Systemic Sclerosis PulmonaryPhase 1/2NCT07542067
Infusion of Allogeneic Stromal Mesenchymal Stem Cells From Wharton´s Jelly in Patients With Diffuse Cutaneous Systemic Sclerosis With Refractory Pulmonary Involvement to Treatment
A Phase 1/2 study testing Mesenchymal Stem Cells from Wharton´s jelly in people with Systemic Sclerosis Pulmonary and Pulmonary Hypertension, run by Fundación Neumologica Colombiana at 1 site in Colombia.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Sclerosis Pulmonary, Pulmonary Hypertension, Pulmonary Fibrosis
- What is being tested?
- Mesenchymal Stem Cells from Wharton´s jelly
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteColombia
- Study dates
- 15 May 2024to 11 Aug 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Pregnancy or absence of appropriate contraception throughout the study.
- Pulmonary artery systolic pressure (PASP) >75mmHg (on echocardiography or after right heart catheterization);
- Theorical DLCO <30%
- Calculated creatinine clearance <30 ml/mn/m2
- Clinical sign of a congestive heart failure refractory ;
- Left ventricular ejection fraction <35% at myocardial scintigraphy or echocardiography;
Show all 18Show fewer
- Chronic atrial fibrillation requiring oral anticoagulant therapy;
- Uncontrolled ventricular arrhythmia;
- Pericardial effusion with hemodynamic compromise assessed by echocardiography.
- Hepatic impairment defined as a persistent increase in transaminases or bilirubin to 3 times normal.
- Psychiatric disorders, including drug taking and alcohol abuse.
- Active neoplasia or concomitant myelodysplasia, antecedent of neoplasia.
- 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.
- 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.
- Risk of poor patient compliance
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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?
Read the detailed description
This study aims to evaluate the therapeutic effects of allogeneic mesenchymal stromal cell infusion as a treatment in patients with systemic sclerosis refractory to conventional therapy. The study population will be selected from the database of families affiliated with Stem Regenerative Medicine according to the inclusion and exclusion criteria and verified by the academic committee.
Administration will be performed intravenously, at a concentration of 2 × 10⁶ mesenchymal cells per kilogram of patient body weight. Infusions will be scheduled in conjunction with cyclophosphamide treatment cycles, ten days after each administration within the cyclophosphamide regimen for each patient.
To assess safety and therapeutic effects, the occurrence of any adverse event will be documented from the start of infusion until completion of the trial at six months. To evaluate treatment response, assessments will be conducted pre-infusion and at six months post-infusion, including clinical variables, paraclinical parameters, and hemodynamic tests. Skin involvement will be evaluated using the modified Rodnan score, along with assessment of nailfold capillaroscopy, pulmonary function, and structural involvement by high-resolution chest tomography (HRCT), diffusion capacity for carbon monoxide (DLCO), and the 6-minute walk test.
As part of cardiovascular assessment, brain natriuretic peptide (BNP) levels and transthoracic echocardiography will be performed. The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) and SYSQ will be used as assessment tools for pulmonary hypertension. A comparison of these parameters before initiation of therapy and after completion of 24 weeks of the infusion protocol will be conducted.
- Being tested
- Mesenchymal Stem Cells from Wharton´s jelly
- Keywords
- refractory systemic sclerosis, systemic sclerosis, pulmonary hypertension, mesenchymal stem cells
- Medical subject headings
- Hypertension, Pulmonary, Pulmonary Fibrosis, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mesenchymal Stem Cells from Wharton´s jellyBiological
intravenous infusion of Mesenchymal Stem Cells from Wharton´s jelly
| Study group | Role | What they receive |
|---|---|---|
| Historical control | Other | A historical control similar characteristics will be selected to compare to the treated patients. Receives: Biological: Mesenchymal Stem Cells from Wharton´s jelly |
What is being measured
The primary outcome is the main question the study is designed to answer.
pulmonary function
forced vital capacity measured by forced spirometry.
Measured: before and after 6 months treatment
pulmonary function
forced expiratory volume in one second. measured by forced spirometry.
Measured: before and after 6 months treatment
lung capacity for gas transfer
DLCO: diffusing capacity for carbon monoxide
Measured: before and after 6 months treatment
submaximal excersice capacity
Submaximal exercise capacity will be assessed using the 6-minute walk test (6MWT), performed according to American Thoracic Society (ATS) guidelines. The test measures the total distance walked in six minutes on a flat surface and is expressed in meters. This assessment is used to quantify functional limitation and prognosis associated with pulmonary hypertension. The primary outcome will be the change in distance walked between baseline and 6 months. A clinically significant improvement is defined as an increase of more than 10% from the baseline distance.
