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Myeloproliferative DisordersPhase 2NCT00787761

Early Administration of ATG Followed by Cyclophosphamide, Busulfan and Fludarabine Before a Donor Stem Cell Transplant in Patients With Hematological Cancer

A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Myeloproliferative Disorders and Kidney Cancer, run by Northside Hospital, Inc. 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
Search all trials

Trial summary

RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. Read more about this study

Who is it for?
Ages Up to 75 years
What is being tested?
anti-thymocyte globulin, busulfan and cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteUnited States
Study dates
Apr 2007to May 2012
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 Up to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

DISEASE CHARACTERISTICS:

  • Histologically confirmed diagnosis of one of the following:
  • Chronic myeloid leukemia (CML)
  • Philadelphia chromosome (Ph)- and/or BCR-ABL-positive disease
  • In chronic or accelerated phase
  • Suboptimal response to imatinib mesylate (i.e., no hematologic complete response by 3 months, no major cytogenetic response by 6 months, or no complete cytogenetic response by 1 year)
  • CML in blastic transformation allowed provided patient achieved complete remission (CR) or second chronic phase after treatment with imatinib mesylate or chemotherapy
  • Chronic lymphocytic leukemia meeting one of the following criteria:
  • Rai stage III or IV disease
  • Rai stage I or II disease that failed standard therapy (i.e., disease is progressing after ≥ 1 course of standard therapy)
  • Non-Hodgkin lymphoma (NHL) meeting one of the following criteria:
  • Indolent NHL
  • Clinical stage III or IV disease or bulky stage II disease (i.e., ≥ one lymphoid mass > 5 cm in ≥ one dimension)
  • Relapsed after primary therapy OR is refractory to therapy
  • Aggressive NHL
  • Is not considered curable with standard chemotherapy or autologous stem cell transplantation (i.e., relapsed after autologous stem cell transplantation)
  • Chemotherapy-responsive disease
  • Multiple myeloma
  • Durie-Salmon stage II or III disease
  • Durie Salmon stage I disease allowed provided β2 microglobulin level > 3 mg/dL
  • Acute myeloid leukemia or acute lymphocytic leukemia
  • In CR (defined as < 5% blasts in bone marrow and no circulating blasts) AND has any of the following poor prognostic features:
  • WBC > 100,000/mm^3 at presentation
  • In second or greater remission
  • Adverse-risk cytogenetics (i.e., Ph1-positive, 11q23 translocation, -5, -7, complex translocations, or other recognized adverse-risk cytogenetics)
  • Renal cell carcinoma
  • Stage IV disease
  • Clear cell morphology
  • Myelodysplastic syndromes
  • Bone marrow blasts ≤ 10% on last bone marrow biopsy prior to transplantation
  • Myeloproliferative disease
  • Anticipated life expectancy on conventional therapy < 10 years
  • No uncomplicated essential thrombocythemia or primary polycythemia
  • Hodgkin lymphoma
  • Relapsed after ≥ 1 standard-dose chemotherapy regimen
  • Not considered curable by autologous stem cell transplantation
  • No clinical evidence of active CNS involvement
  • Previously treated leptomeningeal disease allowed provided CSF cytology is negative at the time of assessment for transplantation
  • Available 6/6 allele match (i.e., HLA-A, B, DRβ1)matched related donor

PATIENT CHARACTERISTICS:

  • ECOG performance status (PS) 0-2 OR Karnofsky PS 60-100%
  • Bilirubin < 3 times normal (unless abnormality due to malignancy)
  • AST and ALT < 3 times normal (unless abnormality due to malignancy)
  • Creatinine ≤ 2.0 mg/dL
  • LVEF ≥ 40% by MUGA or ECHO
  • DLCO ≥ 40% of predicted
  • FEV-1 ≥ 50% of predicted
  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • Deemed to be an appropriate candidate for allogeneic SCT
  • No evidence of myocardial infarction within the past 6 months
  • No psychological or social condition that may interfere with study participation
  • No serious uncontrolled localized or active systemic infection
  • No second malignancy within the past 3 years except for completely excised nonmelanotic skin cancer or in situ carcinoma of the cervix
  • No chronic inflammatory disorder requiring the continued use of glucocorticoids or other immunosuppressive medications
  • No known HIV positivity
  • No hypersensitivity to E. coli-derived proteins

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Blood and Marrow Transplant Group of Georgia

    Atlanta, Georgia, 30342

Other trials in United States

About this study

Official title: Pre-administration of Rabbit Antithymocyte Globulin to Optimize Donor T-Cell Engraftment Following Reduced Intensity Allogeneic Peripheral Blood Progenitor Cell Transplantation From Matched-Related Donors

RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving antithymocyte globulin before the transplant and tacrolimus and methotrexate after the transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well giving antithymocyte globulin together with cyclophosphamide, busulfan, and fludarabine works in treating patients with hematological cancer or kidney cancer undergoing donor stem cell transplant.

