Myeloproliferative DisordersPhase 2NCT00452023

Pegasys® in Patients With Myeloproliferative Diseases

A Phase 2 study testing IFN-alpha2a in people with Myeloproliferative Disorders, run by M.D. Anderson Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The goal of this clinical research study is to see if Pegasys (IFN-alpha2a) can help to control the disease in patients with ET, PV, AMM/MF, and Ph-negative CML. The safety of this treatment will also be studied. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Myeloproliferative Disorders
What is being tested?: IFN-alpha2a
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 83 participants
Where: 1 siteUnited States
Study dates: 7 Apr 2005to 26 May 2023
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Following diagnoses: --ET: Patients with PLT > 600 x10 9 /l documented in the past 12 months; hyperplasia of marrow megakaryocytes in the absence of identifiable cause of thrombocytosis and in the absence of Ph chromosome. Patients with ET and lower PLT will be eligible if attributable to prior ET therapy. --PV: Patients should have Hb >/= 15g/dl (except if patient is having phlebotomies done) and documented past diagnosis.
Performance status </= 2 (ECOG scale).
Age greater than 18 years since disease is extremely rare in younger age group.
Adequate liver function: total bilirubin of </= 2.0 mg/dl (except for patients with Gilbert's Syndrome) and AST (SGOT) or ALT (SGPT) < 3 X ULN (or < 5 X ULN if considered due to tumor), and renal function (serum creatinine </= 2.0 mg/dl).
Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of the M.D. Anderson Cancer Center. The only acceptable consent form is the one approved by the M.D. Anderson Cancer Center IRB.
Willingness and ability to comply with the requirements of the protocol for the duration of the study.
Patients must have been off chemotherapy for 1 week prior to beginning Pegasys and have recovered from the toxic effects of that therapy. Patients may have received hydroxyurea or anagrelide immediately before study entry, and may continue into therapy if treating physician determines this is in the best interest of the patient.

Who cannot join

Pregnant or lactating women.
Patients with prior history of another malignancy or concurrent malignancy, except for the following: basal cell carcinoma of the skin, carcinoma in situ of the cervix, or other malignancies if the patient is disease free >3 years.
Patients with history of ischemic retinopathy.
Patients with history of severe cardiac disease: NYHA Functional Class III or IV, myocardial infarction within 6 months, uncontrolled ventricular tachyarrhythmias or unstable angina.
Patients with history of medically significant psychiatric disease if not controlled, especially endogenous depression (does not include reactive depression post-cancer diagnosis), psychosis and bipolar disease.
Patients with seizure disorders requiring anticonvulsant therapy.

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Patients with known infection with HBV, HIV, or other active systemic infection.
Patients with known autoimmune disease except for rheumatoid arthritis.
Patients with renal disease on hemodialysis.
Patients taking continuous or chronic high-dose systemic steroids; if discontinued, there must be a minimum washout period of one month before study drug is begun.
Patients with known hypersensitivity to PEG-IFN alpha-2a or its components.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Texas MD Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: PEG IFN-alpha2a (Pegasys®) Therapy in Patients With Chronic Myeloproliferative Diseases (Excluding Philadelphia Chromosome Positive Chronic Myeloid Leukemia)

Read the detailed description

IFN-alpha2a has been used for the treatment of a variety of disorders (such as hepatitis C). IFN-alpha2a is a drug that may affect the way infections and malignant diseases develop.

Before treatment starts, you will have blood (around 2 teaspoons) and bone marrow samples collected. To collect a bone marrow sample, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. These samples will be used for tests to confirm the diagnosis of the disease. Women who are able to have children must have a negative blood pregnancy test.

During treatment, you will receive IFN-alpha2a as an injection under the skin once a week. You (or your caregiver) will be taught how to give the injections, and you will receive treatment on an outpatient basis.

Treatment will continue (injections once a week) as long as the disease does not get worse.

If the disease gets worse or you experience any intolerable side effects, you will be taken off the study and your doctor will discuss other treatment options with you.

