_or_measurement_approach":"Number and severity of Raynaud's symptoms assessed with the Raynaud Condition Score."}
• {"endpoint_text":"- Changes in capillary morphology and architecture, as visualized with video-assisted nailfold capillaroscopy by a trained investigator","definition_or_measurement_approach":"Capillary morphology changes visualized by video-assisted nailfold capillaroscopy performed by a trained investigator."}
• {"endpoint_text":"- Changes in laboratory parameters","definition_or_measurement_approach":"Changes in laboratory parameters (unspecified panels) compared over time."}

Netherlands

Earliest CTIS Part Ii Submission Date

25-10-2024

Latest Decision Or Authorization Date

29-10-2024

Processing Time Days

4

Number Of Sites

1

Number Of Participants

20

Primary sponsor

Full Name

Universitair Medisch Centrum Utrecht

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands

Third parties

• {"country":"","full_name":"ZonMW, the Netherlands","duties_or_roles":"Source of monetary support","organisation_type":""}