Read the detailed description

OBJECTIVES:

  • To assess the percentage of patients with hematological malignancies or renal cell carcinoma who achieve > 90% donor T-cell chimerism at 30 days after treatment with reduced-intensity conditioning comprising low-dose anti-thymocyte globulin, low-dose cyclophosphamide, busulfan, and fludarabine phosphate followed by allogeneic peripheral blood progenitor cell transplantation from a matched related donor.
  • To assess the incidence of severe (grade 3 or 4) acute graft-versus-host disease (GVHD) and extensive chronic GVHD in these patients.
  • To assess whether this regimen is associated with reduced transplant-related toxicity and increased tolerability in these patients.
  • To assess the overall safety of this conditioning regimen as measured by 6-month transplant-related mortality in these patients.
  • To determine the efficacy of this regimen in inducing durable remissions in these patients.

OUTLINE:

  • Reduced-intensity conditioning (RIC): Patients receive anti-thymocyte globulin IV over 4-6 hours on day -16 and over 6-8 hours on day -15, fludarabine phosphate IV over 30 minutes on days -7 to -3, busulfan IV over 3 hours on days -4 and -3, and cyclophosphamide IV over 1-2 hours on day -2.
  • Transplantation: Patients undergo allogeneic peripheral blood progenitor cell transplantation on day 0.
  • Graft-vs-host disease (GVHD) prophylaxis: Patients receive oral tacrolimus every 12 hours on days -1 to 90, followed by a taper until day 150. Patients also receive methotrexate IV on days 1, 3, and 6.

Blood samples are collected periodically for pharmacokinetic studies of anti-thymocyte globulin and PCR analysis for chimerism.

After completion of study therapy, patients are followed periodically for up to 3 years.

Being tested
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, methotrexate
Keywords
accelerated phase CML, blastic phase CML, Philadelphia chromosome positive CML, stage I CLL, stage II CLL, stage III CLL, stage IV CLL, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, adult AML with 11q23 (MLL) abnormalities, adult CML in remission, adult ALL in remission, stage IV renal cell cancer, de novo MDS, previously treated MDS, secondary MDS, atypical CML, BCR-ABL negative, chronic myelomonocytic leukemia, MDS/myeloproliferative neoplasm, unclassifiable
Medical subject headings
Myeloproliferative Disorders, Kidney Neoplasms, Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases

What participants receive

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

  • anti-thymocyte globulinBiological

    Also called Thymoglobulin, ATG

    Anti-Thymocyte Globulin (ATG) is commercially available. The 1st vial contains 25 mg ATG, and the 2nd vial contains > 5 mL SWFI diluent. Ampuls must be refrigerated (2º C - 8º C). Do not freeze.

    Reconstitute 25 mg vial with diluent provided by manufacturer. Roll vial gently to dissolve powder. Use contents of vial within 4 hours. Dilute dosage to a final concentration of 0.5 mg/mL in 0.9% sodium chloride injection or 5% dextrose injection. Gently invert admixture 1-2 times to mix. Use admixture solution immediately.

    Infuse the 1st dose over at least 6 hours, and subsequent doses over at least 4 hours. Infuse through a 0.22 micron in-line filter. Premeds include acetaminophen 650 mg PO, diphenhydramine 25-50 mg PO/IV, and methyprednisolone 1mg/kg at the initiation and half-way through ATG administration.

  • busulfanDrug

    Commercially available in 60 mg/10 mL ampuls.

    Dilute busulfan injection in 0.9% sodium chloride injection or dextrose 5% in water. The dilution volume should be 10 times the volume of busulfan injection, ensuring that the final concentration of busulfan is ≥ 0.5 mg/mL.

    Store unopened ampuls at 2º C to 8º C. The diluted solution is stable for up to 8 hours at room temperature (25º C) but the infusion must also be completed within that 8-hour time frame. Dilution of busulfan injection in 0.9% sodium chloride is stable for up to 12 hours under refrigeration (2º C to 8º C) but the infusion must also be completed within that 12-hour time frame.

    IV Bu should be administered via a central venous catheter as a 2-hour infusion every 6 hours for 2 consecutive days for a total of 8 doses.

