Myelodysplastic Syndrome (MDS)Phase 2RISENCT00542828
Rabbit Anti-thymocyte Globulin in the Treatment of Patients With Low to Intermediate-1 Risk Myelodysplastic Syndrome
A Phase 2 study testing Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG) in people with Myelodysplastic Syndrome (MDS), run by Genzyme, a Sanofi Company at 7 sites in 4 countries.
Trial summary
This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myelodysplastic Syndrome (MDS)
- What is being tested?
- Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 7 sitesin 4 countries
- Study dates
- Oct 2007to Jul 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient provided signed written informed consent.
- Patient had pathologically confirmed low or intermediate-1 risk MDS at the time of MDS diagnosis and at the time of screening.
- Patient had received no more than 1 prior treatment for MDS.
- Patient exhibited at least 1 hematologic cytopenia (anemia, neutropenia, or thrombocytopenia) over a period of ≥1 week.
- Patient had documentation of any prior transfusion requirements.
- Patient had an Eastern Cooperative Oncology Group (ECOG) performance score of 0, 1, or 2.
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- Patient was ≥18 and ≤70 years of age at time of signing the informed consent document (ICD).
- Patient was able to adhere to study visit schedule and all other protocol requirements.
- Patient was willing to practice a medically approved method of birth control during participation in the study (at least 12 months after the last infusion of rATG) (fertile male and female patients).
Who cannot join
- Patient was pregnant or lactating.
- Patient has had prior treatment with any ATG.
- Patient has received any immunomodulatory or immunosuppressing agents (excluding steroids) <12 weeks prior to the first infusion of rATG.
- Patient has had a prior hematopoietic stem cell transplantation and/or other organ transplant.
- Patient has had a prior allergic reaction to rabbit proteins or excipients.
- Patient had any of the following subtypes of MDS: refractory anemia with ringed sideroblasts (RARS); chronic myelomonocytic leukemia (CMML) if white blood counts >13x10^9/L; or other MDS/myeloproliferative diseases (MPD).
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- Patient had MDS associated with a 5q chromosomal deletion unless the patient received prior lenalidomide treatment <4 weeks prior to the first infusion of rATG.
- Patient had MDS presumed secondary to exposure to chemicals or treatment with radiotherapy or chemotherapy.
- Patient received any investigational agents within 4 weeks prior to the first infusion of rATG.
- Patient has any of the following abnormalities: serum creatinine >1.5 x upper limit of normal (ULN); aspartate transaminase (AST) and alanine transaminase (ALT) >2.5 x ULN; or serum total bilirubin >1.5 x ULN, except for unconjugated hyperbilirubinemia related to the patient's MDS.
- Patient received any treatment with non-steroidal anti-inflammatory drugs (NSAIDs) within 14 days prior to the start of treatment.
- Patient was known to be human immunodeficiency virus (HIV) positive.
- Patient had any prior diagnosis of malignancy other than MDS, unless the patient had been disease-free for at least 5 years following the completion of curative intent therapy.
- Patient had any serious medical condition (other than MDS) that would limit survival to <2 years.
- Patient had active acute or chronic infection, including cytomegaloviremia (CMV) infection or deep tissue infection.
- Patient had any other serious medical condition, uncontrolled illness (including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia), social condition, or psychiatric illness that would prevent the patient from signing the informed consent document (ICD), or would place the patient at unacceptable risk if he/she participated in the study, or that would limit compliance with study requirements.
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
United Kingdom3 sites
Royal Bournemouth Hospital
Bournemouth, England, BH7 7DW
St. James Hospital
Leeds, England, LS9 7TF
King's College Hospital
London, England, SE5 9RS
Germany2 sites
St. Johannes-Hospital Duisburg
Duisburg, 47166
Medizinische Hochschule Hannover
Hanover, 30625
About this study
Official title: A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome
This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who have never been treated for MDS (i.e., treatment-naïve patients).
