NCT00981799

Brief Summary

Nelarabine has shown significant activity in patients with T-cell malignancies. This study will determine the safety and maximum tolerated dose of the combination of nelarabine, cyclophosphamide and etoposide in patients with first bone marrow relapse of T-ALL, or first relapse of T-LL.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
23

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jun 2010

Longer than P75 for phase_1

Geographic Reach
6 countries

36 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 9, 2009

Completed
13 days until next milestone

First Posted

Study publicly available on registry

September 22, 2009

Completed
8 months until next milestone

Study Start

First participant enrolled

June 1, 2010

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2015

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 18, 2016

Completed
4.2 years until next milestone

Results Posted

Study results publicly available

October 1, 2020

Completed
Last Updated

October 1, 2020

Status Verified

September 1, 2020

Enrollment Period

5.4 years

First QC Date

September 9, 2009

Results QC Date

September 8, 2020

Last Update Submit

September 8, 2020

Conditions

Keywords

RelapseT cellLymphoblasticLeukemiaLymphomaNelarabineTACLNECTAR

Outcome Measures

Primary Outcomes (1)

  • To Determine the Presence of Dose-limiting Toxicities (DLTs) of Nelarabine, Etoposide and Cyclophosphamide When Given in Combination to Children With T-ALL and Bone Marrow Relapse or T-LL.

    Patients will be evaluated based on Dose Level and total courses taken at each dose level and for presence of dose limiting toxicities. Not all patients enrolled at each dose level has been assessed to be evaluable for DLTs. Only those that have met criteria for being evaluable for DLT will be counted in the Overall Number of Participants Analyzed.

    6 months

Secondary Outcomes (1)

  • To Determine the Complete Remission Rate After 1 and 2 Courses of This Therapy in Children With T-ALL and Bone Marrow Relapse or T-LL.

    1-3 months

Study Arms (4)

Nelarabine Dose Level 1

EXPERIMENTAL

The study will begin at Dose Level 1 at 480 mg/m2 Nelarabine (75% of single agent maximum tolerated dose) and 330 mg/m2 Cyclophospamide and will escalate to the next Dose Level if the maximum tolerated dose (MTD) is not exceeded. The first 3 patients will be enrolled into Dose Level 1. If 0/3 experiences dose limiting toxicity (DLT) at a given dose level, then the dose is escalated to the next higher level and 3 more patients are enrolled. If 1/3 experiences DLT at current dose, the up to 3 more patients are accrued at the same dose level. If 2 or more DLTs are observed in a 3-patient or 6-patient cohort at a given dose level, then the MTD has been exceeded, dose escalation will be stopped, and up to 3 additional patients will be enrolled at the next lower dose level (unless 6 patients have already been treated at that prior dose). If the MTD is exceeded at Dose Level 0, the study will be closed.

Drug: NelarabineDrug: EtoposideDrug: CyclophosphamideDrug: MethotrexateDrug: Filgrastim

Nelarabine Dose Level 2

EXPERIMENTAL

Patients in this arm will be administered Nelarabine at 650 mg/m2 (100% of single agent MTD) and 330 mg/m2 Cyclophosphamide.

Drug: NelarabineDrug: EtoposideDrug: CyclophosphamideDrug: MethotrexateDrug: Filgrastim

Nelarabine Dose Level 3

EXPERIMENTAL

Patients in this arm will be administered Nelarabine at 650 mg/m2 (100% of single agent MTD) and 400 mg/m2 Cyclophosphamide

Drug: NelarabineDrug: EtoposideDrug: CyclophosphamideDrug: MethotrexateDrug: Filgrastim

Nelarabine Dose Level 0

EXPERIMENTAL

Patients in this arm will be administered Nelarabine 325 mg/m2 (50% of single agent MTD) and 330 mg/2 Cyclophosphamide. Patients will only enter this arm if the MTD at Dose Level 1 has been exceeded. If the MTD is exceeded at Dose Level 0, the study will be closed.

Drug: NelarabineDrug: EtoposideDrug: CyclophosphamideDrug: MethotrexateDrug: Filgrastim

Interventions

Dose will be assigned at study entry. Nelarabine will be given IV over 60 minutes (given at hours 0 to 1) on days 1 through 5.