During treatment you will have blood (around 1 teaspoon) collected every other week for 2 months, then once every 1 or 2 months for a year, then every 3 months. You will also have bone marrow samples collected every 3 to 6 months during the first year of treatment. After the first year of treatment, bone marrow samples will be collected only when your doctor feels they are needed. The blood and bone marrow samples will be used for tests to check on the response to therapy.

This is an investigational study. IFN-alpha2a has been approved by the FDA for the treatment of hepatitis C and is commercially available. However the use of IFN-alpha2a in this study is investigational. The commercial prepartion of IFN-alpha2a(Pegasys) will be used in this study. Up to 280 participants will take part in this study. All will be enrolled at M. D. Anderson.

Conditions: Myeloproliferative Disorders
Being tested: IFN-alpha2a
Keywords: Myeloproliferative Disorders, Essential Thrombocythemia, Polycythemia Vera, Pegasys, IFN-alpha2a, Pegylated-Interferon Alpha-2A, PEG-IFNa-2a
Medical subject headings: Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IFN-alpha2aDrug
Also called Pegylated-Interferon Alpha-2A, PEG-IFNa-2a, Pegasys
Starting dose 90 microgram (mcg) injection under the skin once a week

Study groups in NCT00452023
Study group	Role	What they receive
IFN-alpha2a 90 Microgram (mcg) weekly	Experimental	Starting dose 90 microgram (mcg) injection under the skin once a week. Receives: Drug: IFN-alpha2a
IFN-alpha2a 450 Microgram (mcg) weekly	Experimental	Starting dose 450 microgram (mcg) injection under the skin once a week. Receives: Drug: IFN-alpha2a
IFN-alpha2a 360 Microgram (mcg) weekly	Experimental	Starting dose 360 microgram (mcg) injection under the skin once a week. Receives: Drug: IFN-alpha2a
IFN-alpha2a 270 Microgram (mcg) weekly	Experimental	Starting dose 270 microgram (mcg) injection under the skin once a week. Receives: Drug: IFN-alpha2a
IFN-alpha2a 180 Microgram (mcg) weekly	Experimental	Starting dose 180 microgram (mcg) injection under the skin once a week. Receives: Drug: IFN-alpha2a

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Patients With Complete Response (CR) or Partial Response (PR)
CR = Reduction of PLT to <440x109/l and disappearance of thromboembolic events, without the use of anagrelide or hydroxyurea
PR = Reduction of PLT by 50% but still >440x109/l or reduction of thromboembolic events by 50%, without the use of anagrelide or hydroxyurea
Measured: Up to 18 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

7 Apr 200526 May 2023

First posted26 Mar 2007 (estimated)
Study start7 Apr 2005
Primary completion26 May 2023
Study completion26 May 2023

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.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Myeloproliferative DisordersStudies of the same condition, open and under way.
More trials run by M.D. Anderson Cancer CenterThe 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: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 83 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00452023
Sponsor's protocol ID: DM03-0109
Other IDs: NCI-2012-01474
Status last verified: Dec 2024
First submitted: 22 Mar 2007
Last updated: 11 Dec 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Masarova L, Yin CC, Cortes JE, Konopleva M, Borthakur G, Newberry KJ, Kantarjian HM, Bueso-Ramos CE, Verstovsek S. Histomorphological responses after therapy with pegylated interferon alpha-2a in patients with essential thrombocythemia (ET) and polycythemia vera (PV). Exp Hematol Oncol. 2017 Nov 9;6:30. doi: 10.1186/s40164-017-0090-5. eCollection 2017.
PubMed 29152412
Masarova L, Patel KP, Newberry KJ, Cortes J, Borthakur G, Konopleva M, Estrov Z, Kantarjian H, Verstovsek S. Pegylated interferon alfa-2a in patients with essential thrombocythaemia or polycythaemia vera: a post-hoc, median 83 month follow-up of an open-label, phase 2 trial. Lancet Haematol. 2017 Apr;4(4):e165-e175. doi: 10.1016/S2352-3026(17)30030-3. Epub 2017 Mar 11.
PubMed 28291640