  • cyclophosphamideDrug

    Cyclophosphamide is commercially available. Cyclophosphamide for injection is available in 2000 mg vials which are reconstituted with 100 ml sterile water for injection.

    The concentration of the reconstituted product is 20 mg/ml. The calculated dose will be diluted further in 250-500 ml of Dextrose 5% in water.

    Reconstituted solutions of lyophilized cyclophosphamide are chemically and physically stable for 24 hours at room temperature or for 6 days in the refrigerator. Specific temperatures are not provided by the manufacturer. Reconstitution of cyclophosphamide with bacteriostatic water containing benzyl alcohol preservative may result in decomposition.

    Each dose will be infused over 1-2 hr (depending on the total volume).

  • fludarabine phosphateDrug

    Also called Fludara

    Fludarabine is commercially available as a white, lyophilized powder. Each vial contains 50 mg of fludarabine, 50 mg of mannitol and sodium hydroxide to adjust pH.

    Intact vials should be stored under refrigeration. Reconstituted vials are stable for 16 days and solutions diluted in D5W or NS are stable for 48 hours at room temperature or under refrigeration.

    Fludarabine should be reconstituted with 2 mL of Sterile Water for Injection, USP. Each mL of the resulting solution will contain 25 mg of fludarabine, 25 mg of mannitol, and sodium hydroxide to adjust the pH to 7-8.5. The product should be further diluted for intravenous administration in 5% Dextrose for Injection, USP or in 0.9% Sodium Chloride, USP.

    Fludarabine will be administered as an IV infusion over 30 minutes.

  • methotrexateDrug

    Also called Amethopterin, MTX

    Commercially available for injection in 2 mL (2.5 mg/mL), 2 mL, 4 mL, 8 mL, 10 mL (25 mg/mL) vials, or 20 mg, 25 mg, 50 mg, or 100 mg vials for reconstitution.

    Vials requiring reconstitution should be reconstituted to a concentration of 25 mg/mL.

    Intact vials should be stored at room temperature and protected from light. Once opened, solutions containing preservatives are stable for 4 weeks at room temperature and up to 3 months refrigerated.

    Administer via slow IV push.

  • tacrolimusDrug

    Also called Prograf, FK506

    Tacrolimus is commercially available as an injection (5 mg/mL; 1 mL ampuls) and as oral capsules (1 mg and 5 mg).

    Tacrolimus injection must be diluted prior to IV infusion with 0.9% sodium chloride or 5% dextrose injection to a concentration of 4-20 μg/mL. Solutions should be prepared in non-PVC plastic or glass. Tacrolimus injection and diluted solutions of the drug should be inspected visually for particulate matter and discoloration prior to administration whenever solution and container permit.

    Oral therapy should be started as possible as per protocol and 8 to 12 hours after stopping intravenous therapy. Oral doses will be administered twice a day.

    Store tacrolimus capsules and injection at controlled room temperature, 15-30º C (59-86º F).

  • nonmyeloablative allogeneic HSCTProcedure or surgery

    As demonstrated by groups in Houston, Jerusalem \& Seattle, RICT has been used to treat hematologic \& solid malignancies with related \& unrelated donors. Although adequate comparisons of RICT versus ablative alloHCT remain to be reported, the studies of RICT so far suggest that TRM is generally less than would be expected for similar patients undergoing ablative alloHCT; incidence of acute \& chronic GVHD is similar or less than ablative alloHCT; autologous hematopoietic recovery is more common than seen following ablative alloHCT if graft failure occurs; powerful GVT effects can be seen but are dependent on high levels of donor T-cell chimerism and RICT are less effective than ablative alloHCT in controlling aggressive malignancies

Study groups in NCT00787761
Study groupRoleWhat they receive
ATG, Cytoxan, Bu/Flu based Allogeneic TransplantExperimentalAll patients will receive an ATG, Cyclosphosphamide, Busulfan and Fludarabine based Allogeneic Transplant
Receives: Biological: anti-thymocyte globulin; Drug: busulfan; Drug: cyclophosphamide; Drug: fludarabine phosphate; Drug: methotrexate; Drug: tacrolimus; Procedure: nonmyeloablative allogeneic HSCT

What is being measured

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

  1. Achievement of > 90% (Full) Donor Chimerism in the T-cell Lineage as Measured by PCR at Day 30 Post-transplantation

    Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism.

    Measured: Day 30

Secondary outcomes (7)
  1. T-cell and Myeloid Chimerism at Days 90 Post-transplantation (>90% Chimerism)

    Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism.

    Measured: Day 90

  2. T-cell and Myeloid Chimerism at Days 180 Post-transplantation (>90%)

    Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism.