Measured: before and after 6 months treatment
cardiopulmonary hemodinamycs
Pulmonary vascular resistance (PVR) will be measured by right heart catheterization and expressed in Wood units. This parameter is used to assess pulmonary vascular involvement in pulmonary hypertension, previously confirmed by standard hemodynamic criteria. The outcome will be the change in PVR between baseline and 6 months after intervention.
Measured: before and after 6 months treatment
cardiopulmonary hemodynamics
Systolic pulmonary artery pressure (sPAP) will be measured by right heart catheterization and expressed in mmHg. This parameter is used to assess the severity of pulmonary hypertension. Measurements will be obtained prior to mesenchymal stromal cell infusion and repeated at 6 months after intervention. The outcome will be the change in sPAP between baseline and 6 months.
Measured: before and after 6 months treatment
cardiopulmonary hemodinamycs
diastolic pulmonary artery pressure
Measured: before and after 6 months treatment
cardiopulmonary hemodinamycs
right and left ventricle ejection fraction.
Measured: before and after 6 months treatment
quality of life and functional status
CAMPHOR score
Measured: before and after 6 months treatment
quality of life and functional status
Ssq
Measured: before and after 6 months treatment
parenchymatous pulmonary compromise
high resolution chest CT
Measured: before and after 6 months treatment
Secondary outcomes (6)
cutaneous fibrosis
RODNAN score
Measured: before and after 6 months treatment
adverse events and security profile
Serum creatinine levels will be measured using standard laboratory methods and expressed in mg/dL. This parameter is used to assess renal function. The outcome will be the change in serum creatinine levels between baseline and 6 months after intervention.
Measured: before and after 6 months treatment
adverse events and security profile
blood cells counts
Measured: before and after 6 months treatment
adverse events and security profile
urinalysis
Measured: before and after 6 months treatment
adverse events and security profile
BUN
Measured: before and after 6 months treatment
adverse events and security profile
transaminases
Measured: before and after 6 months treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2026
- Study start15 May 2024
- Primary completion30 Jun 2025
- Study completion11 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Sclerosis PulmonaryStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The administration of mesenchymal stem cells will be carried out intravenously at a concentration of 2 X 10\^6 mesenchymal cells per kilogram of patient weight. The infusions will be carried out nested at cyclophosphamide application cycles. They will be carried out ten days after each application of the cyclophosphamide schedule for each patient.
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundación Neumologica Colombiana
- Sponsor type
- University, hospital or other
- Collaborators
- CryoHoldco LATAM, Universidad de la Sabana
- Responsible party
- Sponsor
- Investigators
- John Londono, MD,PhD, Principal investigator, Universidad de la Sabana
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07542067
- Sponsor's protocol ID
- 80-14-10-2019
- Status last verified
- Apr 2026
- First submitted
- 20 May 2025
- Last updated
- 21 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Infusion of Allogeneic Stromal Mesenchymal Stem Cells From Wharton´s Jelly in Patients With Diffuse Cutaneous Systemic Sclerosis With Refractory Pulmonary Involvement to Treatment
- ClinicalTrials.gov ID
- NCT07542067
- Acronym / sponsor ID
- 80-14-10-2019
- Keywords
- refractory systemic sclerosis, systemic sclerosis, pulmonary hypertension, mesenchymal stem cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Fundación Neumologica ColombianaUniversity, hospital or other
- Collaborators
- CryoHoldco LATAM, Universidad de la Sabana
- Study start
- 15 May 2024 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 11 Aug 2025 (actual)
- First posted
- 21 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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?
Detailed description
This study aims to evaluate the therapeutic effects of allogeneic mesenchymal stromal cell infusion as a treatment in patients with systemic sclerosis refractory to conventional therapy. The study population will be selected from the database of families affiliated with Stem Regenerative Medicine according to the inclusion and exclusion criteria and verified by the academic committee.
Administration will be performed intravenously, at a concentration of 2 × 10⁶ mesenchymal cells per kilogram of patient body weight. Infusions will be scheduled in conjunction with cyclophosphamide treatment cycles, ten days after each administration within the cyclophosphamide regimen for each patient.
