Bortezomib With Chemotherapy for Relapsed Pediatric Acute Lymphoblastic Leukemia (ALL)

NCT ID: NCT00440726

Last Updated: 2020-02-19

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

31 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-08-04

Study Completion Date

2011-02-26

Brief Summary

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This is a Phase I/II study of a drug called bortezomib given in combination with chemotherapy drugs used to treat acute lymphoblastic leukemia (ALL) that has come back (recurred). Bortezomib is a drug that has been approved by the Food and Drug Administration (FDA) for treating adults with multiple myeloma which is a type of blood cancer. Bortezomib has been shown to cause cancer cells to die in studies done on animals (mice). Studies have been done that have shown that some adults and children with cancer have shown a response to bortezomib when it is used alone. Studies have also been done in adults to evaluate the dose of bortezomib that can be safely given in combination with other chemotherapy drugs.

Detailed Description

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All patients will receive 1 course of chemotherapy unless medical complications prevent the administration of some of the drugs. Treatment will last about 1 month.

Treatment on this study will consist of a combination of 7 anti-cancer medications. The 7 anti-cancer medicines are bortezomib, vincristine, dexamethasone, PEG-asparaginase, doxorubicin, cytarabine (Ara-C), and methotrexate (MTX).

If you are in the Phase I portion of this study, you will be given an assigned dose of bortezomib. The dose of bortezomib will be based on doses given in previous studies done with adults and children. At each dose level of bortezomib, between 3 and 6 children will receive bortezomib in combination with chemotherapy. If the side effects are not too severe, the next group of children will receive a higher dose. The dose will continue to be increased until we find the dose that causes serious side effects. Your dose of bortezomib will not be increased. If you have bad side effects, your dose may be decreased.

The dose used during the Phase 2 part of this study will be determined by the outcome of the Phase I study. The highest dose used in Phase I that was tolerated without serious side effects will be the one used in Phase 2.

Conditions

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Acute Lymphoblastic Leukemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Ph 1 Dose Escalation

Intervention: Bortezomib with chemotherapy (dexamethasone, PEG-asparaginase, doxorubicin, cytarabine, methotrexate, and vincristine) and Triple IT therapy for patients who are CNS 2 or 3 at study entry. 3+3 escalation design.

Group Type EXPERIMENTAL

Bortezomib

Intervention Type DRUG

Intravenous on days 1, 4, 8 and 11. Dose assigned at study entry.

Dexamethasone

Intervention Type DRUG

10 mg/m2/day divided BID, oral administration for 14 days.

PEG-asparaginase

Intervention Type DRUG

2500 IU/m2/day, intramuscular injection on Days 2, 8, 15 and 22

Doxorubicin

Intervention Type DRUG

60 mg/m2/day IV over 15 minutes on Day 1

Cytarabine

Intervention Type DRUG

Given intrathecally on Day 1 of course 1 at the dose defined by age below.

* 30 mg for patients age 1-1.99
* 50 mg for patients age 2-2.99
* 70 mg for patients \>3 years of age

Methotrexate

Intervention Type DRUG

Given intrathecally to all patients who are CNS 1 at study entry on Day 15 at the dose defined by age below.

* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients \>9 years of age

Vincristine

Intervention Type DRUG

1.5 mg/m2/dose IV push (maximum single dose 2 mg) on Days 1, 8, 15 and 22.

Triple IT Therapy

Intervention Type DRUG

Triple IT therapy will be given intrathecally on Day 8, 15, and 22 for patients who are CNS 2 and CNS 3 at study entry. Regimen/dosing as follows:

Methotrexate-

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Cytarabine:

* \<2 years: 16 mg
* 2 - \<3 y: 20 mg
* 3 - \<9 y: 24 mg
* \>=9 y: 30 mg

Hydrocortisone:

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Ph 2 Efficacy and Safety

Intervention: Bortezomib with chemotherapy (dexamethasone, PEG-asparaginase, doxorubicin, cytarabine, methotrexate, and vincristine) and Triple IT therapy for patients who are CNS 2 or 3 at study entry. Patients receive bortezomib at maximum tolerated dose (as established in the Phase 1 portion of the study) and are assessed for response and toxicity.

Group Type EXPERIMENTAL

Bortezomib

Intervention Type DRUG

Intravenous on days 1, 4, 8 and 11. Dose assigned at study entry.

Dexamethasone

Intervention Type DRUG

10 mg/m2/day divided BID, oral administration for 14 days.

