NCT01592045

Brief Summary

The purpose of this study is to compare the pharmacokinetics (blood levels) and safety of chimeric (ch) 14.18 manufactured by two independent drug makers (United Therapeutics \[UTC\] or the National Cancer Institute \[NCI\]).

Trial Health

87
On Track

Trial Health Score

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

Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Aug 2012

Geographic Reach
1 country

13 active sites

Status
completed

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

April 30, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 4, 2012

Completed
3 months until next milestone

Study Start

First participant enrolled

August 1, 2012

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2014

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

September 23, 2015

Completed
Last Updated

September 23, 2015

Status Verified

August 1, 2015

Enrollment Period

1.8 years

First QC Date

April 30, 2012

Results QC Date

June 3, 2015

Last Update Submit

August 20, 2015

Conditions

Outcome Measures

Primary Outcomes (2)

  • Area Under the Plasma Concentration Curve (AUC)

    Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin

    PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment

  • Peak Plasma Concentration (Cmax)

    Twenty-two PK samples will be obtained at the following timepoints: Courses 1 and 3: Day: 0 Days: 3, 4, 5 and 6: post ch14.18 Days 7:10 to 14 hours post ch14.18 Days 9 to 11: Single sample Days 14 to 17: Single sample Courses 2 and 4: Day 0: Pre-IL-2 Day 7: Pre-ch14.18 Day 10 (Course 4 only): Post ch14.18 End of Treatment: Within 2 weeks post isotretinoin

    PK samples obtained during Courses 1 and 3: Days 0, 3, 4, 5, 6, 7, 9, 10, 11, 14, 15, 16, 17; PK samples obtained during Courses 2 and 4: Days 0, 7, 10; End of Treatment

Study Arms (2)

Sequence 1

EXPERIMENTAL

UTC ch14.18 for two courses and NCI ch14.18 for three courses

Biological: ch14.18 -NCIBiological: ch14.18-UTCBiological: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)Biological: Aldesleukin (IL-2)Drug: Isotretinoin

Sequence 2

EXPERIMENTAL

NCI ch14.18 for two courses and UTC ch14.18 for three courses

Biological: ch14.18 -NCIBiological: ch14.18-UTCBiological: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)Biological: Aldesleukin (IL-2)Drug: Isotretinoin

Interventions

ch14.18 -NCIBIOLOGICAL

25 mg/m\^2/day IV for four consecutive days

Sequence 1Sequence 2
ch14.18-UTCBIOLOGICAL

17.5 mg/m\^2/day IV for four consecutive days

Sequence 1Sequence 2

GM-CSF will be administered SC at a dose of 250 mcg/m\^2/day for 14 days during Courses 1, 3, and 5.

Sequence 1Sequence 2

Aldesleukin (IL-2) will be administered IV at a dose of 3 MIU/m\^2/day for the first week and at a dose of 4.5 MIU/m\^2/day for the second week during Courses 2 and 4.

Sequence 1Sequence 2

Isotretinoin (13-cis-retinoic acid; ISOT) will be administered by mouth over six courses as follows: If weight \> 12 kg: 80 mg/m\^2/dose twice daily (total daily dose is 160 mg/m\^2/day, divided twice daily). If weight ≤ 12 kg: 2.67 mg/kg/dose twice daily (total daily dose is 5.33 mg/kg/day, divided twice daily).

Also known as: 13-cis-retinoic acid
Sequence 1Sequence 2

Eligibility Criteria

AgeUp to 8 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Diagnosis of high-risk neuroblastoma
  • years of age or younger at diagnosis of high-risk neuroblastoma
  • Patients must have completed therapy including intensive induction followed by autologous stem cell transplantation (ASCT) and radiotherapy
  • \* Radiotherapy may be waived for patients who either have small adrenal masses which are completely resected up front, or who never have an identifiable primary tumor
  • Must meet the International Neuroblastoma Response Criteria (INRC) for CR, VGPR, or PR for primary site, soft tissue metastases, and bone metastases AND must also meet the protocol specified criteria for bone marrow response as follows:
  • \* No more than 10% tumor (of total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy
  • Patient who have no tumor seen on the prior bone marrow, and then have ≤ 10% tumor on any of the bilateral marrow aspirate/biopsy specimens done at pre-ASCT and/or pre-enrollment evaluation will also be eligible
  • No more than 12 months from starting the first induction chemotherapy after diagnosis to the date of ASCT
  • \* For patients who became high-risk neuroblastoma after initial non-high risk disease, the 12 months period should start from the date of induction therapy for high-risk neuroblastoma to the date of ASCT
  • No progressive disease at time of registration except for protocol-specified bone marrow response
  • Adequate hematological, renal, hepatic, pulmonary and cardiac function
  • CNS toxicity \< Grade 2

You may not qualify if:

  • Prior anti-GD2 antibody therapy
  • Prior vaccine therapy for neuroblastoma
  • Concurrent anti-cancer or immunosuppressive therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Healthcare of Atlanta - Egleston

Atlanta, Georgia, 30322, United States

Location

The University of Chicago

Chicago, Illinois, 60637, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States

Location

Children's Mercy Hospital (Kansas)

Kansas City, Missouri, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63310, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

dinutuximabGranulocyte-Macrophage Colony-Stimulating FactoraldesleukinInterleukin-2Isotretinoin

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, PeripheralNeuroectodermal Tumors, PrimitiveNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological FactorsInterleukinsLymphokinesRetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesPigments, Biological

Results Point of Contact

Title
Allison Lim
Organization
United Therapeutics Corporation

Study Officials

  • Araz Marachelian, MD

    Children's Hospital Los Angeles

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 30, 2012

First Posted

May 4, 2012

Study Start

August 1, 2012

Primary Completion

June 1, 2014

Study Completion

June 1, 2014

Last Updated

September 23, 2015

Results First Posted

September 23, 2015

Record last verified: 2015-08

Locations