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LeukemiaPhase 2NCT00350181

Sirolimus & Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor HCT

A Phase 2 study testing Sirolimus, MMF and BCNU in people with Leukemia and Lymphoma, Non-Hodgkin, run by Stanford University 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

GVHD prophylaxis of sirolimus and mycophenolate mofetil for patients undergoing matched related allogeneic transplant for acute and chronic leukemia, MDS, high risk NHL and HL Read more about this study

Who is it for?
Ages 2 to 60 years
What is being tested?
Sirolimus, MMF and BCNU
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
1 siteUnited States
Study dates
Aug 2006to Apr 2010
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 2 to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Disease Categories: (one of the following)
  • AML, age 2 - 60 years beyond 2nd remission or relapsed/refractory disease
  • AML, age 51-60 years of age, in first or subsequent remission or relapsed/refractory disease
  • AML with multilineage dysplasia
  • ALL, age 2 - 60 years beyond 2nd remission or relapsed/refractory disease
  • ALL, age 51 - 60 years in first or subsequent remission or relapsed/refractory disease
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  • CML Beyond 2nd chronic phase or in blast crisis
  • MDS; Includes World Health Organization classifications of refractory anemia with excess blasts-1 (RAEB-1), RAEB-2 and therapy-related MDS
  • Myeloproliferative disorders; MDS with poor long-term survival including myeloid metaplasia and myelofibrosis
  • High risk NHL in first remission
  • Relapsed or refractory NHL
  • HL beyond first remission
  • Males and females of any ethnic background 2 - 60 years of age
  • Karnofsky Performance Status ≥ 70% or Lansky performance status > 70% for patients < 16 years of age.
  • Matched related donor identified: 6/6 HLA-A, B and DRB1
  • Willingness to take oral medications during the transplantation period
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Prior myeloablative allogeneic or autologous HCT
  • HIV infection
  • Pregnant
  • Lactating females
  • Evidence of uncontrolled active infection
  • Organ Dysfunction:
Show all 15Show fewer
  • Serum creatinine > 1.5 mg/dL or 24 hour creatinine clearance < 50 ml/min
  • Direct bilirubin, ALT or AST > 2 x ULN
  • In adults DLCO < 60% predicted and in children room air oxygen saturation < 92%
  • In adults, left ventricular ejection fraction < 45% and in children, shortening fraction < 26%
  • Fasting Cholesterol > 300 mg/dL or Triglycerides > 300 mg/dL while on lipid-lowering agents.
  • Patients receiving investigational drugs unless cleared by the PI.
  • Patients with prior malignancies except basal cell carcinoma or treated carcinoma in-situ.
  • Cancer treated with curative intent > 5 years will be allowed.
  • Cancer treated with curative intent ≤ 5 years will not be allowed with PI approval.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Stanford University School of Medicine

    Stanford, California, 94305

Other trials in United States

About this study

Official title: Sirolimus and Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation

GVHD prophylaxis of sirolimus and mycophenolate mofetil for patients undergoing matched related allogeneic transplant for acute and chronic leukemia, MDS, high risk NHL and HL

Read the detailed description

To explore the novel combination of sirolimus and mycophenolate mofetil (MMF) as graft versus host disease (GVHD) prevention in HLA matched related donor blood or marrow transplantation (BMT). This study will report the toxicities associated with this drug combination and also explore possible correlations between specific blood cell types and antibody production during this therapy.

Being tested
Sirolimus, MMF, BCNU, VP-16, CY
Medical subject headings
Leukemia, Lymphoma, Non-Hodgkin, Hematologic Diseases

What participants receive

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

  • SirolimusDrug

    Also called rapamycin

    Immunosuppressant beginning day -3 with 12 mg oral loading dose, 4 mg/day orally for adults

  • MMFDrug

    Also called Mycophenolate Mofetil

    Immunosuppressant given through IV on day 0 at 15 mg/kg twice daily ≥ 2hr after the completion for the donor cell infusion