    Measured: 180 days

  3. Number of Patients Who Experience Severe (Grade 3 or 4) Acute Graft-versus-host Disease

    number of patients who experienced post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea.

    Measured: Day 100

  4. Number of Patients Experiencing Extensive Chronic Graft Versus Host Disease (GVHD)

    Patients who had post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea.

    Measured: 2 years

  5. Non-relapse Mortality (NRM) at Day 180 Post-transplantation

    non-relapse mortality refers to the death of a patient for causes other than relapsed disease.

    Measured: Day 180

  6. Disease-free Survival (DFS) at 24 Months

    Disease Free survival is measured by the amount of time a patient spends in a disease free state after being transplanted.

    Measured: 24 months

  7. Overall Survival (OS) at 24 Months

    Overall survival refers to the length of time a patient is alive after transplant regardless of whether they have progressive or relapsed disease.

    Measured: 24 months

Timeline

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

  1. First posted10 Nov 2008 (estimated)
  2. Study startApr 2007
  3. Primary completionJul 2010
  4. Study completionMay 2012

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00787761
Sponsor's protocol ID
CDR0000618483
Other IDs
BMTGG-NSH-807
Status last verified
Aug 2012
First submitted
7 Nov 2008
Last updated
1 Oct 2012
Data monitoring committee
No

Publications

  • Rao SS, Peters SO, Crittenden RB, Stewart FM, Ramshaw HS, Quesenberry PJ. Stem cell transplantation in the normal nonmyeloablated host: relationship between cell dose, schedule, and engraftment. Exp Hematol. 1997 Feb;25(2):114-21.

    PubMed 9015211
  • Storb R, Yu C, Wagner JL, Deeg HJ, Nash RA, Kiem HP, Leisenring W, Shulman H. Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation. Blood. 1997 Apr 15;89(8):3048-54.

    PubMed 9108426
  • Giralt S, Estey E, Albitar M, van Besien K, Rondon G, Anderlini P, O'Brien S, Khouri I, Gajewski J, Mehra R, Claxton D, Andersson B, Beran M, Przepiorka D, Koller C, Kornblau S, Korbling M, Keating M, Kantarjian H, Champlin R. Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy. Blood. 1997 Jun 15;89(12):4531-6.

    PubMed 9192777
  • Slavin S, Nagler A, Naparstek E, Kapelushnik Y, Aker M, Cividalli G, Varadi G, Kirschbaum M, Ackerstein A, Samuel S, Amar A, Brautbar C, Ben-Tal O, Eldor A, Or R. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998 Feb 1;91(3):756-63.

    PubMed 9446633
  • McSweeney PA, Niederwieser D, Shizuru JA, Sandmaier BM, Molina AJ, Maloney DG, Chauncey TR, Gooley TA, Hegenbart U, Nash RA, Radich J, Wagner JL, Minor S, Appelbaum FR, Bensinger WI, Bryant E, Flowers ME, Georges GE, Grumet FC, Kiem HP, Torok-Storb B, Yu C, Blume KG, Storb RF. Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects. Blood. 2001 Jun 1;97(11):3390-400. doi: 10.1182/blood.v97.11.3390.

    PubMed 11369628
  • Champlin R, Khouri I, Anderlini P, De Lima M, Hosing C, McMannis J, Molldrem J, Ueno N, Giralt S. Nonmyeloablative preparative regimens for allogeneic hematopoietic transplantation. Biology and current indications. Oncology (Williston Park). 2003 Jan;17(1):94-100; discussion 103-7.

    PubMed 12599934
  • Platzbecker U, Ehninger G, Schmitz N, Bornhauser M. Reduced-intensity conditioning followed by allogeneic hematopoietic cell transplantation in myeloid diseases. Ann Hematol. 2003 Aug;82(8):463-468. doi: 10.1007/s00277-003-0680-7. Epub 2003 Jun 21.

    PubMed 12910373
  • Storb R. Non-myeloablative allogeneic transplantation -- state-of-the-art. Pediatr Transplant. 2004 Jun;8 Suppl 5:12-8. doi: 10.1111/j.1398-2265.2004.00189.x.

    PubMed 15125701

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Myeloproliferative Disorders and Kidney Cancer, run by Northside Hospital, Inc. 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?

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 1 Oct 2012. 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 1 Oct 2012). 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?

Achievement of > 90% (Full) Donor Chimerism in the T-cell Lineage as Measured by PCR at Day 30 Post-transplantation (time frame: Day 30). 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 NCT00787761, which also lists 8 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 1 Oct 2012.

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