PEG-asparaginase

Intervention Type DRUG

2500 IU/m2/day, intramuscular injection on Days 2, 8, 15 and 22

Doxorubicin

Intervention Type DRUG

60 mg/m2/day IV over 15 minutes on Day 1

Cytarabine

Intervention Type DRUG

Given intrathecally on Day 1 of course 1 at the dose defined by age below.

* 30 mg for patients age 1-1.99
* 50 mg for patients age 2-2.99
* 70 mg for patients \>3 years of age

Methotrexate

Intervention Type DRUG

Given intrathecally to all patients who are CNS 1 at study entry on Day 15 at the dose defined by age below.

* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients \>9 years of age

Vincristine

Intervention Type DRUG

1.5 mg/m2/dose IV push (maximum single dose 2 mg) on Days 1, 8, 15 and 22.

Triple IT Therapy

Intervention Type DRUG

Triple IT therapy will be given intrathecally on Day 8, 15, and 22 for patients who are CNS 2 and CNS 3 at study entry. Regimen/dosing as follows:

Methotrexate-

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Cytarabine:

* \<2 years: 16 mg
* 2 - \<3 y: 20 mg
* 3 - \<9 y: 24 mg
* \>=9 y: 30 mg

Hydrocortisone:

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Interventions

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Bortezomib

Intravenous on days 1, 4, 8 and 11. Dose assigned at study entry.

Intervention Type DRUG

Dexamethasone

10 mg/m2/day divided BID, oral administration for 14 days.

Intervention Type DRUG

PEG-asparaginase

2500 IU/m2/day, intramuscular injection on Days 2, 8, 15 and 22

Intervention Type DRUG

Doxorubicin

60 mg/m2/day IV over 15 minutes on Day 1

Intervention Type DRUG

Cytarabine

Given intrathecally on Day 1 of course 1 at the dose defined by age below.

* 30 mg for patients age 1-1.99
* 50 mg for patients age 2-2.99
* 70 mg for patients \>3 years of age

Intervention Type DRUG

Methotrexate

Given intrathecally to all patients who are CNS 1 at study entry on Day 15 at the dose defined by age below.

* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients \>9 years of age

Intervention Type DRUG

Vincristine

1.5 mg/m2/dose IV push (maximum single dose 2 mg) on Days 1, 8, 15 and 22.

Intervention Type DRUG

Triple IT Therapy

Triple IT therapy will be given intrathecally on Day 8, 15, and 22 for patients who are CNS 2 and CNS 3 at study entry. Regimen/dosing as follows:

Methotrexate-

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Cytarabine:

* \<2 years: 16 mg
* 2 - \<3 y: 20 mg
* 3 - \<9 y: 24 mg
* \>=9 y: 30 mg

Hydrocortisone:

* \<2 years: 8 mg
* 2 - \<3 y: 10 mg
* 3 - \<9 y: 12 mg
* \>=9 y: 15 mg

Intervention Type DRUG

Other Intervention Names

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Velcade Decadron Oncaspar Adriamycin Rubex Cytosar-U Ara-C Arabinosylcytosine Otrexup Rasuvo Rheumatrex Trexall Amethopterin Oncovin Leurocristine

Eligibility Criteria

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Inclusion Criteria

The eligibility criteria listed below are interpreted literally and cannot be waived.

1. Age Patients must be \< 21 years of age when originally diagnosed with ALL. Patient must be \> 1 year of age at study entry.
2. Diagnosis Patients must have relapsed or refractory ALL with a M3 marrow (marrow blasts \>25%). Patients with CNS I, II or III or testicular disease are eligible.
3. Performance Level Karnofsky \> 50% for patients \> 10 years of age and Lansky \> 50% for patients \< 10 years of age.
4. Prior Therapy Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.

1. Prior anthracycline exposure: Patients must have less than 400mg/m2 lifetime exposure of anthracycline chemotherapy.
2. Stem Cell Transplant (SCT): Patients are eligible after allogeneic stem cell transplant as long as patients are not actively being treated for graft-versus-host-disease (GvHD).
3. Patients should not have received previous therapy using bortezomib (Velcdade® or PS-341).
4. During the phase I portion of the trial, there is no limit on the number of prior treatment regimens. Patients with persistent disease after an induction attempt are eligible.
5. During the phase II portion of the trial, patients must have had two or more prior therapeutic attempts defined as:

* Persistent initial disease after two induction attempts, or
* Relapse after one-reinduction attempt (2nd relapse), or
* Persistent disease after first relapse and initial re-induction attempt

(Patients in first relapse are not eligible for the phase II portion of the study)
6. During the phase II portion of the trial, patients must have no more than 3 prior therapeutic attempts and it must be at least 3 months since the last treatment with a "VPLD" induction/re-induction regimen.
5. Reproductive Function

1. Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed prior to enrollment.
2. Female patients with infants must agree not to breastfeed their infants while on this study.
3. Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study.

