Chronic Myelomonocytic LeukemiaPhase 2NCT00015990
Thalidomide in Treating Patients With Myelodysplastic Syndrome
A Phase 2 study testing thalidomide and laboratory biomarker analysis in people with Chronic Myelomonocytic Leukemia and de Novo Myelodysplastic Syndromes, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes
- What is being tested?
- thalidomide and laboratory biomarker analysis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2001to Feb 2007
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Pre-transfusion hemoglobin =< 10 g/dL
- Pre-transfusion platelet count =< 50,000/μL
- Absolute neutrophil count < 1000/μL
- Total bilirubin ≤ 1.5 x UNL
- Alkaline phosphatase ≤ 3 x UNL
- AST ≤ 3 x UNL
Show all 16Show fewer
- Creatinine ≤ 1.5 x UNL
- A diagnosis of MDS as demonstrated in the bone marrow; any subtypes are eligible including:
- Refractory anemia (cytopenia)
- Refractory anemia with ringed sideroblasts
- Chronic myelomonocytic leukemia
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- Unclassified MDS
- Patients with refractory anemia with excess blasts in transformation who are not candidates for (or who decline) induction chemotherapy are eligible; those patients who were candidates for (and accepted) induction chemotherapy should have failed at least 1 chemotherapy regimen prior to entry
- Patients who are candidates for marrow transplantation should have this option discussed prior to study entry
Who cannot join
- Any of the following as this regimen may be harmful to a developing fetus or nursing child:
- Pregnant women
- Nursing women
- Women of childbearing potential or their sexual partners who are unwilling to employ 2 adequate methods of contraception (condoms, diaphragm, birth control pills, injections, intrauterine device [IUD], surgical sterilization, subcutaneous implants, or abstinence, etc.)
- Peripheral neuropathy (by history or clinical exam)
- Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (< 20 mg/d) for reasons other than MDS are allowed
- Uncontrolled infections
Trial locations and contacts
1 site in 1 country.
United States1 site
North Central Cancer Treatment Group
Rochester, Minnesota, 55905
About this study
Official title: Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome
Read the detailed description
OBJECTIVES:
- Determine whether thalidomide improves cytopenias in patients with myelodysplastic syndromes.
II. Determine the toxicity of this regimen in these patients. III. Determine whether this regimen down regulates the peripheral blood levels of tumor necrosis factor alpha, interferon gamma, and interleukin-12 and whether these changes correlate with clinical response in these patients.
IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution and whether these changes correlate with clinical response in these patients.
- Determine the effect of this regimen on bone marrow microvessel density and whether these effects correlate with clinical response in these patients.
OUTLINE: This is a multicenter study. Patients are stratified according to prognosis (favorable vs unfavorable). (Favorable stratum closed to accrual 12/28/01)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
Patients are followed every 6 months for 1 year and then annually for 4 years.
PROJECTED ACCRUAL: A total of 20-58 patients (10-29 per stratum) will be accrued for this study within 20 months. (Favorable stratum closed to accrual 12/28/01)
- Conditions
- Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation
- Being tested
- thalidomide, laboratory biomarker analysis
- Medical subject headings
- Leukemia, Myelomonocytic, Chronic, 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.
thalidomideDrug
Also called Kevadon, Synovir, THAL, Thalomid
Given orally
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (thalidomide) | Experimental | Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity. Receives: Drug: thalidomide; Other: laboratory biomarker analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al
Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Measured: Up to 3 months
Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
Measured: Up to 5 years
Secondary outcomes (4)
Survival time
Estimated using the method of Kaplan-Meier.
Measured: Time from registration to death due to any cause, assessed up to 5 years
Time to disease progression
Estimated using the method of Kaplan-Meier.
Measured: Time from registration to documentation of disease progression, assessed up to 5 years
Duration of response measured using criteria reported by Cheson et al
Measured: Date at which the patient's objective status is first noted to be either a CHR or PR to the date progression is documented, assessed up to 5 years
Time to treatment failure
Measured: Time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 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 Feb 2004 (estimated)
- Study startApr 2001
- Primary completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelomonocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Alvaro Moreno Aspitia, Principal investigator, North Central Cancer Treatment Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00015990
- Sponsor's protocol ID
- NCI-2012-01856
- Other IDs
- N998B, U10CA025224, CDR0000068580
- Status last verified
- Jan 2013
- First submitted
- 6 May 2001
- Last updated
- 24 Jan 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study
- ClinicalTrials.gov ID
- NCT00015990
- Acronym / sponsor ID
- NCI-2012-01856
- Conditions
- Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Apr 2001
- Primary completion
- Feb 2007 (actual)
- First posted
- 27 Feb 2004 (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.
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome
Detailed description
OBJECTIVES:
- Determine whether thalidomide improves cytopenias in patients with myelodysplastic syndromes.
II. Determine the toxicity of this regimen in these patients. III. Determine whether this regimen down regulates the peripheral blood levels of tumor necrosis factor alpha, interferon gamma, and interleukin-12 and whether these changes correlate with clinical response in these patients.
IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution and whether these changes correlate with clinical response in these patients.
- Determine the effect of this regimen on bone marrow microvessel density and whether these effects correlate with clinical response in these patients.
OUTLINE: This is a multicenter study. Patients are stratified according to prognosis (favorable vs unfavorable). (Favorable stratum closed to accrual 12/28/01)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
Patients are followed every 6 months for 1 year and then annually for 4 years.
PROJECTED ACCRUAL: A total of 20-58 patients (10-29 per stratum) will be accrued for this study within 20 months. (Favorable stratum closed to accrual 12/28/01)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (thalidomide) | Experimental | Drug: thalidomide, Other: laboratory biomarker analysisPatients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Pre-transfusion hemoglobin =< 10 g/dL
- Pre-transfusion platelet count =< 50,000/μL
- Absolute neutrophil count < 1000/μL
- Total bilirubin ≤ 1.5 x UNL
- Alkaline phosphatase ≤ 3 x UNL
Key exclusion criteria (7 listed)
- Any of the following as this regimen may be harmful to a developing fetus or nursing child:
- Peripheral neuropathy (by history or clinical exam)
- Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (< 20 mg/d) for reasons other than MDS are allowed
- Uncontrolled infections
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 |
|---|---|---|
| thalidomideAlso: Kevadon, Synovir, THAL, ThalomidGiven orally | Drug | Treatment (thalidomide) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (thalidomide) |
Primary outcome
Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al
Time frame: Up to 3 months
Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
Time frame: Up to 5 years
4 secondary outcomes
Survival time
Time frame: Time from registration to death due to any cause, assessed up to 5 years
Time to disease progression
Time frame: Time from registration to documentation of disease progression, assessed up to 5 years
Duration of response measured using criteria reported by Cheson et al
Time frame: Date at which the patient's objective status is first noted to be either a CHR or PR to the date progression is documented, assessed up to 5 years
Time to treatment failure
Time frame: Time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 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 24 Jan 2013.
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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.
Alvaro Moreno Aspitia principal investigator
North Central Cancer Treatment Group
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 thalidomide and laboratory biomarker analysis in people with Chronic Myelomonocytic Leukemia and de Novo Myelodysplastic Syndromes, run by National Cancer Institute (NCI) at 1 site in United States.
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 16 inclusion and 7 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 24 Jan 2013. 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 United States. 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?
Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al (time frame: Up to 3 months). The study lists 2 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 24 Jan 2013.