Hematologic DiseasesPhase 1/2NCT00856505
Safety Study of Calcineurin Inhibitor Free GvHD Prophylaxis in Allogeneic Stem Cell Transplantation
A Phase 1/2 study testing Everolimus and mycophenolate sodium in people with Hematologic Diseases, run by University Hospital Freiburg at 1 site in Germany.
Trial summary
In stem cell transplantation as treatment for malignant diseases, calcineurin inhibitors like cyclosporine A are commonly used to prevent tissue destruction (GvHD) by activated donor immune cells. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hematologic Diseases
- What is being tested?
- Everolimus and mycophenolate sodium
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteGermany
- Study dates
- Mar 2008to Mar 2011
- 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
- Diagnosis of hematologic malignancies, indicated for allogeneic stem cell transplantation:
- acute myeloid leukemia (AML), in CR1, ≥ CR2, primary refractory, relapse
- chronic myeloid leukemia (CML), in chronic phase, in acceleration or blast crisis
- myelodysplastic syndrome (MDS), RA/RARS (transfusion dependent), RAEB, RAEB-t and CMML
- Lymphoma:
- plasmocytoma
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- immunocytoma (M. Waldenström)
- chronic-lymphatic leukemia (CLL)
- additional low and high grade Non-Hodgkin Lymphoma
- Hodgkins disease
- HLA-matched (HLA-A, -B, -DRB1) related or unrelated donor available
- Signed informed consent
Who cannot join
- CNS involvement by underlying disease
- Pulmonary disease with VC < 55%, DLCO < 40%
- Cardiac ejection fraction < 30%, uncontrollable arrhythmia
- Creatinin > 1,5 mg/dl or Creatinin-Clearance < 30 ml/min
- Bilirubin > 2 mg/dl
- Active Hepatitis B or C
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- HIV serologic positive
- Pregnancy and lactation
- Pre-menstrual women without medical safe contraception
- Participation on another clinical trial in between 30 days before start or during the study only if the clinical trial interferes with the outcome measures.
- Known allergy to study medication or ingredients of the formulation
- Drug- or alcohol abuse
- Non-compliance
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
University Medical Center, Division Hematology/Oncology
Freiburg im Breisgau, Baden-Wurttemberg, 79104
Reinhard Marks, MD, 49-761-270 ext. 3278, reinhard.marks@uniklinik-freiburg.de
Reinhard Marks, MD
About this study
Official title: Everolimus and Mycophenolate Sodium as GvHD Prophylaxis in Allogeneic Stem Cell Transplantation
In stem cell transplantation as treatment for malignant diseases, calcineurin inhibitors like cyclosporine A are commonly used to prevent tissue destruction (GvHD) by activated donor immune cells. The hypothesis for this study is, that replacing calcineurin inhibitors by everolimus and mycophenolate as GvHD prophylaxis not only reduces toxicity of the treatment but also improves tolerance induction of the donor T cells toward the host, eventually increasing the safety of stem cell transplantation.
- Conditions
- Hematologic Diseases
- Being tested
- Everolimus and mycophenolate sodium
- Keywords
- Hematopoietic Stem Cell Transplantation, Drug Therapy, Combination, Immunosuppression
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Everolimus and mycophenolate sodiumDrug
Everolimus tablets, 1.5mg/day bid, dosage adjusted to plasma levels Mycophenolate sodium, 720mg/day bid Duration: Mycophenolate tapering starts at day 56 after stem cell transplantation Everolimus tapering starts at day 100 after stem cell transplantation if no GvHD evident
| Study group | Role | What they receive |
|---|---|---|
| Everolimus and mycophenolate sodium | Experimental | Combination of experimental immunosuppressants for GvHD prophylaxis Receives: Drug: Everolimus and mycophenolate sodium |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity according to CTCAE v3.0
Measured: after 100 days and one year after treatment start
Secondary outcomes (4)
Hematopoietic engraftment
Measured: day 30 after stem cell transplantation
Incidence of acute and chronic GvHD
Measured: one year after stem cell transplantation
Progression free survival
Measured: Day 100 and one year after stem cell transplantation
Overall survival
Measured: day 100 and one year after stem cell transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2009 (estimated)
- Study startMar 2008
- Primary completionMar 2010 (estimated)
- Study completionMar 2011 (estimated)
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 Hematologic DiseasesStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital Freiburg
- Sponsor type
- University, hospital or other
- Investigators
- Juergen Finke, MD, Principal investigator, University Medical Center Freiburg, Div. Hematology/OncologyReinhard Marks, MD, Study director, University Medical Center Freiburg, Div. Hematology/Oncology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00856505
- Sponsor's protocol ID
- 00557
- Status last verified
- Mar 2009
- First submitted
- 4 Mar 2009
- Last updated
- 5 Mar 2009
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Schafer H, Blumel-Lehmann J, Ihorst G, Bertz H, Wasch R, Zeiser R, Finke J, Marks R. A prospective single-center study on CNI-free GVHD prophylaxis with everolimus plus mycophenolate mofetil in allogeneic HCT. Ann Hematol. 2021 Aug;100(8):2095-2103. doi: 10.1007/s00277-021-04487-y. Epub 2021 Mar 23.
