HepatoblastomaPhase 2NCT02011126
Imetelstat Sodium in Treating Younger Patients With Relapsed or Refractory Solid Tumors
A Phase 2 study testing Imetelstat Sodium and Laboratory Biomarker Analysis in people with Hepatoblastoma and Previously Treated Childhood Rhabdomyosarcoma, run by Children's Oncology Group.
Trial summary
This phase II trial studies the side effects and how well imetelstat sodium works in treating younger patients with relapsed or refractory solid tumors. Imetelstat sodium may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- Hepatoblastoma, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer
- What is being tested?
- Imetelstat Sodium and Laboratory Biomarker Analysis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 30 Jun 2014to 25 Mar 2016
- Phase
- Phase 2
- 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 1 year to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with any of the following tumors who have relapsed or refractory disease are eligible:
- Osteosarcoma
- Ewing?s sarcoma / peripheral primitive neuroectodermal tumor (PNET)
- Rhabdomyosarcoma
- Neuroblastoma (measurable or evaluable disease)
- Hepatoblastoma
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- Patients must have had histologic verification of malignancy at original diagnosis or relapse
- Patients must have radiographically measurable disease (with the exception of neuroblastoma)
- Measurable disease is defined as the presence of at least one lesion on magnetic resonance imaging (MRI) or computed tomography (CT) scan that can be accurately measured with the longest diameter a minimum of 10 mm in at least one dimension (CT scan slice thickness no greater than 5 mm)
- Note: the following do not qualify as measurable disease:
- Malignant fluid collections (e.g., ascites, pleural effusions)
- Bone marrow infiltration
- Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as defined below for neuroblastoma
- Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
- Previously radiated lesions that have not demonstrated clear progression post radiation
- Leptomeningeal lesions that do not meet the measurements noted above
- Patients with neuroblastoma who do not have measurable disease but have evaluable disease on 131I-metaiodobenzylguanidine (MIBG) scans are eligible
- Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
- Myelosuppressive chemotherapy: patients with solid tumors must not have received myelosuppressive chemotherapy within 3 weeks of enrollment onto this study (6 weeks if prior nitrosourea)
- Hematopoietic growth factors: at least 7 days must have elapsed since the completion of therapy with a growth factor; at least 14 days must have elapsed after receiving pegfilgrastim
- Biologic (anti-neoplastic agent): at least 7 days must have elapsed since completion of therapy with a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur
- Monoclonal antibodies: at least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody
- Radiotherapy: >= 2 weeks must have elapsed since local palliative radiation therapy (XRT) (small port); >= 6 weeks must have elapsed since treatment with therapeutic doses of MIBG; >= 3 months must have elapsed if prior craniospinal XRT was received, if >= 50% of the pelvis was irradiated, or if total body irradiation (TBI) was received; >= 6 weeks must have elapsed if other substantial bone marrow irradiation was given
- Stem cell transplant or rescue without TBI: no evidence of active graft versus (vs.) host disease and >= 2 months must have elapsed since transplant
- For patients with solid tumors without bone marrow involvement:
- Peripheral absolute neutrophil count (ANC) >= 1000/uL
- For patients with solid tumors without bone marrow involvement:
- Platelet count >= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions within a 7 day period prior to enrollment)
- For patients with solid tumors without bone marrow involvement:
- Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions).
