Myelodysplastic SyndromesPhase 3NCT00004208
Antithymocyte Globulin and Cyclosporine in Treating Patients With Myelodysplastic Syndrome
A Phase 3 study testing ATG + CSA and Supportive care in people with Myelodysplastic Syndromes, run by Swiss Cancer Institute at 1 site in Switzerland.
Trial summary
The main objective of this trial is to evaluate the efficacy and toxicity of intensive immuno-suppression with ATG + CSA versus best supportive care in patients with transfusion dependent low and intermediate risk myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- ATG + CSA and Supportive care
- Type of treatment
- Pill or oral medicine, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 86 participants
- Where
- 1 siteSwitzerland
- Study dates
- Aug 2000to Oct 2011
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Hypoplastic MDS, or MDS subtype RA, RAS or RAEB with ≤ 10% blasts, or RAEB with 10-20% blasts and the patient refusing or not eligible for the MDS high risk protocol (EORTC 06961)
- Transfusion dependence < 24 months or neutrophils < 0.5 × 109/l.
- ECOG/SAKK performance status ≤ 2
- Age > 18 years
- No active uncontrolled infection
- No prior chemotherapy or radiotherapy
- No history of heart failure, clinically relevant cardiac arrhythmia or other hemato-oncological disease
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Antithymocyte Globulin (ATG) and Cyclosporine (CSA) to Treat Patients With Myelodysplastic Syndrome (MDS). A Randomized Trial Comparing ATG + CSA With Best Supportive Care
The main objective of this trial is to evaluate the efficacy and toxicity of intensive immuno-suppression with ATG + CSA versus best supportive care in patients with transfusion dependent low and intermediate risk myelodysplastic syndrome (MDS).
Read the detailed description
This trial will answer the question whether immuno-suppression improves hematopoesis and reduces transfusion requirements analogous to patients with aplastic anemia as the short-term outcome, and whether immuno-suppression accelerates leukemic transformation and influences survival as the long-term outcome.
Primary endpoint: best response (CR + PR) rate at month 6
- Conditions
- Myelodysplastic Syndromes
- Being tested
- ATG + CSA, Supportive care
- Keywords
- refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Refractory, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATG + CSADrug
Mérieux; horse antithymocyte globulin (ATG) + Cyclosporine A (CSA)
Supportive careBehavioural
Patients randomized to this arm will be treated as outpatients.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: ATG + CSA | Active comparator | Treatment consists of 15 mg/kg ATG (Mérieux; horse antithymocyte globulin; i.e. 1.5 vial/10 kg of body weight/day) given over 8-12 hours for 5 consecutive days.
Cyclosporine A (CSA) will be administered orally in a dose of 2.5 mg/kg bid starting day 1 and continued through day 180. Receives: Drug: ATG + CSA |
| Arm B: Supportive care | Other | Patients randomized to this arm will be treated as outpatients. Receives: Behavioral: Supportive care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best response rate (CR + PR)
Measured: 6 months
Secondary outcomes (4)
Response rate at month 3, time to response, quality and duration of response, and proportions of relapse/progression in responders
Measured: 2 and 5 years after first response
Overall survival
Measured: 2 and 5 years
Leukemia-free survival
Measured: 2 and 5 years
Transformation-free survival
Measured: 2 and 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startAug 2000
- Primary completionOct 2006
- Study completionOct 2011
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 Myelodysplastic SyndromesStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swiss Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jakob R. Passweg, MS, Study chair, Kantonsspital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004208
- Sponsor's protocol ID
- SAKK 33/99
- Other IDs
- SWS-SAKK-33/99
- Status last verified
- Mar 2015
- First submitted
- 21 Jan 2000
- Last updated
- 11 Mar 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Passweg JR, Giagounidis AA, Simcock M, Aul C, Dobbelstein C, Stadler M, Ossenkoppele G, Hofmann WK, Schilling K, Tichelli A, Ganser A. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care--SAKK 33/99. J Clin Oncol. 2011 Jan 20;29(3):303-9. doi: 10.1200/JCO.2010.31.2686. Epub 2010 Dec 13.
PubMed 21149672
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Antithymocyte Globulin (ATG) and Cyclosporine (CSA) to Treat Patients With Myelodysplastic Syndrome (MDS). A Randomized Trial Comparing ATG + CSA With Best Supportive Care
- ClinicalTrials.gov ID
- NCT00004208
- Acronym / sponsor ID
- SAKK 33/99
- Conditions
- Myelodysplastic Syndromes
- Keywords
- refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- Swiss Cancer InstituteUniversity, hospital or other
- Study start
- Aug 2000
- Primary completion
- Oct 2006 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 27 Jan 2003 (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.
The main objective of this trial is to evaluate the efficacy and toxicity of intensive immuno-suppression with ATG + CSA versus best supportive care in patients with transfusion dependent low and intermediate risk myelodysplastic syndrome (MDS).
Detailed description
This trial will answer the question whether immuno-suppression improves hematopoesis and reduces transfusion requirements analogous to patients with aplastic anemia as the short-term outcome, and whether immuno-suppression accelerates leukemic transformation and influences survival as the long-term outcome.
Primary endpoint: best response (CR + PR) rate at month 6
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A: ATG + CSA | Active comparator | Drug: ATG + CSATreatment consists of 15 mg/kg ATG (Mérieux; horse antithymocyte globulin; i.e. 1.5 vial/10 kg of body weight/day) given over 8-12 hours for 5 consecutive days. Cyclosporine A (CSA) will be administered orally in a dose of 2.5 mg/kg bid starting day 1 and continued through day 180. |
| Arm B: Supportive care | Other | Behavioral: Supportive carePatients randomized to this arm will be treated as outpatients. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| ATG + CSAMérieux; horse antithymocyte globulin (ATG) + Cyclosporine A (CSA) | Drug | Arm A: ATG + CSA |
| Supportive carePatients randomized to this arm will be treated as outpatients. | Behavioural | Arm B: Supportive care |
Primary outcome
Best response rate (CR + PR)
Time frame: 6 months
4 secondary outcomes
Response rate at month 3, time to response, quality and duration of response, and proportions of relapse/progression in responders
Time frame: 2 and 5 years after first response
Overall survival
Time frame: 2 and 5 years
Leukemia-free survival
Time frame: 2 and 5 years
Transformation-free survival
Time frame: 2 and 5 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 11 Mar 2015.
- Results publication: PubMed 21149672Passweg JR, Giagounidis AA, Simcock M, Aul C, Dobbelstein C, Stadler M, Ossenkoppele G, Hofmann WK, Schilling K, Tichelli A, Ganser A. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care--SAKK 33/99. J Clin Oncol. 2011 Jan 20;29(3):303-9. doi: 10.1200/JCO.2010.31.2686. Epub 2010 Dec 13.
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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.
Jakob R. Passweg, MS study chair
Kantonsspital Basel
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ATG + CSA and Supportive care in people with Myelodysplastic Syndromes, run by Swiss Cancer Institute at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 11 Mar 2015. 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 Switzerland. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Best response rate (CR + PR) (time frame: 6 months). 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 NCT00004208, 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 11 Mar 2015.