NCT07542067Befejeződött1/2. fázis
Infusion of Allogeneic Stromal Mesenchymal Stem Cells From Wharton´s Jelly in Patients With Diffuse Cutaneous Systemic Sclerosis With Refractory Pulmonary Involvement to Treatment
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2026. ápr. 21.
Rövid összefoglaló (regiszter, angolul)
Progressive SSc is an entity with limited therapeutic alternatives and a survival rate of less than 45% within the first 3 to 5 years. The disease causes severe limitations in quality of life, ranging from functional impairment to depression. Up to 20% of patients become refractory to conventional treatment with disease-modifying anti-rheumatic drugs (DMARDs) and cyclophosphamide therapy. This condition favors progression to visceral involvement, including gastrointestinal, pulmonary, and pulmonary hypertension manifestations. The latter, considered a poor prognostic factor, increases mortality in this patient population and drastically affects quality of life. For this reason, different therapeutic options have been considered, including cell transplantation and stem cell use. Among the options studied to date are stromal mesenchymal cells derived from Wharton's jelly. These cells have been administered via intravenous infusion or direct application in various disease scenarios, ranging from vascular involvement to interstitial lung disease and pulmonary hypertension, with promising results in terms of clinical progression, quality of life improvement, and prognostic indices. This therapy has demonstrated a favorable safety profile at the time of administration and a low rate of adverse events, with self-limiting fever being the most frequent event. Based on the above and considering the possibility of offering patients without therapeutic alternatives for the disease, in addition to palliative options, an intravenous infusion of stromal mesenchymal stem cells derived from Wharton's jelly is proposed for three patients with progressive SSc refractory to conventional therapy, with pulmonary involvement due to pulmonary hypertension. Under this premise, the research question posed in this study is: What are the effects of the infusion of allogeneic mesenchymal stromal cells derived from Wharton's jelly in patients with systemic sclerosis refractory to conventional treatment with methotrexate or cyclophosphamide, in a population of three patients with severe pulmonary involvement due to pulmonary hypertension?
- Állapot (regiszter)
- Befejeződött
- Fázis (regiszter)
- 1/2. fázis
- Vizsgálattípus
- Beavatkozással járó
- Résztvevők száma
- 1 (tényleges)
- Első benyújtás
- 2025. máj. 20.
- Kezdés
- 2024. máj. 15.
- Elsődleges befejezés
- 2025. jún. 30.
- Fő szponzor
- Fundación Neumologica Colombiana
- NCT-szám
- NCT07542067
Mit vállalsz
- Vizsgálat típusa: Beavatkozással járó · 1/2. fázis
- Bevont résztvevők: 1
- 2024. máj. 15. és az elsődleges befejezés (2025. jún. 30.) között
- 1 helyszín, 1 országban
- Vizitek és vizsgálatok: kérdezd a vizsgálat csapatát, a regiszter nem sorolja fel őket
A(z) ClinicalTrials.gov regisztermezőiből összeállítva. A vizsgálat felépítését írja le.
Ki nem vehet részt
Szó szerint a(z) ClinicalTrials.gov forrásból, témák szerint csoportosítva. Hogy ki vehet részt, azt csak a vizsgálat csapata dönti el.
Korábbi kezelés
- Chronic atrial fibrillation requiring oral anticoagulant therapy;
- Active neoplasia or concomitant myelodysplasia, antecedent of neoplasia.
Más betegség
- Pulmonary artery systolic pressure (PASP) >75mmHg (on echocardiography or after right heart catheterization);
- Theorical DLCO <30%
- Clinical sign of a congestive heart failure refractory ;
- Uncontrolled ventricular arrhythmia;
- Psychiatric disorders, including drug taking and alcohol abuse.
- Uncontrolled systemic hypertension.
- Uncontrolled acute or chronic infection, HIV1, 2 or HTLV-1, 2seropositivity.
- Chronic hepatitis B or C active.
- Significant exposure to bleomycin, toxic oils, vinyl chloride, trichloroethylene or silica; eosinophilia-myalgia syndrome, eosinophilia fasciitis.
Laborértékek
- Calculated creatinine clearance <30 ml/mn/m2
- Left ventricular ejection fraction <35% at myocardial scintigraphy or echocardiography;
- Hepatic impairment defined as a persistent increase in transaminases or bilirubin to 3 times normal.
- Bone marrow failure defined by neutropenia <0.5 x 109 / L, thrombocytopenia <50 x 109 / L, anemia <8 g / dL, CD4 lymphopenia <200 x 106 / L.
Egyéb
- Pregnancy or absence of appropriate contraception throughout the study.
- Pericardial effusion with hemodynamic compromise assessed by echocardiography.
- Risk of poor patient compliance
Kiket keres a vizsgálat
- Age> 18 years and <65 years.
- Established diagnosis of systemic sclerosis according to the criteria of the American College of Rheumatology
- SSc of poor prognosis, involving life-threatening severe visceral involvement (cardiac or pulmonary hypertension ), lack of response to conventional immunosuppressive therapy used in severe forms of the disease according to the European recommendations of EUSTAR and EBMT, relying on high doses of IV cyclophosphamide (either in monthly bolus for at least six months); or SSc with life-threatening pulmonary hypertension. Patients may or may not have pulmonary fibrosis.
- Signed informed consent.
- Presence of a consenting MSC donor
- Affiliation to social security.
Életkor a regiszterben: 18 év – 65 év · Nem: nők és férfiak · Egészséges önkénteseket nem fogad
Helyszínek (1)
Helyszínek a regiszter sorrendjében. Ha a profilodba mented a városodat, távolság szerint rendeződnek.
| Helyszín | Város | Ország | Helyszín állapota |
|---|---|---|---|
| Clinica Universidad de La Sabana | Bogotá | Kolumbia | Ismeretlen állapot |
Ez az oldal tájékoztatás, nem orvosi tanács. Bármely vizsgálatról az orvosoddal beszélj.