- Conditions
- Myelodysplastic Syndrome (MDS)
- Being tested
- Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG)Biological
Also called Rabbbit Anti-human thymocyte immunoglobulin
All patients were to be treated with rATG 3.75 mg/kg/day administered by intravenous (IV) infusion over ≥6 hours for 5 consecutive days (cumulative dose: 18.75 mg/kg)
| Study group | Role | What they receive |
|---|---|---|
| Thymoglobulin | Experimental | Not described Receives: Biological: Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Achieved Hematologic Improvement (HI)
This is a measure of HI in the erythroid, platelet, and neutrophil lineages. Note that HI was observed in the erythroid lineage only, which is defined as a participant who had a >=1.5 g/dL increase in hemoglobin from baseline (pretreatment value must have been <11 g/dL) and who had a relevant reduction of units of red blood cell (RBC) transfusions by an absolute number of >=4 RBC transfusions over 8 weeks as compared with the pretreatment transfusion number in the previous 8 weeks. These criteria were taken from the 2006 International Working Group criteria.
Measured: 12 months
Secondary outcomes (11)
Number of Participants With Duration of HI
This is a measure of the duration of HI for those participants who achieved HI. Duration of HI is defined as the time from confirmation of HI response to the date of first documentation of HI relapse or death due to any cause, whichever occurs first.
Measured: 36 months
Number of Participants Who Achieved Disease Remission
Disease remission is defined as a participant whose best response to therapy was a complete remission or partial remission. A complete remission is defined as: bone marrow <=5% myeloblasts with normal maturation of all cell lines; persistent dysplasia noted; and peripheral blood hemoglobin >=11 g/dL, platelets >=100 x 10^9/L, neutrophils >=1.0 x 10^9/L, and blasts 0%. Partial remission is defined as: all complete remission criteria if abnormal before treatment, except bone marrow blasts decreased by >=50% over pretreatment, but still >5%; and cellularity and morphology not relevant.
Measured: 36 months
Duration of Disease Remission
This is a measure of the duration of overall disease remission only for those participants who achieved an overall remission. Duration of overall remission is defined as the time from first documentation of overall remission to the date of first documentation of disease relapse or death due to any cause, whichever occurs first.
Measured: 36 months
Number of Participants Who Achieved Transfusion Independence
This is a measure of transfusion independence, which is defined as a participant with no transfusions for a period of 8 consecutive calendar weeks after first dose. Transfusion independence was to be calculated only for those participants who had documented transfusions during the 8 weeks prior to enrollment.
Measured: 36 months
Number of Participants With Duration of Transfusion Independence
This is a measure of the duration of transfusion independence only for those participants who achieved transfusion independence. Duration of transfusion independence is defined as the longest period of time during which a participant requires no transfusions.
Measured: 36 months
Number of Participants With a Relapse Following HI
This is a measure of relapse following HI, which is defined as a participant who experiences at least one of the following: >=50% decrease from maximum response levels in granulocytes or platelets; >=1.5 g/dL reduction in hemoglobin; or transfusion dependence.
Measured: 36 months
Number of Participants With a Relapse Following Overall Remission
This is a measure of relapse following an overall remission only for participants who experienced either a complete or partial remission. Relapse following an overall remission is defined as a participant who meets any of the following criteria: a return to pretreatment bone marrow blast percentage; decrease of >=50% from maximum remission levels in neutrophils or platelets; reduction in hemoglobin concentration by >=1.5 g/dL from maximum remission levels; or transfusion dependence.
Measured: 36 months
Number of Participants With Progression-free Survival
This is a measure of a progression-free survival which is defined as the time from the participant's first dose to the date of disease progression, lost to follow-up or death due to any cause, whichever occurs first.
Measured: 36 months
Number of Participants With Transformation to Acute Myeloid Leukemia
This is a measure of transformation to acute myeloid leukemia only for participants who have bone marrow assessments. Transformation to acute myeloid leukemia is defined as the earliest date a participant experiences bone marrow blasts of >=20% after the start of treatment.
Measured: 36 months
Number of Participants With a Cytogenetic Response
This is a measure of cytogenic response for participants whose best response to therapy is a either a complete or partial cytogenetic response. A complete cytogenetic response is defined as the disappearance of the chromosomal abnormality without appearance of new ones. A partial cytogenetic response is defined as at least 50% reduction of the chromosomal abnormality.
