SclerodermaPhase 2AST-MOMANCT01895244
Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis
A Phase 2 study testing Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells in people with Scleroderma and Cardiac Involvement, run by University Hospital Tuebingen at 1 site in Germany.
Trial summary
Autologous stem cell therapy has been shown to be effective in patients with systemic sclerosis. Nevertheless treatment is associated with treatment related mortality and patients die during follow up despite successful transplantation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Scleroderma, Cardiac Involvement, Autologous Stem Cell Transplantation
- What is being tested?
- Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteGermany
- Study dates
- Sep 2012to Jun 2024
- Phase
- Phase 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
- Diagnosis of progressive systemic sclerosis <7 years
- Progressive course despite cyclophosphamide pretreatment
- Cyclophosphamide i.v.: at least 3 x with 500-1000 mg/m² every 3-4 weeks or
- Cyclophosphamide p.o. with at least 100mg/day for at least 2 months or
- Contraindication to treatment with cyclophosphamide
- Progress defined as at least one of the following criteria:
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- Increase in the mRSS
- Worsening of the lung function
- Increase in fibrosis/alveolitis in thorax CT
- Worsening kidney function through manifestation of systemic sclerosis
- Limited or diffuse cutaneous progressive form of Ssc with organ manifestation in the lungs/heart or kidneys
Who cannot join
- Age <18 years
- Pregnancy or inadequate contraception
- Severe heart failure with ejection fraction (EF) < 30% in echo
- Pulmonary arterial hypertension with systolic pulmonary arterial pressure (PAPsys) >50mm Hg
- Kidney insufficiency: creatinine clearance <30 ml/min
- Reduced lung function
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- Inspiratory vital capacity (IVC) < 50% of normal
- Carbon monoxide (CO)-Diffusion capacity SB < 40%
- Previously damaged bone marrow
- Leukopenia < 2,000/µl
- Thrombopenia < 100,000/µl
- Previous myelotoxic treatment:
- Cyclophosphamide > 50g cumulative (relative)
- Infection (Hepatitis B/C, HIV, Salmonella carrier, syphilis, relative: history of tuberculosis)
- Severe concomitant psychiatric illness (depression, psychosis)
- Substance dependence
- Continued nicotine abuse
- Continued alcohol abuse
- Continued drug abuse
- Consent not given
- Poor compliance
Trial locations and contacts
1 site in 1 country.
Germany1 site
University Hospital Tuebingen; Department of oncology, hematology, rheumatology, immunology and pulmology
Tübingen, 72076
About this study
Official title: Highdose Chemotherapy and Transplantation of 34+ Selected Stem Cell for Progressive Systemic Sclerosis - Modification According to Manifestation
Autologous stem cell therapy has been shown to be effective in patients with systemic sclerosis. Nevertheless treatment is associated with treatment related mortality and patients die during follow up despite successful transplantation.
Intention of this trial is to improve overall survival by modifying the existing protocol used for the ASTIS trial.
To reduce treatment toxicity we reduce the dose of Cyclophosphamide (CYC) for mobilisation to 2x1g.
Especially in patients with cardiac manifestations we also modify the conditioning regimen by adding thiotepa and reducing CYC; as CYC has known cardiotoxic side effects.
- Being tested
- Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
- Keywords
- scleroderma, autologous stem cell transplantation, thiotepa, cluster of differentiation (CD) 34 selection
- Medical subject headings
- Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cellsDrug
If no active alveolitis: mobilisation with 2x1g Cyclophosphamide If active alveolitis: mobilisation with 2x1.5g Cyclophosphamid If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG
| Study group | Role | What they receive |
|---|---|---|
| Conditioning with CYC/ antithymocyte globulin (ATG) | Experimental | Each patient receives stem cell transplantation open label with cluster of differentiation (CD)34 selected stem cells mobilisation and conditioning depending on manifestation If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG Receives: Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells |
| Conditioning with CYC/Thiotepa/ATG | Experimental | In patients with cardiac manifestations as defined in the protocol the conditioning for stem cell transplantation is changed to Cyclophosphamide (CYC), thiotepa and ATG Receives: Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficay - Overall survival
Number of patients that are alive after 3 years
Measured: 3 years
Secondary outcomes (4)
Safety - Treatment related mortality
Treatment related mortality: number of patients who die during the first 100 days after transplantation
Measured: 100 days
Time to engraftment
Time in days from day 0 to platelet count > 20.000 and granulocytes >500/µl
Measured: 2 months
Progression free survival
Time after transplantation without symptoms of disease activity
Measured: 3 years
Efficacy - Lung function test and Skin
Number of patients that achieve either improvement of >25% in mRSS or > 10% in FVC or DLCO
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2013 (estimated)
- Study startSep 2012
- Primary completionSep 2021
- Study completionJun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SclerodermaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Joerg Henes, University Hospital Tuebingen
- Investigators
- Joerg C Henes, MD, Principal investigator, University Hospital Tuebingen, Department of oncology, hematology, rheumatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01895244
- Sponsor's protocol ID
- AST MOMA
- Acronym
- AST-MOMA
- Status last verified
- Jul 2024
- First submitted
- 1 Jul 2013
- Last updated
- 29 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Henes JC, Koetter I, Horger M, Schmalzing M, Mueller K, Eick C, Bauer A, Vogel W, Kanz L. Autologous stem cell transplantation with thiotepa-based conditioning in patients with systemic sclerosis and cardiac manifestations. Rheumatology (Oxford). 2014 May;53(5):919-22. doi: 10.1093/rheumatology/ket464. Epub 2014 Jan 22.