Exclusion Criteria

1. Drug Allergies

Patients will be excluded if they have allergies to the following:
* Asparaginase products
* Boron
* Mannitol
2. Renal Function Patients will be excluded if their serum creatinine is \> 2 x the upper limit of normal for age at the institution's laboratory.
3. Liver/Pancreatic Function

1. Direct bilirubin \> 1.5x the institutional ULN for age. A total bilirubin result that is less than 1.5 times the institutional ULN for age may be used for eligibility if a direct bilirubin result is not available.
2. SGPT (ALT) \> 4 x institutional ULN
3. Grade 3 or greater pancreatitis as defined by the CTCAE v3.0
4. History of any L-asparaginase induced pancreatitis
5. Amylase or Lipase \> 2 x institutional ULN
4. Cardiac Function Patients will be excluded if their shortening fraction by echocardiogram is less than 30%.
5. Patients with Down Syndrome are excluded.
6. Infection

* Patients will be excluded if they have an active uncontrolled infection.
* Patients will be excluded if they have had a positive culture within 2 weeks of study entry.
7. Patients with grade 2 or greater motor or sensory neuropathy per CTC 3.0 criteria.
8. Patients planning on receiving other investigational agents while on this study. (An investigational agent is defined as any drug not currently approved for use in humans.)
9. Patients planning on receiving other anti-cancer therapies while on this study. Hydroxyurea for cyto-reduction is allowed prior to the start of therapy.
10. Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
11. Patients who have started protocol therapy prior to enrollment. Patient may still enroll if IT therapy was given within 72 hours of study enrollment as part of the diagnostic lumbar procedure.
Minimum Eligible Age

1 Year

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Therapeutic Advances in Childhood Leukemia Consortium

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Yoav Messinger, MD

Role: STUDY_CHAIR

Children's Hospital and Clinics of Minnesota

Locations

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City of Hope

Duarte, California, United States

Site Status

Miller Children's Hospital

Long Beach, California, United States

Site Status

Childrens Hospital Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital & Research Center Oakland

Oakland, California, United States

Site Status

Stanford University Medical Center

Palo Alto, California, United States

Site Status

UCSF School of Medicine

San Francisco, California, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

University of Miami Cancer Center

Miami, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Johns Hopkins / Sydney Kimmel Cancer Center

Baltimore, Maryland, United States

Site Status

Dana Farber Cancer Center

Boston, Massachusetts, United States

Site Status

C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

Site Status

Childrens Hospital & Clinics of Minnesota

Minneapolis, Minnesota, United States

Site Status

New York University Medical Center

New York, New York, United States

Site Status

Children's Hospital New York-Presbyterian

New York, New York, United States

Site Status

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Sydney Children's Hospital

Randwick, New South Wales, Australia

Site Status

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Site Status

Universidade Federale de Sao Paulo/Hospital Sao Paulo

São Paulo, , Brazil

Site Status

Sick Kids

Toronto, Ontario, Canada

Site Status

Countries

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United States Australia Brazil Canada

References

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Messinger Y, Gaynon P, Raetz E, Hutchinson R, Dubois S, Glade-Bender J, Sposto R, van der Giessen J, Eckroth E, Bostrom BC. Phase I study of bortezomib combined with chemotherapy in children with relapsed childhood acute lymphoblastic leukemia (ALL): a report from the therapeutic advances in childhood leukemia (TACL) consortium. Pediatr Blood Cancer. 2010 Aug;55(2):254-9. doi: 10.1002/pbc.22456.

Reference Type RESULT
PMID: 20582937 (View on PubMed)

Messinger YH, Gaynon PS, Sposto R, van der Giessen J, Eckroth E, Malvar J, Bostrom BC; Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) Consortium. Bortezomib with chemotherapy is highly active in advanced B-precursor acute lymphoblastic leukemia: Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) Study. Blood. 2012 Jul 12;120(2):285-90. doi: 10.1182/blood-2012-04-418640. Epub 2012 May 31.

Reference Type RESULT
PMID: 22653976 (View on PubMed)

Related Links

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http://www.tacl.us

Click here for more information about this protocol and the Therapeutic Advances in Childhood Leukemia Consortium

Other Identifiers

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T2005-003

Identifier Type: -

Identifier Source: org_study_id

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