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Myelodysplastic Syndromes
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Active, not recruitingPhase 2
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All Myeloproliferative Disorders trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: PEG IFN-alpha2a (Pegasys®) Therapy in Patients With Chronic Myeloproliferative Diseases (Excluding Philadelphia Chromosome Positive Chronic Myeloid Leukemia)
ClinicalTrials.gov ID: NCT00452023
Acronym / sponsor ID: DM03-0109
Conditions: Myeloproliferative Disorders
Keywords: Myeloproliferative Disorders, Essential Thrombocythemia, Polycythemia Vera, Pegasys, IFN-alpha2a, Pegylated-Interferon Alpha-2A, PEG-IFNa-2a
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 83 participants (actual)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Study start: 7 Apr 2005 (actual)
Primary completion: 26 May 2023 (actual)
Study completion: 26 May 2023 (actual)
First posted: 26 Mar 2007 (estimated)
Results: Posted 11 Dec 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

IFN-alpha2a has been used for the treatment of a variety of disorders (such as hepatitis C). IFN-alpha2a is a drug that may affect the way infections and malignant diseases develop.

Treatment will continue (injections once a week) as long as the disease does not get worse.

If the disease gets worse or you experience any intolerable side effects, you will be taken off the study and your doctor will discuss other treatment options with you.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 5

Arm	Type	Receives
IFN-alpha2a 90 Microgram (mcg) weekly	Experimental	Drug: IFN-alpha2aStarting dose 90 microgram (mcg) injection under the skin once a week.
IFN-alpha2a 450 Microgram (mcg) weekly	Experimental	Drug: IFN-alpha2aStarting dose 450 microgram (mcg) injection under the skin once a week.
IFN-alpha2a 360 Microgram (mcg) weekly	Experimental	Drug: IFN-alpha2aStarting dose 360 microgram (mcg) injection under the skin once a week.
IFN-alpha2a 270 Microgram (mcg) weekly	Experimental	Drug: IFN-alpha2aStarting dose 270 microgram (mcg) injection under the skin once a week.
IFN-alpha2a 180 Microgram (mcg) weekly	Experimental	Drug: IFN-alpha2aStarting dose 180 microgram (mcg) injection under the skin once a week.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Following diagnoses: --ET: Patients with PLT > 600 x10 9 /l documented in the past 12 months; hyperplasia of marrow megakaryocytes in the absence of identifiable cause of thrombocytosis and in the absence of Ph chromosome. Patients with ET and lower PLT will be eligible if attributable to prior ET therapy. --PV: Patients should have Hb >/= 15g/dl (except if patient is having phlebotomies done) and documented past diagnosis.
Performance status </= 2 (ECOG scale).
Age greater than 18 years since disease is extremely rare in younger age group.
Adequate liver function: total bilirubin of </= 2.0 mg/dl (except for patients with Gilbert's Syndrome) and AST (SGOT) or ALT (SGPT) < 3 X ULN (or < 5 X ULN if considered due to tumor), and renal function (serum creatinine </= 2.0 mg/dl).
Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of the M.D. Anderson Cancer Center. The only acceptable consent form is the one approved by the M.D. Anderson Cancer Center IRB.

Key exclusion criteria (11 listed)

Pregnant or lactating women.
Patients with prior history of another malignancy or concurrent malignancy, except for the following: basal cell carcinoma of the skin, carcinoma in situ of the cervix, or other malignancies if the patient is disease free >3 years.
Patients with history of ischemic retinopathy.
Patients with history of severe cardiac disease: NYHA Functional Class III or IV, myocardial infarction within 6 months, uncontrolled ventricular tachyarrhythmias or unstable angina.
Patients with history of medically significant psychiatric disease if not controlled, especially endogenous depression (does not include reactive depression post-cancer diagnosis), psychosis and bipolar disease.