PubMed 33755792
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Everolimus and Mycophenolate Sodium as GvHD Prophylaxis in Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT00856505
- Acronym / sponsor ID
- 00557
- Conditions
- Hematologic Diseases
- Keywords
- Hematopoietic Stem Cell Transplantation, Drug Therapy, Combination, Immunosuppression
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- University Hospital FreiburgUniversity, hospital or other
- Study start
- Mar 2008
- Primary completion
- Mar 2010 (estimated)
- Study completion
- Mar 2011 (estimated)
- First posted
- 5 Mar 2009 (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.
In stem cell transplantation as treatment for malignant diseases, calcineurin inhibitors like cyclosporine A are commonly used to prevent tissue destruction (GvHD) by activated donor immune cells. The hypothesis for this study is, that replacing calcineurin inhibitors by everolimus and mycophenolate as GvHD prophylaxis not only reduces toxicity of the treatment but also improves tolerance induction of the donor T cells toward the host, eventually increasing the safety of stem cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus and mycophenolate sodium | Experimental | Drug: Everolimus and mycophenolate sodiumCombination of experimental immunosuppressants for GvHD prophylaxis |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Diagnosis of hematologic malignancies, indicated for allogeneic stem cell transplantation:
- acute myeloid leukemia (AML), in CR1, ≥ CR2, primary refractory, relapse
- chronic myeloid leukemia (CML), in chronic phase, in acceleration or blast crisis
- myelodysplastic syndrome (MDS), RA/RARS (transfusion dependent), RAEB, RAEB-t and CMML
- Lymphoma:
Key exclusion criteria (13 listed)
- CNS involvement by underlying disease
- Pulmonary disease with VC < 55%, DLCO < 40%
- Cardiac ejection fraction < 30%, uncontrollable arrhythmia
- Creatinin > 1,5 mg/dl or Creatinin-Clearance < 30 ml/min
- Bilirubin > 2 mg/dl
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 |
|---|---|---|
| Everolimus and mycophenolate sodiumEverolimus tablets, 1.5mg/day bid, dosage adjusted to plasma levels Mycophenolate sodium, 720mg/day bid Duration: Mycophenolate tapering starts at day 56 after stem cell transplantation Everolimus tapering starts at day 100 after stem cell transplantation if no GvHD evident | Drug | Everolimus and mycophenolate sodium |
Primary outcome
Toxicity according to CTCAE v3.0
Time frame: after 100 days and one year after treatment start
4 secondary outcomes
Hematopoietic engraftment
Time frame: day 30 after stem cell transplantation
Incidence of acute and chronic GvHD
Time frame: one year after stem cell transplantation
Progression free survival
Time frame: Day 100 and one year after stem cell transplantation
Overall survival
Time frame: day 100 and one year after stem cell transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2009.
- Derived from this study: PubMed 33755792Schafer H, Blumel-Lehmann J, Ihorst G, Bertz H, Wasch R, Zeiser R, Finke J, Marks R. A prospective single-center study on CNI-free GVHD prophylaxis with everolimus plus mycophenolate mofetil in allogeneic HCT. Ann Hematol. 2021 Aug;100(8):2095-2103. doi: 10.1007/s00277-021-04487-y. Epub 2021 Mar 23.
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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.
Juergen Finke, MD principal investigator
University Medical Center Freiburg, Div. Hematology/Oncology
Reinhard Marks, MD study director
University Medical Center Freiburg, Div. Hematology/Oncology
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 Everolimus and mycophenolate sodium in people with Hematologic Diseases, run by University Hospital Freiburg 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 12 inclusion and 13 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: status not confirmed, as of the sponsor's last update on 5 Mar 2009. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Reinhard Marks, MD (49-761-270- ext. 3278, reinhard.marks@uniklinik-freiburg.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Toxicity according to CTCAE v3.0 (time frame: after 100 days and one year after treatment start). 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 NCT00856505, 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 5 Mar 2009.