PubMed 24459219
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Highdose Chemotherapy and Transplantation of 34+ Selected Stem Cell for Progressive Systemic Sclerosis - Modification According to Manifestation
- ClinicalTrials.gov ID
- NCT01895244
- Acronym / sponsor ID
- AST-MOMA / AST MOMA
- Keywords
- scleroderma, autologous stem cell transplantation, thiotepa, cluster of differentiation (CD) 34 selection
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- Sep 2012 (actual)
- Primary completion
- Sep 2021 (actual)
- Study completion
- Jun 2024 (actual)
- First posted
- 10 Jul 2013 (estimated)
- 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.
Autologous stem cell therapy has been shown to be effective in patients with systemic sclerosis. Nevertheless treatment is associated with treatment related mortality and patients die during follow up despite successful transplantation.
Intention of this trial is to improve overall survival by modifying the existing protocol used for the ASTIS trial.
To reduce treatment toxicity we reduce the dose of Cyclophosphamide (CYC) for mobilisation to 2x1g.
Especially in patients with cardiac manifestations we also modify the conditioning regimen by adding thiotepa and reducing CYC; as CYC has known cardiotoxic side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Conditioning with CYC/ antithymocyte globulin (ATG) | Experimental | Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cellsEach patient receives stem cell transplantation open label with cluster of differentiation (CD)34 selected stem cells mobilisation and conditioning depending on manifestation If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG |
| Conditioning with CYC/Thiotepa/ATG | Experimental | Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cellsIn patients with cardiac manifestations as defined in the protocol the conditioning for stem cell transplantation is changed to Cyclophosphamide (CYC), thiotepa and ATG |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis of progressive systemic sclerosis <7 years
- Progressive course despite cyclophosphamide pretreatment
- Cyclophosphamide i.v.: at least 3 x with 500-1000 mg/m² every 3-4 weeks or
- Cyclophosphamide p.o. with at least 100mg/day for at least 2 months or
- Contraindication to treatment with cyclophosphamide
Key exclusion criteria (21 listed)
- Age <18 years
- Pregnancy or inadequate contraception
- Severe heart failure with ejection fraction (EF) < 30% in echo
- Pulmonary arterial hypertension with systolic pulmonary arterial pressure (PAPsys) >50mm Hg
- Kidney insufficiency: creatinine clearance <30 ml/min
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 |
|---|---|---|
| Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cellsIf no active alveolitis: mobilisation with 2x1g Cyclophosphamide If active alveolitis: mobilisation with 2x1.5g Cyclophosphamid If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG | Drug | Conditioning with CYC/ antithymocyte globulin (ATG), Conditioning with CYC/Thiotepa/ATG |
Primary outcome
Efficay - Overall survival
Time frame: 3 years
Number of patients that are alive after 3 years
4 secondary outcomes
Safety - Treatment related mortality
Time frame: 100 days
Time to engraftment
Time frame: 2 months
Progression free survival
Time frame: 3 years
Efficacy - Lung function test and Skin
Time frame: 3 years
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 29 Jul 2024.
- Derived from this study: PubMed 24459219Henes JC, Koetter I, Horger M, Schmalzing M, Mueller K, Eick C, Bauer A, Vogel W, Kanz L. Autologous stem cell transplantation with thiotepa-based conditioning in patients with systemic sclerosis and cardiac manifestations. Rheumatology (Oxford). 2014 May;53(5):919-22. doi: 10.1093/rheumatology/ket464. Epub 2014 Jan 22.
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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.
Joerg C Henes, MD principal investigator
University Hospital Tuebingen, Department of oncology, hematology, rheumatology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells in people with Scleroderma and Cardiac Involvement, run by University Hospital Tuebingen at 1 site in Germany.
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 11 inclusion and 21 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 29 Jul 2024. 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 Germany. 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 phase mean?
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?
Efficay - Overall survival (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01895244, which also lists 1 linked publication.
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 29 Jul 2024.