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NeuroblastomaPhase 1NCT01916187

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

A Phase 1 study testing Imetelstat in people with Neuroblastoma, run by NCIC Clinical Trials Group at 1 site in Canada.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 months to 18 years
Conditions the study is for
Neuroblastoma
What is being tested?
Imetelstat
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteCanada
Study dates
30 Jul 2013to 16 Jan 2014
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 18 months to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.
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  • 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)
  • Hemoglobin ≥80 g/L (may receive RBC transfusions) Patients with known bone marrow metastatic disease will be eligible for the study and may receive transfusions, provided ≤25% of bone marrow is involved, and provided they are not known to be refractory to red cell or platelet transfusions.
  • Patients will be eligible as long as blood count criteria are met. If patients then experience prolonged myelosuppression, bone marrow examination can be requested to determine if the low blood counts are due to malignant infiltration of the marrow or to therapy induced hypoplasia/aplasia.
  • Adequate Renal And Cardiac Function, defined as:
  • Serum creatinine ≤1.5 x upper limit of normal for age or
  • Measured GFR ≥70 mL/min/1.73 m2
  • Adequate Liver Function, defined as:
  • AST and ALT ≤2.5 x upper limit normal for age
  • Total bilirubin ≤1.5 x upper limit normal for age
  • Serum albumin ≥ 20
  • Adequate Coagulation Function, defined as:
  • PTT <1.2 x upper limit normal
  • Patient or guardian consent must be obtained on all patients according to local Institutional and/or University Human Experimentation Committee requirements. Children > 8 years old whose parent or guardian has signed consent on their behalf may also sign assent if desired. It will be the responsibility of the local participating investigators to obtain the necessary local clearance, and to indicate in writing to the NCIC CTG Study Coordinator that such clearance has been obtained, before the trial can commence in that centre.
  • Patients must be accessible for treatment and follow-up. Patients registered on this trial must be treated and followed at the participating centre. This implies there must be reasonable geographical limits (for example: two hour's driving distance) placed on patients being considered for this trial.
  • Protocol treatment is to begin within 5 working days of patient registration.

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • CHU Sainte-Justine

    Montreal, Quebec, H3T 1C5

Other trials in Canada

About this study

Official title: A Pilot Study of Imetelstat Given Intravenously on Day 1 and 8 of a 21 Day Schedule Alone and With Standard 13-Cis-Retinoic Acid in Children With Recurrent and/or Refractory Neuroblastoma

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.

Read the detailed description

This research is being done because imetelstat has been shown to slow the growth of tumours in animals and may also be doing so in adults, but we are not sure if it can also slow tumour growth in children and offer better results than standard treatment. Laboratory studies suggest imetelstat may increase the activity of 13-cis-retinoic acid, which is used to treat neuroblastoma, although this is not yet proven in patients.

Conditions
Neuroblastoma
Being tested
Imetelstat
Medical subject headings
Neuroblastoma

What participants receive

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

  • ImetelstatDrug

Study groups in NCT01916187
Study groupRoleWhat they receive
ImetelstatExperimental285 mg/m2/dose, IV, for 2 hours. Days 1 and 8 every three weeks.
Receives: Drug: Imetelstat

What is being measured

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

  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.

    Measured: 24 month

Secondary outcomes (2)

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

    Bone marrow aspirates, biopsies and blood samples for CC assay: at baseline, end of cycles 1 and 2 then end of every other cycle will be assessed in patients with relapsed and/or refractory neuroblastoma to assess the impact of imetelstat on hematopoietic stem cells and neuroblastoma tumour initiating cells.

    Measured: 24 months

  2. Changes in plasma C-circles

    In patients with relapsed and/or refractory neuroblastoma to evaluate:

    • The correlation of tumour and plasma C-circles.
    • The role of plasma C-circles as a tumour biomarker for alternative lengthening of telomeres (ALT).
    • Changes in plasma C-circles induced by treatment with imetelstat.

    Measured: 24 months

Timeline

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

  1. First posted5 Aug 2013 (estimated)
  2. Study start30 Jul 2013
  3. Primary completion22 Nov 2013
  4. Study completion16 Jan 2014

Why it stopped: drug company withdrew support following re-examination of benefit-risk assessment for the investigational use of imetelstat in this population

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
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01916187
Sponsor's protocol ID
I212
Status last verified
Apr 2020
First submitted
31 Jul 2013
Last updated
21 Sep 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Imetelstat in people with Neuroblastoma, run by NCIC Clinical Trials Group at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 8 exclusion criteria. 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: withdrawn, as of the sponsor's last update on 21 Sep 2020. 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 Canada. 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 withdrawn before enrolling participants, so there are no results.

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?

Effectiveness of Imetelstat in pediatric dose (time frame: 24 month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01916187.

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 21 Sep 2020.

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