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MyelofibrosisPhase 2NCT00572897

Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis

A Phase 2 study testing Fludarabine, Melphalan +/- ATG in people with Myelofibrosis, run by John Mascarenhas at 12 sites in 4 countries.

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

Stem cell transplantation is used to treat may types of diseases. There a 2 types of transplants, conventional (very intense) and reduced intensity-non-myeloablative, also called mini-transplants. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Myelofibrosis
What is being tested?
Fludarabine, Melphalan +/- ATG
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
66 participants
Where
12 sitesin 4 countries
Study dates
Aug 2007to 15 Jun 2015
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 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients with the following disease: Idiopathic myelofibrosis, or spent PV-, or ET-related myelofibrosis in chronic phase (<20% blast cells in the bone marrow) with Lille score >1 at any time, or platelet <100K.
  • Age 18-65 years.
  • ECOG performance status < 3.
  • Life expectancy >3 months.
  • Adequate cardiac function, normal LVEF ≥ 45% by MUGA or echocardiogram and adequate pulmonary function DLCO ≥ 50% of predicted.
  • Serum creatinine < 1.1 x the upper limit of normal (ULN) or Creatinine Clearance >50 ml/min.
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  • Serum bilirubin < 2.0 mg/dl, SGPT <2.5 x upper limit of normal
  • No evidence of chronic active hepatitis or cirrhosis
  • HIV-negative
  • Patient is not pregnant
  • Patient or guardian able to sign informed consent.
  • Patients with >20% myeloblasts in the blood or marrow, extramedullary blast cell proliferation or large foci of blasts in bone marrow biopsy specimens are not eligible.
  • Pretransplant splenectomy: MMM patients with variable degrees of splenomegaly, or splenectomized, are eligible to be enrolled. Any decision of having a patient splenectomized prior to transplant will be made in each center prior to enrolling the patient in the study.
  • Patients should be off treatment with investigational for at least 4 weeks and have recovered from all toxicities.

Who cannot join

  • Pregnancy
  • HIV positive
  • > 20% myeloblasts in the peripheral blood or bone marrow
  • LVEF < 45%
  • DLCO < 50% of predicted
  • ECOG performance status ≥ 3
  • Chronic active hepatitis or cirrhosis
  • Chronic renal insufficiency

Trial locations and contacts

12 sites in 4 countries.

12 sites in 4 countries

United States7 sites

  • Johns Hopkins

    Baltimore, Maryland, 21205

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

  • University of Illinois at Chicago

    Chicago, Illinois, 60612

  • Ohio State Univesity

    Columbus, Ohio, 43210

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Weill Cornell Medical College

    New York, New York, 10065

  • University of Utah

    Salt Lake City, Utah, 84132

Other trials in United States

Italy3 sites

  • Ospedali Riuniti di Bergamo

    Bergamo, Bergamo, 24128

  • University of Florence

    Florence, IL, 60302

  • University of San Martino

    San Martino

Other trials in Italy

Canada1 site

  • Princess Margaret Hospital

    Toronto, Ontario, M5G2M9

Other trials in Canada

Sweden1 site

  • Regionala etikprovningsnamnden Goteborg

    Gothenburg, 60302

Other trials in Sweden

About this study

Stem cell transplantation is used to treat may types of diseases. There a 2 types of transplants, conventional (very intense) and reduced intensity-non-myeloablative, also called mini-transplants.

This study proposes to use a conditioning regimen for allogeneic transplantation along with a reduced intensity transplant. Conditioning regiment is the name for the combination of chemotherapy drugs that is given to patients before receiving a transplantation of donor stem cells. It is hoped that the regimen designed for this study proves to be less toxic and has an equal or better anticancer effect than the regimens that are normally used. The regimen being used is a combination of two chemotherapy drugs, fludarabine and melphalan. This regimen has been studied in recipients of matched sibling transplants and in recipients of alternative donor stem cells in other hematologic malignancies. Those subjects, who receive stem cells from an unrelated donor, will also receive and additional drug called ATG or anti thymocyte globulin. ATG suppresses the immune system, thus reducing the chances for the recipient rejecting the transplant (graft).