- For patients with solid tumors and known bone marrow metastatic disease:
- Peripheral absolute neutrophil count (ANC) >= 750/uL
- For patients with solid tumors and known bone marrow metastatic disease:
- Platelet count >= 50,000/uL
- For patients with solid tumors and known bone marrow metastatic disease:
- Hemoglobin >= 8.0 g/dL
- Transfusions are permitted to meet both the platelet and hemoglobin criteria; patients must not be known to be refractory to red blood cell or platelet transfusions
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
- 0.6 mg/dL (1 to < 2 years of age)
- 0.8 mg/dL (2 to < 6 years of age)
- 1.0 mg/dL (6 to < 10 years of age)
- 1.2 mg/dL (10 to < 13 years of age)
- 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)
- 1.7 mg/dL (male) or 1.4 mg/dL (female) (>= 16 years of age)
- Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 110 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)
- Serum albumin >= 2 g/dL
- Activated partial thromboplastin time (aPTT) =< 1.2 x upper limit of normal
Who cannot join
- Patients who are pregnant or breast-feeding are not eligible for this study; negative 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 an effective contraceptive method for the duration of study therapy; study drug may also potentially be secreted in milk and therefore breastfeeding women are excluded
- Growth factors that support platelet or white cell number or function must not have been administered within the 7 days prior to enrollment (14 days if pegfilgrastim)
- Patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible
- Patients who are currently receiving another investigational drug are not eligible
- Patients who are currently receiving other anti-cancer agents are not eligible
- Anti-graft-versus-host disease (GVHD) or agents to prevent organ rejection post-transplant:
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- patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial
- Patients who have an uncontrolled infection are not eligible
- Patients who have received prior treatment with imetelstat are not eligible
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
- Patients with prior allogeneic transplants are not eligible
About this study
Official title: A Phase II Study of Imetelstat (GRN163L, NSC# 754228) in Children With Relapsed or Refractory Solid Tumors
This phase II trial studies the side effects and how well imetelstat sodium works in treating younger patients with relapsed or refractory solid tumors. Imetelstat sodium may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the objective response rate, defined as partial response or better, of imetelstat (imetelstat sodium) in children with relapsed or refractory solid tumors.
II. To further define and describe the toxicities associated with imetelstat in children with recurrent/refractory solid tumors.
SECONDARY OBJECTIVES:
- To determine the time to progression following treatment with imetelstat in children with relapsed or refractory solid tumors.
TERTIARY OBJECTIVES:
- To measure tumor telomere length in archival samples, and to correlate telomere length to the clinical outcome of the study.
OUTLINE:
Patients receive imetelstat sodium intravenously (IV) over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity.
- Conditions
- Hepatoblastoma, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Neuroblastoma
- Being tested
- Imetelstat Sodium, Laboratory Biomarker Analysis
- Medical subject headings
- Hepatoblastoma, Neuroectodermal Tumors, Primitive, Peripheral, Neuroblastoma, Osteosarcoma, Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imetelstat SodiumDrug
Also called GRN163L, GRN163L, Sodium Salt
Given IV
Laboratory Biomarker AnalysisOther
Optional correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (imetelstat sodium) | Experimental | Patients receive imetelstat sodium IV over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity. Receives: Drug: Imetelstat Sodium; Other: Laboratory Biomarker Analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response (complete response [CR] or partial response [PR]) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria or MIBG scoring criteria
Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed accounting for the two-stage design. Each disease stratum will be reported separately.
Measured: Up to 126 days (6 courses)
Incidence of grade 3 or higher adverse events using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Toxicity tables will be constructed to summarize the observed incidence in each reporting period by type of toxicity and grade. The relative frequency of each type of toxicity will be quantified as the number of toxicity-evaluable cycles in which the adverse event (AE) was noted at grade 3 or higher considered by the treating physician to be possibly, probably or definitely related to one of the agents in the regimen divided by the number of toxicity-evaluable courses administered to patients enrolled on the trial.
Measured: Up to 126 days (6 courses)
Secondary outcomes (1)
Progression-free survival
The probability of remaining progression-free as a function of days since enrollment will be calculated according to the method of Gray accounting for censoring and the competing events.