To assess safety and therapeutic effects, the occurrence of any adverse event will be documented from the start of infusion until completion of the trial at six months. To evaluate treatment response, assessments will be conducted pre-infusion and at six months post-infusion, including clinical variables, paraclinical parameters, and hemodynamic tests. Skin involvement will be evaluated using the modified Rodnan score, along with assessment of nailfold capillaroscopy, pulmonary function, and structural involvement by high-resolution chest tomography (HRCT), diffusion capacity for carbon monoxide (DLCO), and the 6-minute walk test.
As part of cardiovascular assessment, brain natriuretic peptide (BNP) levels and transthoracic echocardiography will be performed. The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) and SYSQ will be used as assessment tools for pulmonary hypertension. A comparison of these parameters before initiation of therapy and after completion of 24 weeks of the infusion protocol will be conducted.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The administration of mesenchymal stem cells will be carried out intravenously at a concentration of 2 X 10^6 mesenchymal cells per kilogram of patient weight. The infusions will be carried out nested at cyclophosphamide application cycles. They will be carried out ten days after each application of the cyclophosphamide schedule for each patient.
| Arm | Type | Receives |
|---|---|---|
| Historical control | Other | Biological: Mesenchymal Stem Cells from Wharton´s jellyA historical control similar characteristics will be selected to compare to the treated patients. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (18 listed)
- Pregnancy or absence of appropriate contraception throughout the study.
- Pulmonary artery systolic pressure (PASP) >75mmHg (on echocardiography or after right heart catheterization);
- Calculated creatinine clearance <30 ml/mn/m2
- Clinical sign of a congestive heart failure refractory ;
- Left ventricular ejection fraction <35% at myocardial scintigraphy or echocardiography;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mesenchymal Stem Cells from Wharton´s jellyintravenous infusion of Mesenchymal Stem Cells from Wharton´s jelly | Biological | Historical control |
Primary outcome
pulmonary function
Time frame: before and after 6 months treatment
forced vital capacity measured by forced spirometry.
pulmonary function
Time frame: before and after 6 months treatment
forced expiratory volume in one second. measured by forced spirometry.
lung capacity for gas transfer
Time frame: before and after 6 months treatment
DLCO: diffusing capacity for carbon monoxide
submaximal excersice capacity
Time frame: before and after 6 months treatment
Submaximal exercise capacity will be assessed using the 6-minute walk test (6MWT), performed according to American Thoracic Society (ATS) guidelines. The test measures the total distance walked in six minutes on a flat surface and is expressed in meters. This assessment is used to quantify functional limitation and prognosis associated with pulmonary hypertension. The primary outcome will be the change in distance walked between baseline and 6 months. A clinically significant improvement is defined as an increase of more than 10% from the baseline distance.
cardiopulmonary hemodinamycs
Time frame: before and after 6 months treatment
Pulmonary vascular resistance (PVR) will be measured by right heart catheterization and expressed in Wood units. This parameter is used to assess pulmonary vascular involvement in pulmonary hypertension, previously confirmed by standard hemodynamic criteria. The outcome will be the change in PVR between baseline and 6 months after intervention.
cardiopulmonary hemodynamics
Time frame: before and after 6 months treatment
Systolic pulmonary artery pressure (sPAP) will be measured by right heart catheterization and expressed in mmHg. This parameter is used to assess the severity of pulmonary hypertension. Measurements will be obtained prior to mesenchymal stromal cell infusion and repeated at 6 months after intervention. The outcome will be the change in sPAP between baseline and 6 months.
cardiopulmonary hemodinamycs
Time frame: before and after 6 months treatment
diastolic pulmonary artery pressure
cardiopulmonary hemodinamycs
Time frame: before and after 6 months treatment
right and left ventricle ejection fraction.
quality of life and functional status
Time frame: before and after 6 months treatment
CAMPHOR score
quality of life and functional status
Time frame: before and after 6 months treatment
Ssq
parenchymatous pulmonary compromise
Time frame: before and after 6 months treatment
high resolution chest CT
6 secondary outcomes
cutaneous fibrosis
Time frame: before and after 6 months treatment
adverse events and security profile
Time frame: before and after 6 months treatment
adverse events and security profile
Time frame: before and after 6 months treatment
adverse events and security profile
Time frame: before and after 6 months treatment
adverse events and security profile
Time frame: before and after 6 months treatment
adverse events and security profile
Time frame: before and after 6 months treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Londono, MD,PhD principal investigator
Universidad de la Sabana
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Mesenchymal Stem Cells from Wharton´s jelly in people with Systemic Sclerosis Pulmonary and Pulmonary Hypertension, run by Fundación Neumologica Colombiana at 1 site in Colombia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Colombia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
pulmonary function (time frame: before and after 6 months treatment). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2026.