Measured: 36 months
Number of Participants With a Marrow Remission
This is a measure of bone marrow complete remission for participants who experience a remission. Bone marrow complete remission is defined as a bone marrow assessment of <=5% myeloblasts and decrease by >=50% over pretreatment.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2007 (estimated)
- Study startOct 2007
- Primary completionMay 2009
- Study completionJul 2009
Why it stopped: Study terminated due to slow enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic Syndrome (MDS)Studies 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
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00542828
- Sponsor's protocol ID
- ThymoHEM01206
- Other IDs
- 2007-002532-28
- Acronym
- RISE
- Status last verified
- Mar 2015
- First submitted
- 10 Oct 2007
- Last updated
- 3 Apr 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT00542828
- Acronym / sponsor ID
- RISE / ThymoHEM01206
- Conditions
- Myelodysplastic Syndrome (MDS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated due to slow enrollment.
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Oct 2007
- Primary completion
- May 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 12 Oct 2007 (estimated)
- Results
- Posted 27 Apr 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who have never been treated for MDS (i.e., treatment-naïve patients).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thymoglobulin | Experimental | Biological: Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patient provided signed written informed consent.
- Patient had pathologically confirmed low or intermediate-1 risk MDS at the time of MDS diagnosis and at the time of screening.
- Patient had received no more than 1 prior treatment for MDS.
- Patient exhibited at least 1 hematologic cytopenia (anemia, neutropenia, or thrombocytopenia) over a period of ≥1 week.
- Patient had documentation of any prior transfusion requirements.
Key exclusion criteria (16 listed)
- Patient was pregnant or lactating.
- Patient has had prior treatment with any ATG.
- Patient has received any immunomodulatory or immunosuppressing agents (excluding steroids) <12 weeks prior to the first infusion of rATG.
- Patient has had a prior hematopoietic stem cell transplantation and/or other organ transplant.
- Patient has had a prior allergic reaction to rabbit proteins or excipients.
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 |
|---|---|---|
| Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG)Also: Rabbbit Anti-human thymocyte immunoglobulinAll patients were to be treated with rATG 3.75 mg/kg/day administered by intravenous (IV) infusion over ≥6 hours for 5 consecutive days (cumulative dose: 18.75 mg/kg) | Biological | Thymoglobulin |
Primary outcome
Number of Participants Who Achieved Hematologic Improvement (HI)
Time frame: 12 months
This is a measure of HI in the erythroid, platelet, and neutrophil lineages. Note that HI was observed in the erythroid lineage only, which is defined as a participant who had a >=1.5 g/dL increase in hemoglobin from baseline (pretreatment value must have been <11 g/dL) and who had a relevant reduction of units of red blood cell (RBC) transfusions by an absolute number of >=4 RBC transfusions over 8 weeks as compared with the pretreatment transfusion number in the previous 8 weeks. These criteria were taken from the 2006 International Working Group criteria.
11 secondary outcomes
Number of Participants With Duration of HI
Time frame: 36 months
Number of Participants Who Achieved Disease Remission
Time frame: 36 months
Duration of Disease Remission
Time frame: 36 months
Number of Participants Who Achieved Transfusion Independence
Time frame: 36 months
Number of Participants With Duration of Transfusion Independence
Time frame: 36 months
Number of Participants With a Relapse Following HI
Time frame: 36 months
Number of Participants With a Relapse Following Overall Remission
Time frame: 36 months
Number of Participants With Progression-free Survival
Time frame: 36 months
Number of Participants With Transformation to Acute Myeloid Leukemia
Time frame: 36 months
Number of Participants With a Cytogenetic Response
Time frame: 36 months
Number of Participants With a Marrow Remission
Time frame: 36 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Medical Monitor study director
Genzyme, a Sanofi Company
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG) in people with Myelodysplastic Syndrome (MDS), run by Genzyme, a Sanofi Company at 7 sites in 4 countries.
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 9 inclusion and 16 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: stopped early, as of the sponsor's last update on 3 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom, Germany, France, Netherlands. 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?
Yes. Results are posted in the registry (first posted 27 Apr 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants Who Achieved Hematologic Improvement (HI) (time frame: 12 months). 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 3 Apr 2015.