Also known as: Arranon, Compound 506U78
Nelarabine Dose Level 0Nelarabine Dose Level 1Nelarabine Dose Level 2Nelarabine Dose Level 3

100 mg/m2/day IV over 2 hours (given at hours 1 to 3) on days 1 through 5

Also known as: VePesid, Etopophos, VP-16
Nelarabine Dose Level 0Nelarabine Dose Level 1Nelarabine Dose Level 2Nelarabine Dose Level 3

Dose will be assigned at study entry, IV as a 30-60 minute infusion (given at hours 3 to 4) on days 1 through 5.

Also known as: Cytoxan
Nelarabine Dose Level 0Nelarabine Dose Level 1Nelarabine Dose Level 2Nelarabine Dose Level 3

Give between day 29 and 36 or when ANC\>750 and PLTS\>75,000 - whichever comes first (but not prior to day 22) at the dose defined by age below, ideally in conjunction with BM evaluation. Given intrathecally at the dose defined by age below. 8 mg for patients age greater than or equal to 1, but \<2 years of age 10 mg for patients age greater than or equal to 2, but \<3 years of age 12 mg for patients greater than or equal to 3, but \< 9 years of age 15 mg for patients greater than or equal to \>9 years of age

Also known as: MTX, Amethopterin, Trexall
Nelarabine Dose Level 0Nelarabine Dose Level 1Nelarabine Dose Level 2Nelarabine Dose Level 3

5 micrograms/kg/day IV or SC will begin on Day 6 and end when the ANC is \> 1000/mm3 for two consecutive days.

Also known as: Neupogen, GCSF, granulocyte colony stimulating factor
Nelarabine Dose Level 0Nelarabine Dose Level 1Nelarabine Dose Level 2Nelarabine Dose Level 3

Eligibility Criteria

Age1 Year - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Patients to be enrolled in the dose-escalation portion of this study must have T-cell ALL or T-cell lymphoblastic lymphoma (LL) in first relapse or must have failed primary induction chemotherapy (ie, never attained a complete remission following an initial course of standard therapy for T-ALL or T-LL). Patients to be enrolled in the cohort expansion portion of this study (ie, those treated at the recommended phase 2 dose) must have T-cell ALL in first relapse or must have failed primary induction chemotherapy (ie, never attained a complete remission following an initial course of standard therapy for T-ALL). T-LL patients are not eligible for the cohort expansion phase.
  • Patients with T-cell ALL must have greater than 25% blasts in the bone marrow with or without extramedullary disease.
  • Patients with T-cell LL must have recurrent disease, documented by clinical or radiographic criteria, as well as histologic verification of the malignancy at original diagnosis. Patients with T-cell LL enrolled in the phase I dose-escalation study are not required to have measurable disease; however, patients enrolled in the phase II cohort expansion at the MTD must have measurable disease.
  • Patients may have CNS 1 or CNS 2 disease but not CNS 3.
  • ECOG 0-2 or Karnofsky ≥ 50% for patients \> 16 years of age; Lansky ≥ 50% for patients ≤16 years of age.
  • Patients may be enrolled on study regardless of the timing of prior Intrathecal therapy; however, they MAY NOT BEGIN TREATMENT ON THIS PROTOCOL UNTIL A MINIMUM OF 7 DAYS HAS ELAPSED SINCE PRIOR INTRATHECAL THERAPY.
  • At least 6 weeks must have elapsed since administration of nitrosureas.
  • At least 12 weeks must have elapsed since administration of craniospinal or hemipelvic radiation.
  • Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment.
  • Female patients with infants must agree not to breastfeed their infants while on this study.
  • Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for a minimum of 6 months after study treatment.
  • Adequate renal function defined as serum creatinine ≤ 1.5x upper limit of normal (ULN) for age. If the serum creatinine is above these values, the calculated creatinine clearance or radioisotope GFR must be ≥ 70 mL/min/1.73m2.
  • Total bilirubin ≤ 1.5x ULN for age. If the total bilirubin is elevated, patient will still be eligible if the conjugated (direct) serum bilirubin ≤ ULN for age.
  • ALT ≤ 5x ULN of normal for age.
  • Adequate cardiac function defined as shortening fraction of ≥ 27% by echocardiogram or ejection fraction ≥ 45% by gated radionuclide study.
  • +4 more criteria

You may not qualify if:

  • Patients with Down syndrome are excluded.
  • Patients with pre-existing Grade 2 (or greater) peripheral motor or sensory neurotoxicity per the CTCAE 3.0 as determined by the treating physician or a neurologist.
  • Patients with a history of prior veno-occlusive disease (VOD) or findings consistent with a diagnosis of VOD, defined as: conjugated serum bilirubin \>1.4 mg/dL AND unexplained weight gain greater than 10% of baseline weight or ascites AND hepatomegaly or right upper quadrant pain without another explanation, OR reversal of portal vein flow on ultrasound, OR pathological confirmation of VOD on liver biopsy.
  • Previous hematopoetic stem cell transplantation.
  • Patients with a prior seizure disorder requiring anti-convulsant therapy are not eligible to receive nelarabine. For the purposes of this study, this includes any patient that has received anticonvulsant therapy to prevent/treat seizures in the prior two years.
  • Positive blood culture within 48 hours of study enrollment.
  • Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection.
  • Plan to administer non-protocol chemotherapy, radiation therapy, or immunotherapy during the study period.
  • Any significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (37)

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital Orange County

Orange, California, United States

Location

UCSF School of Medicine

San Francisco, California, 94143-0106, United States

Location

The Children's Hospital, University of Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

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

Location

University of Miami Cancer Center

Miami, Florida, 33136, United States

Location

Children's Healthcare of Atlanta, Emory University

Atlanta, Georgia, United States

Location

Lurie Children's Hospital

Chicago, Illinois, United States

Location

Johns Hopkins University

Baltimore, Maryland, United States

Location

Dana Farber

Boston, Massachusetts, United States

Location

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109-0914, United States

Location

Childrens Hospital & Clinics of Minnesota

Minneapolis, Minnesota, 55404-4597, United States

Location

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

Location

New York University Medical Center

New York, New York, 10016, United States

Location

Children's Hospital New York-Presbyterian

New York, New York, 10032, United States

Location

Levine Children's Hospital at Carolinas Medical Center

Charlotte, North Carolina, 28203, United States

Location

Rainbow Babies

Cleveland, Ohio, United States

Location

Nationwide Childrens Hospital

Columbus, Ohio, United States

Location

Oregon Health and Science University

Portland, Oregon, United States

Location

St. Jude

Memphis, Tennessee, 38105-3678, United States

Location

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

Location

University of Texas at Southwestern

Dallas, Texas, United States

Location

Cook Children's Hospital

Fort Worth, Texas, United States

Location

Primary Children's

Salt Lake City, Utah, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Location

Children's Hospital at Westmead

Westmead, New South Wales, Australia

Location

Royal Children's Hospital

Brisbane, Queensland, Australia

Location

Royal Children's Hospital, Melbourne

Melbourne, Victoria, Australia

Location

Sydney Children's Hospital

Sydney, Australia

Location

St. Anna Children's Hospital

Vienna, Austria

Location

Hospital for Sick Kids

Toronto, Ontario, Canada

Location

Sainte Justine University Hospital

Montreal, Quebec, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

CHU Lille

Lille, France

Location

Bambino Gesù Hospital

Rome, Italy

Location

Erasmus MC - Sophia

Rotterdam, Netherlands

Location

Related Links

MeSH Terms

Conditions

Precursor T-Cell Lymphoblastic Leukemia-LymphomaRecurrenceLeukemiaLymphoma

Interventions

nelarabineEtoposideetoposide phosphateCyclophosphamideMethotrexateFilgrastimGranulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Precursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, LymphoidNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

PodophyllotoxinTetrahydronaphthalenesNaphthalenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic ChemicalsPolycyclic CompoundsGlucosidesGlycosidesCarbohydratesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus CompoundsAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsColony-Stimulating FactorsGlycoproteinsGlycoconjugatesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Results Point of Contact

Title
Clinical Research Coordinator, Consortia
Organization
Therapeutic Advancements of Childhood Leukemia and Lymphoma

Study Officials

  • Jim Whitlock, MD

    The Hospital for Sick Children

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
GT60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 9, 2009

First Posted

September 22, 2009

Study Start

June 1, 2010

Primary Completion

November 1, 2015

Study Completion

July 18, 2016

Last Updated

October 1, 2020

Results First Posted

October 1, 2020

Record last verified: 2020-09

Locations