Read more

The purpose of this study is to observe if reduced intensity transplants can be used to allow engraftment or "take" of the donor's bone marrow. Studies conducted in the past show this type of transplant is much less toxic than traditional bone marrow transplants. Reduced intensity transplants may be better tolerated by patients who may experience serious side effects from standard (very intense) stem cell transplant.

The study has been recently amended to follow all subjects for survival.

Read the detailed description

This study is designed as a single arm Phase II clinical trial in patients with myelofibrosis who are eligible for transplantation from a related donor or from an unrelated donor source. Patients will be accrued into two separate strata defined by donor type. Each of the two strata will be analyzed separately.

Patients will be followed yearly from time of enrollment into the study to assess clinical response and overall, progression and event free survival, as well as incidence and degree of acute and chronic GVHD. We will estimate cumulative survival and transplant related mortality in patients enrolled in each of the two strata.

Conditions
Myelofibrosis
Being tested
Fludarabine, Melphalan +/- ATG
Keywords
Myelofibrosis, Myeloproliferative disease, Stem Cell Transplant, Allogeneic Transplant, Conditioning Regimen, Reduced Intensity Regimen, Fludarabine, Melphalan, ATG
Medical subject headings
Primary Myelofibrosis, Myeloproliferative Disorders

What participants receive

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

  • Fludarabine, Melphalan +/- ATGDrug

    Conditioning regimen for Allogenic Stem Cell Transplant:

    Related Donor Fludarabine days -6 to -2 (30mg/m2 IVPB over 30 minutes daily) Melphalan days -3 to -2 (70mg/m2 IVPB over 30 minutes daily)

    Unrelated Donor Fludarabine days -6 to -2 (30mg/m2 IVPB over 30 minutes daily) Melphalan days -3 to -2 (70mg/m2 IVPB over 30 minutes daily) ATG (Thymoglobulin®) days -3 to -1 (0.5 mg/kg IV on day -3 [given over 6 hours], and 2 mg/kg on days -2 and -1 [given over 4 hours])

Study groups in NCT00572897
Study groupRoleWhat they receive
Fludarabine, Melphalan +/- ATGExperimentalFludarabine, Melphalan +/- ATG
Receives: Drug: Fludarabine, Melphalan +/- ATG

What is being measured

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

  1. The Primary Endpoint is Progression-free Survival.

    Number of participants alive at 2 years who are progression-free

    Measured: 2 years

Secondary outcomes (5)

  1. Response Outcomes

    assessed according to the IWG Criteria

    Measured: 180 days

  2. Overall Survival

    The number of patients alive at last follow-up.

    Measured: 73 months

  3. Absolute Neutrophil Count (ANC)

    Patients with ANC ≥0.5 × 10^9/L

    Measured: 2 years

  4. PLT

    Patients with PLT ≥20 × 109/L

    Measured: 2 years

  5. Transplant-related Mortality

    Transplant-related Mortality including Graft-versus-host disease (GVHD)

    Measured: 2 years

Timeline

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

  1. First posted13 Dec 2007 (estimated)
  2. Study startAug 2007
  3. Primary completionAug 2011
  4. Study completion15 Jun 2015

Resources

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
66 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00572897
Sponsor's protocol ID
GCO 07-0548-00101
Other IDs
P01CA108671-01A2, MPD-RC 101
Status last verified
Apr 2017
First submitted
11 Dec 2007
Last updated
15 May 2017
Data monitoring committee
Yes

Publications

  • Rondelli D, Goldberg JD, Isola L, Price LS, Shore TB, Boyer M, Bacigalupo A, Rambaldi A, Scarano M, Klisovic RB, Gupta V, Andreasson B, Mascarenhas J, Wetzler M, Vannucchi AM, Prchal JT, Najfeld V, Orazi A, Weinberg RS, Miller C, Barosi G, Silverman LR, Prosperini G, Marchioli R, Hoffman R. MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. Blood. 2014 Aug 14;124(7):1183-91. doi: 10.1182/blood-2014-04-572545. Epub 2014 Jun 24.

    PubMed 24963042

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Fludarabine, Melphalan +/- ATG in people with Myelofibrosis, run by John Mascarenhas at 12 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 14 inclusion and 8 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 15 May 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States, Italy, Canada, Sweden. 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 2 May 2016). 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?

The Primary Endpoint is Progression-free Survival. (time frame: 2 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 NCT00572897, 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 15 May 2017.

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