Measured: Date of enrollment until the end progression-free interval (PFI) date, calculated as the date of disease progression, date of death, date of removal of all tumor by surgery or last patient contact, whichever occurs first, assessed up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2013 (estimated)
- Study start30 Jun 2014 (estimated)
- Primary completion25 Mar 2016 (estimated)
Why it stopped: IND no longer available.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HepatoblastomaStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Patrick Thompson, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02011126
- Sponsor's protocol ID
- ADVL1321
- Other IDs
- NCI-2013-01640, ADVL1321, ADVL1321, U10CA098543
- Status last verified
- May 2014
- First submitted
- 10 Dec 2013
- Last updated
- 7 Aug 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Imetelstat (GRN163L, NSC# 754228) in Children With Relapsed or Refractory Solid Tumors
- ClinicalTrials.gov ID
- NCT02011126
- Acronym / sponsor ID
- ADVL1321
- Conditions
- Hepatoblastoma, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: IND no longer available.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 30 Jun 2014 (estimated)
- Primary completion
- 25 Mar 2016 (estimated)
- First posted
- 13 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies the side effects and how well imetelstat sodium works in treating younger patients with relapsed or refractory solid tumors. Imetelstat sodium may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Detailed description
PRIMARY OBJECTIVES:
- To determine the objective response rate, defined as partial response or better, of imetelstat (imetelstat sodium) in children with relapsed or refractory solid tumors.
II. To further define and describe the toxicities associated with imetelstat in children with recurrent/refractory solid tumors.
SECONDARY OBJECTIVES:
- To determine the time to progression following treatment with imetelstat in children with relapsed or refractory solid tumors.
TERTIARY OBJECTIVES:
- To measure tumor telomere length in archival samples, and to correlate telomere length to the clinical outcome of the study.
OUTLINE:
Patients receive imetelstat sodium intravenously (IV) over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (imetelstat sodium) | Experimental | Drug: Imetelstat Sodium, Other: Laboratory Biomarker AnalysisPatients receive imetelstat sodium IV over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (49 listed)
- Patients with any of the following tumors who have relapsed or refractory disease are eligible:
- Patients must have had histologic verification of malignancy at original diagnosis or relapse
- Patients must have radiographically measurable disease (with the exception of neuroblastoma)
- Patients with neuroblastoma who do not have measurable disease but have evaluable disease on 131I-metaiodobenzylguanidine (MIBG) scans are eligible
- Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
Key exclusion criteria (11 listed)
- Patients who are pregnant or breast-feeding are not eligible for this study; negative 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 an effective contraceptive method for the duration of study therapy; study drug may also potentially be secreted in milk and therefore breastfeeding women are excluded
- Growth factors that support platelet or white cell number or function must not have been administered within the 7 days prior to enrollment (14 days if pegfilgrastim)
- Patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible
- Patients who are currently receiving another investigational drug are not eligible
- Patients who are currently receiving other anti-cancer agents are not eligible
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imetelstat SodiumAlso: GRN163L, GRN163L, Sodium SaltGiven IV | Drug | Treatment (imetelstat sodium) |
| Laboratory Biomarker AnalysisOptional correlative studies | Other | Treatment (imetelstat sodium) |
Primary outcome
Objective response (complete response [CR] or partial response [PR]) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria or MIBG scoring criteria
Time frame: Up to 126 days (6 courses)
Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed accounting for the two-stage design. Each disease stratum will be reported separately.
Incidence of grade 3 or higher adverse events using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time frame: Up to 126 days (6 courses)
Toxicity tables will be constructed to summarize the observed incidence in each reporting period by type of toxicity and grade. The relative frequency of each type of toxicity will be quantified as the number of toxicity-evaluable cycles in which the adverse event (AE) was noted at grade 3 or higher considered by the treating physician to be possibly, probably or definitely related to one of the agents in the regimen divided by the number of toxicity-evaluable courses administered to patients enrolled on the trial.
1 secondary outcome
Progression-free survival
Time frame: Date of enrollment until the end progression-free interval (PFI) date, calculated as the date of disease progression, date of death, date of removal of all tumor by surgery or last patient contact, whichever occurs first, assessed up to 3 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrick Thompson principal investigator
Children's Oncology Group
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Imetelstat Sodium and Laboratory Biomarker Analysis in people with Hepatoblastoma and Previously Treated Childhood Rhabdomyosarcoma, run by Children's Oncology Group.
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 49 inclusion and 11 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 7 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Objective response (complete response [CR] or partial response [PR]) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria or MIBG scoring criteria (time frame: Up to 126 days (6 courses)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Aug 2018.