AzTrials
AzTrials

LeukemiaPhase 1NCT00068302

Sirolimus in Treating Young Patients With Relapsed or Refractory Acute Leukemia or Non-Hodgkin's Lymphoma

A Phase 1 study testing sirolimus in people with Leukemia and Lymphoma, run by Children's Hospital of Philadelphia at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

RATIONALE: Drugs used in chemotherapy such as sirolimus use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of sirolimus in treating young patients with relapsed or refractory acute leukemia or non-Hodgkin's lymphoma. Read more about this study

Who is it for?
Ages Up to 21 years
Conditions the study is for
Leukemia, Lymphoma
What is being tested?
sirolimus
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
Jan 2003to Jul 2013
Phase
Phase 1
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 Up to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Histologically confirmed diagnosis of 1 of the following:
  • Acute lymphoblastic leukemia (ALL) OR acute myeloid leukemia (AML)
  • At least 25% blasts in the bone marrow
  • Recurrent or refractory disease
  • Non-Hodgkin's lymphoma (NHL)
  • Second or greater relapse as determined by physical or radiological evidence
  • Disease for which there is no known curative therapy

PATIENT CHARACTERISTICS:

Age

  • 21 and under

Performance status

  • Karnofsky 50-100% (patients over 10 years of age)
  • Lansky 50-100% (patients 10 years of age and under)

Life expectancy

  • At least 4 weeks

Hematopoietic

  • Absolute neutrophil count at least 1,000/mm^3*
  • Platelet count at least 75,000/mm^3 (transfusion independent)*
  • Hemoglobin at least 8.0 g/dL (may receive red blood cells (RBC) transfusions)* NOTE: *Patients with ALL, AML, and NHL with tumor metastatic to bone marrow, with granulocytopenia, anemia, and/or thrombocytopenia are eligible, but will not be evaluable for hematological toxicity

Hepatic

  • Bilirubin no greater than 1.5 times normal
  • alanine aminotransferase (ALT) no greater than 5 times normal
  • Albumin at least 2 g/dL

Renal

  • Creatinine based on age, as follows:
  • No greater than 0.8 mg/dL (5 years of age and under)
  • No greater than 1.0 mg/dL (6 to 10 years of age)
  • No greater than 1.2 mg/dL (11 to 15 years of age)
  • No greater than 1.5 mg/dL (over 15 years of age) OR
  • Creatinine clearance or radioisotope glomerular filtration rate at least 70 mL/min

Cardiovascular

  • Shortening fraction at least 28% by echocardiogram OR
  • Ejection fraction at least 50% by gated radionuclide

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Able to ingest oral medication
  • No known allergy to sirolimus, tacrolimus, or other mammalian target of rapamycin (mTOR) inhibitors
  • No uncontrolled active infection
  • Fungal disease must be stable for at least 2 weeks prior to study entry
  • Documented negative blood cultures prior to study entry for patients with bacteremia
  • No active graft-versus-host disease

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Recovered from prior immunotherapy
  • More than 1 week since prior hematopoietic growth factors except for epoetin alfa
  • At least 7 days since prior biologic antineoplastic agents
  • At least 3 months since prior bone marrow or stem cell transplantation

Chemotherapy

  • Recovered from all prior chemotherapy
  • More than 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas)
  • Prior hydroxyurea within the past 2 weeks is allowed provided peripheral blast count has been stable or rising for at least 3 days

Endocrine therapy

  • Prior corticosteroids within the past 2 weeks are allowed provided peripheral blast count has been stable or rising for at least 3 days

Radiotherapy

  • Recovered from prior radiotherapy
  • At least 2 weeks since prior local palliative radiotherapy
  • At least 4 weeks since prior craniospinal radiotherapy or radiation to the pelvis of 50% or more
  • At least 4 weeks since prior substantial bone marrow radiotherapy
  • No concurrent radiotherapy, except for emergent situations or persistent extramedullary disease with resolution of bone marrow disease

Surgery

  • Not specified

Other

  • No other concurrent investigational antineoplastic drugs
  • No concurrent administration of any of the following:
  • Ketoconazole
  • Tacrolimus
  • Cyclosporine
  • Rifampin
  • Diltiazem

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: A Phase I Trial Of Sirolimus In Relapsed/Refractory Leukemia And Non-Hodgkin's Lymphoma

RATIONALE: Drugs used in chemotherapy such as sirolimus use different ways to stop cancer cells from dividing so they stop growing or die.

PURPOSE: This phase I trial is studying the side effects and best dose of sirolimus in treating young patients with relapsed or refractory acute leukemia or non-Hodgkin's lymphoma.

Read the detailed description

OBJECTIVES:

  • Determine the maximum tolerated dose of sirolimus in pediatric patients with refractory or relapsed acute leukemia or non-Hodgkin's lymphoma.
  • Determine the dose-limiting toxic effects of this drug in these patients.
  • Determine the trough levels produced by this drug in these patients.
  • Determine the anti-leukemia/lymphoma activity of this drug in these patients.

OUTLINE: This is an open-label, dose-escalation study.

Patients receive oral sirolimus once daily on days 1-21. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of sirolimus until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed for survival.

PROJECTED ACCRUAL: A total of 3-30 patients will be accrued for this study within 2 years.

Conditions
Leukemia, Lymphoma
Being tested
sirolimus
Keywords
recurrent childhood lymphoblastic lymphoma, recurrent childhood small noncleaved cell lymphoma, recurrent childhood large cell lymphoma, recurrent childhood acute myeloid leukemia, recurrent childhood acute lymphoblastic leukemia
Medical subject headings
Leukemia, Lymphoma, Burkitt Lymphoma, Dendritic Cell Sarcoma, Interdigitating, Precursor Cell Lymphoblastic Leukemia-Lymphoma

What participants receive

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

  • sirolimusDrug

    Also called rapamycin, Rapamune

    3-6 subjects will be enrolled into each dose level

Study groups in NCT00068302
Study groupRoleWhat they receive
SirolimusExperimentalThis is a dose escalation study including 4-dose levels. Subjects will receive a one-time loading dose of sirolimus on day 0, time 0. Subsequent dosing at the assigned dose level will start 24 hours following the initial loading dose
Receives: Drug: sirolimus

What is being measured

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

  1. Toxicity as assessed by Common Toxicity Criteria (CTC) toxicity criteria after the first course of treatment

    Subjects will be assessed for toxicity on days 3, 7 and 21

    Measured: within 21 days following administration of sirolimus

Secondary outcomes (1)
  1. Response as assessed by radiologic scans after each course of treatment

    Response will be assessed on day 21 of cycle 1

    Measured: day 21

Timeline

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

  1. First posted11 Sep 2003 (estimated)
  2. Study startJan 2003
  3. Primary completionApr 2009
  4. Study completionJul 2013

Why it stopped: Recruiting/enrolling participants halted prematurely but potentially will resume

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00068302
Sponsor's protocol ID
CDR0000321392
Other IDs
CHP-755, CHP-IRB-2002-12-3086
Status last verified
Jul 2013
First submitted
10 Sep 2003
Last updated
12 Mar 2015
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing sirolimus in people with Leukemia and Lymphoma, run by Children's Hospital of Philadelphia 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: stopped early, as of the sponsor's last update on 12 Mar 2015. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 was stopped early (reason given: Recruiting/enrolling participants halted prematurely but potentially will resume). Results are not available in the source record.

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?

Toxicity as assessed by Common Toxicity Criteria (CTC) toxicity criteria after the first course of treatment (time frame: within 21 days following administration of sirolimus). 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 12 Mar 2015.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in