  • BCNUDrug

    Also called Carmustine, BiCNU

    15 mg/kg, IV

  • VP-16Drug

    Also called etoposide

    60 mg/kg, IV

  • CYDrug

    Also called cyclophosphamide, cytophosphane, Endoxan

    For subjects aged 18-60 with lymphoma 100 mg/kg, IV For subjects aged with AML, ALL or CML 18-50 60 mg/kg, IV For subjects aged 51-60 with MDS, AML or ALL or patients age 18-60 with MDS, secondary AML or non-CML myeloproliferative disease 45 mg/kg

  • FTBIDrug

    Also called total body irradiation

    1320 cGy delivered in 11 120 cGy fractions over 4 day

  • BUDrug

    Also called busulfan

    BU 1 mg/kg every 6hr x 4 doses, IV

Study groups in NCT00350181
Study groupRoleWhat they receive
Regimen Treatment 1ExperimentalFor subjects 18-60 years old with lymphoma: (BCNU+ VP-16 +CY) BCNU 15 mg / kg (maximum dose 550 mg/m² actual body weight) on day -6. VP 60 mg / kg on day 4 and CY 100 mg / kg on day -2. Followed by Sirolimus and MMF as prophylaxis
Receives: Drug: Sirolimus; Drug: MMF; Drug: BCNU; Drug: VP-16; Drug: CY
Regimen Treatment 2ExperimentalFor subjects 18-50 years old with AML, ALL or CML: (VP-16 +CY+ FBI) Patients aged 18-50 years with AML, ALL or CML: FTBI 1320 cGy delivered in 11 120 cGy fractions over 4 days on days -8 through -5. VP 60 mg / kg on day -4 and CY 60 mg / kg on day -2. Followed by Sirolimus and MMF as prophylaxis
Receives: Drug: Sirolimus; Drug: MMF; Drug: VP-16; Drug: CY; Drug: FTBI
Regimen Treatment 3ExperimentalFor subjects 51-60 years with MDS, AML or ALL or 18-60 with MDS, secondary AML pr non-CML myeloproliferative disease: (BU+ VP-16 +CY) BU 1 mg/kg every 6 hours X 14 doses on days -9 through -6 with target concentration at steady state of X 800 ng / ml based on first dose pharmacokinetics. VP 60 mg / kg on day -5 and CY 45 mg / kg per day -2 days on day -3 and day -2. Followed by Sirolimus and MMF as prophylaxis
Receives: Drug: Sirolimus; Drug: MMF; Drug: VP-16; Drug: CY; Drug: BU

What is being measured

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

  1. To evaluate the incidence of grade II-IV acute GVHD with sirolimus and mycophenolate mofetil GVHD prophylaxis.

    Measured: D+100 post-transplant

Timeline

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

  1. First posted10 Jul 2006 (estimated)
  2. Study startAug 2006
  3. Primary completionAug 2007
  4. Study completionApr 2010

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
Parallel groups
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00350181
Sponsor's protocol ID
IRB-06112
Other IDs
97168, BMT184, 6112
Status last verified
Sep 2021
First submitted
5 Jul 2006
Last updated
23 Sep 2021
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Johnston L, Florek M, Armstrong R, McCune JS, Arai S, Brown J, Laport G, Lowsky R, Miklos D, Shizuru J, Sheehan K, Lavori P, Negrin R. Sirolimus and mycophenolate mofetil as GVHD prophylaxis in myeloablative, matched-related donor hematopoietic cell transplantation. Bone Marrow Transplant. 2012 Apr;47(4):581-8. doi: 10.1038/bmt.2011.104. Epub 2011 May 9.

    PubMed 21552302

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sirolimus, MMF and BCNU in people with Leukemia and Lymphoma, Non-Hodgkin, run by Stanford University 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 17 inclusion and 15 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 23 Sep 2021. 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?

To evaluate the incidence of grade II-IV acute GVHD with sirolimus and mycophenolate mofetil GVHD prophylaxis. (time frame: D+100 post-transplant). 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 NCT00350181, 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 23 Sep 2021.

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