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
IFN-alpha2aAlso: Pegylated-Interferon Alpha-2A, PEG-IFNa-2a, PegasysStarting dose 90 microgram (mcg) injection under the skin once a week	Drug	IFN-alpha2a 180 Microgram (mcg) weekly, IFN-alpha2a 270 Microgram (mcg) weekly, IFN-alpha2a 360 Microgram (mcg) weekly, IFN-alpha2a 450 Microgram (mcg) weekly, IFN-alpha2a 90 Microgram (mcg) weekly

Primary outcome

Number of Patients With Complete Response (CR) or Partial Response (PR)
Time frame: Up to 18 years
CR = Reduction of PLT to <440x109/l and disappearance of thromboembolic events, without the use of anagrelide or hydroxyurea PR = Reduction of PLT by 50% but still >440x109/l or reduction of thromboembolic events by 50%, without the use of anagrelide or hydroxyurea

Results and safety

Results posted to the registry on 11 Dec 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
IFN-alpha2a 450 Microgram (mcg) Injection	3	3
IFN-alpha2a 360 Microgram (mcg) Injection	3	3
IFN-alpha2a 270 Microgram (mcg) Injection	19	19
IFN-alpha2a 180 Microgram (mcg) Injection	26	26
IFN-alpha2a 90 Microgram (mcg) Injection	32	32

Primary outcome: Number of Patients With Complete Response (CR) or Partial Response (PR)

Time frame: Up to 18 years. Count of participants. Unit: Participants

Measure	IFN-alpha2a 450 Microgram (mcg) n = 3	IFN-alpha2a 360 Microgram (mcg) n = 3	IFN-alpha2a 270 Microgram (mcg) n = 19	IFN-alpha2a 180 Microgram (mcg) n = 26	IFN-alpha2a 90 Microgram (mcg) n = 32
Value	3	3	14	23	23

Adverse events

Time frame: Up to 18 years, one month and 23 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
IFN-alpha2a 450 Microgram (mcg)	1 of 3 (33.3%)	2 of 3 (66.7%)	0 of 3
IFN-alpha2a 360 Microgram (mcg)	0 of 3 (0%)	3 of 3 (100%)	0 of 3
IFN-alpha2a 270 Microgram (mcg)	3 of 19 (15.8%)	9 of 19 (47.4%)	0 of 19
IFN-alpha2a 180 Microgram (mcg)	3 of 26 (11.5%)	8 of 26 (30.8%)	2 of 26
IFN-alpha2a 90 Microgram (mcg)	7 of 32 (21.9%)	7 of 32 (21.9%)	1 of 32

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29152412Masarova L, Yin CC, Cortes JE, Konopleva M, Borthakur G, Newberry KJ, Kantarjian HM, Bueso-Ramos CE, Verstovsek S. Histomorphological responses after therapy with pegylated interferon alpha-2a in patients with essential thrombocythemia (ET) and polycythemia vera (PV). Exp Hematol Oncol. 2017 Nov 9;6:30. doi: 10.1186/s40164-017-0090-5. eCollection 2017.
Derived from this study: PubMed 28291640Masarova L, Patel KP, Newberry KJ, Cortes J, Borthakur G, Konopleva M, Estrov Z, Kantarjian H, Verstovsek S. Pegylated interferon alfa-2a in patients with essential thrombocythaemia or polycythaemia vera: a post-hoc, median 83 month follow-up of an open-label, phase 2 trial. Lancet Haematol. 2017 Apr;4(4):e165-e175. doi: 10.1016/S2352-3026(17)30030-3. Epub 2017 Mar 11.
MD Anderson Cancer CenterLink provided by the sponsor.

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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.

Lucia Masarova, MD principal investigator

M.D. Anderson Cancer Center

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 IFN-alpha2a in people with Myeloproliferative Disorders, run by M.D. Anderson Cancer Center 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 7 inclusion and 11 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 11 Dec 2024. 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?

Yes. Results are posted in the registry (first posted 11 Dec 2024). 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 Patients With Complete Response (CR) or Partial Response (PR) (time frame: Up to 18 years). 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 NCT00452023, which also lists 2 linked publications.

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 Dec 2024.