Primary MyelofibrosisPhase 2NCT00015821
Thalidomide in Treating Patients With Myelofibrosis
A Phase 2 study testing thalidomide and laboratory biomarker analysis in people with Primary Myelofibrosis, 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 myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis
- 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
- 43 participants
- Where
- 1 siteUnited States
- Study dates
- May 2000to Dec 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
- Histologically confirmed myelofibrosis with myeloid metaplasia
- Agnogenic myeloid metaplasia
- Post-polycythemic myeloid metaplasia
- Post-thrombocythemic myeloid metaplasia
- No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis
- No chromosomal translocation t(9;22) or bcr/abl gene rearrangement
Show all 27Show fewer
- Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood
- Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly
- Performance status - ECOG 0-2
- Absolute neutrophil count greater than 750/mm^3
- Platelet count less than 400,000/mm^3
- WBC less than 50,000/mm^3
- Bilirubin no greater than 2 mg/dL (if total bilirubin elevated, direct bilirubin must be normal)
- AST no greater than 3 times upper limit of normal (ULN)
- Alkaline phosphatase no greater than 3 times ULN
- Creatinine no greater than 1.5 mg/dL
- Creatinine clearance at least 60 mL/min
- Not pregnant or nursing
- Negative pregnancy test
- Fertile women must use at least 1 highly active method AND 1 additional effective method of contraception for at least 4 weeks before study, during study, and for at least 4 weeks after study
- Fertile men must use effective contraception during study and for at least 4 weeks after study
- No uncontrolled infection
- No concurrent condition that would preclude study
- No peripheral neuropathy
- At least 1 month since prior interferon, pirfenidone, anagrelide, or epoetin alfa
- At least 1 month since prior hydroxyurea or other chemotherapy
- At least 1 month since prior corticosteroids or androgen derivatives
Who cannot join
None listed.
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: A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells.
Read the detailed description
PRIMARY OBJECTIVES:
- To investigate whether thalidomide, a potent inhibitor of angiogenic and fibrogenic growth factors, is an effective therapeutic agent in patients with MMM. Specifically, to assess whether thalidomide improves anemia and/or organomegaly in patients with MMM.
II. To assess the effects of thalidomide on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF, and their respective receptors.
OUTLINE: This is a multicenter study.
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
Patients are followed every 6 months until 5 years from study entry.
- Conditions
- Primary Myelofibrosis
- Being tested
- thalidomide, laboratory biomarker analysis
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
thalidomideDrug
Given PO
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (thalidomide) | Experimental | Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy. 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, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
The proportion of successes will be estimated using the Binomial point estimator (number of successes divided by the total number of evaluable patients) and 95% confidence intervals calculated using the Duffy-Santner algorithm for multi-stage designs.
Measured: Up to 5 years
Secondary outcomes (3)
Survival
Kaplan-Meier survival curves will be generated to estimate survival.
Measured: Number of days from registration date to the date of death or last follow-up, assessed up to 5 years
Time to progression
Kaplan-Meier survival curves will be generated to estimate time to progression.
Measured: Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years
Response duration
Kaplan-Meier survival curves will be generated to estimate response duration.
Measured: Number of days from the first date that objective status = complete or partial response was recorded to the date of disease progression or date of death, whichever comes first, 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 posted23 May 2003 (estimated)
- Study startMay 2000
- Primary completionDec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary MyelofibrosisStudies 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
- 43 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Michelle Elliott, Principal investigator, North Central Cancer Treatment Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00015821
- Sponsor's protocol ID
- NCI-2012-01853
- Other IDs
- NCI-2012-01853, CDR0000068367, NCCTG-N9982, N9982, N9982, U10CA025224
- Status last verified
- Oct 2013
- First submitted
- 6 May 2001
- Last updated
- 8 Oct 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
- ClinicalTrials.gov ID
- NCT00015821
- Acronym / sponsor ID
- NCI-2012-01853
- Conditions
- Primary Myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- May 2000
- Primary completion
- Dec 2007 (actual)
- First posted
- 23 May 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.
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells.
Detailed description
PRIMARY OBJECTIVES:
- To investigate whether thalidomide, a potent inhibitor of angiogenic and fibrogenic growth factors, is an effective therapeutic agent in patients with MMM. Specifically, to assess whether thalidomide improves anemia and/or organomegaly in patients with MMM.
II. To assess the effects of thalidomide on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF, and their respective receptors.
OUTLINE: This is a multicenter study.
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
Patients are followed every 6 months until 5 years from study entry.
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 for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (27 listed)
- Histologically confirmed myelofibrosis with myeloid metaplasia
- No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis
- No chromosomal translocation t(9;22) or bcr/abl gene rearrangement
- Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood
- Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| thalidomideGiven PO | Drug | Treatment (thalidomide) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (thalidomide) |
Primary outcome
Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
Time frame: Up to 5 years
The proportion of successes will be estimated using the Binomial point estimator (number of successes divided by the total number of evaluable patients) and 95% confidence intervals calculated using the Duffy-Santner algorithm for multi-stage designs.
3 secondary outcomes
Survival
Time frame: Number of days from registration date to the date of death or last follow-up, assessed up to 5 years
Time to progression
Time frame: Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years
Response duration
Time frame: Number of days from the first date that objective status = complete or partial response was recorded to the date of disease progression or date of death, whichever comes first, 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 8 Oct 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.
Michelle Elliott 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 Primary Myelofibrosis, 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 27 inclusion and 0 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 8 Oct 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, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart. (time frame: Up to 5 years). 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 8 Oct 2013.