Imetelstat Given Intravenously Alone and With Standard 13-Cis-Retinoic Acid in Children With Neuroblastoma

NCT01916187 | Withdrawn Phase 1 DMC

• 1 other identifier: I212
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine the best dose of imetelstat when given alone for patients with neuroblastoma and also when given in combination with 13-cis-retinoic acid.

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• Effectiveness of Imetelstat in pediatric dose

  Response and progression will be evaluated in this study using the revised international criteria (1.1) proposed by the RECIST (Response Evaluation Criteria in Solid Tumours) committee in patients with relapsed and/or refractory neuroblastoma, to confirm the feasibility of administering imetelstat given at the recommended pediatric dose as determined in the Children's Oncology Group Study ADVL1112 (a phase I study of imetelstat, a telomerase inhibitor, in children with recurrent or refractory solid tumours and lymphoma), alone and in combination with 13-cis-retinoic acid.

  24 month

Secondary Outcomes (2)

• Impact of imetelstat on hematopoietic stem cells and neuroblastoma tumour cells.

  24 months

• Changes in plasma C-circles

  24 months

Study Arms (1)

Imetelstat

EXPERIMENTAL

285 mg/m2/dose, IV, for 2 hours. Days 1 and 8 every three weeks.

Drug: Imetelstat

Interventions

Imetelstat DRUG

Imetelstat

Eligibility Criteria

• Age: 18 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Histological verification of neuroblastoma at either original diagnosis or relapse.
• Patients must have recurrent or refractory neuroblastoma with either measurable or evaluable disease (defined by a positive nuclear scan such as bone scan or metaiodobenzylguanidine (MIBG) scan). If a lesion is isolated and /or previously irradiated and stable, a proven positive biopsy will be required to be eligible.
• Current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life.
• Must have recovered from the acute effects of prior chemotherapy, immunotherapy or radiotherapy prior to study entry as follows:
• At least 3 weeks from completion of last active cancer therapy and recovered from toxic effects of that therapy to ≤ grade 1.
• At least 6 weeks post hematopoietic stem cell rescue following myeloablative therapy.
• At least 3 months must have elapsed if prior total body irradiation, craniospinal XRT or if ≥ 50% radiation of pelvis.
• At least 6 weeks must have elapsed if other substantial bone marrow irradiation.
• At least 6 weeks from prior MIBG therapy.
• Age \>18 months and ≤18 years at the time of study entry.
• Performance Status:
• Patients ≤10 years: Lansky ≥50 Patients \>10 years: Karnofsky ≥60% - Laboratory Requirements: (must be done within 7 days prior to registration)
• Adequate Bone Marrow Function, defined as:
• Absolute neutrophil count (ANC) ≥1.0 x 10\^9/L
• Platelets ≥100 x 10\^9/L (transfusion independent defined as not receiving platelet transfusions within 7 days prior to registration)

You may not qualify if:

• Patients with \>25% bone marrow involvement will not be enrolled.
• Patients must be able to take oral medication and have no gastrointestinal abnormalities (e.g. bowel obstruction or previous gastric resection) which would lead to inadequate absorption of 13-cisretinoic acid.
• Known HIV, hepatitis B or hepatitis C infections.
• Imetelstat animal and in vitro studies suggest it is not genotoxic or teratogenic. However, 13-cis-retinoic acid is known to be teratogenic. Pregnancy tests must be obtained in girls who are post menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two reliable contraceptive methods. Pregnant or breast-feeding females will not be entered on this study due to the potential fetal and teratogenic adverse effects.
• Concurrent Medications:
• Patients receiving other investigational agents will not be enrolled.
• Patient receiving other anti-cancer agents will not be enrolled.
• Patients with CNS metastasis will need to submit to a baseline MRI obtained within 21 days prior to registration. Patients with evidence of current or prior CNS hemorrhage will be excluded. Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study will not be enrolled.

Sponsors & Collaborators

• NCIC Clinical Trials Group: lead
• Geron Corporation: collaborator

Study Sites (1)

CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

imetelstat

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Victor Lewis

  Alberta Children's Hospital, Southern Alberta Children's Cancer Program, AB Canada

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2013
• First Posted: August 5, 2013
• Study Start: July 30, 2013
• Primary Completion: November 22, 2013
• Study Completion: January 16, 2014
• Last Updated: September 21, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)