Medulloblastoma, ChildhoodPhase 1NCT01697514
A Study of LY2940680 in Pediatric Medulloblastoma or Rhabdomyosarcoma
A Phase 1 study testing LY2940680 in people with Medulloblastoma, Childhood and Rhabdomyosarcoma, run by Eli Lilly and Company at 4 sites in United States.
Trial summary
The purpose of this study is to find a recommended dose level of LY2940680 that can be safely given to children with medulloblastoma or rhabdomyosarcoma that has returned or doesn't respond to initial treatment. The study will also explore the changes in a cancer marker levels. Read more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- Medulloblastoma, Childhood, Rhabdomyosarcoma
- What is being tested?
- LY2940680
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 4 sitesUnited States
- Study dates
- Jul 2013to Jul 2016
- 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 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- For Part A: Have a diagnosis of recurrent or refractory rhabdomyosarcoma or medulloblastoma and have had histologic verification of malignancy at original diagnosis or relapse.
- For Part B: Have a diagnosis of recurrent or refractory medulloblastoma and have had histologic verification of malignancy at original diagnosis or relapse.
- 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.
- Karnofsky score must be at least 50% for participants >16 years of age, and Lansky score must be at least 50% for participants 16 years of age or less. Participants who are unable to walk because of paralysis, but who are in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
- Have fully recovered from the acute toxic effects of all prior anticancer chemotherapy.
- Participants with solid tumors must not have received myelosuppressive chemotherapy within 3 weeks of enrollment in this study (6 weeks, if previously treated with nitrosourea).
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- Hematopoietic growth factors: At least 14 days after the last dose of a longacting growth factor (eg, Neulasta®) or 7 days for short-acting growth factor.
- Biologic (antineoplastic agent): At least 7 days after the last dose of a biologic agent.
- Immunotherapy: At least 6 weeks since the completion of any type of immunotherapy (eg, tumor vaccines).
- Monoclonal antibodies: At least 3 half-lives of the antibody after the last dose of a monoclonal antibody.
- Radiation therapy (XRT): ≥8 weeks for local irradiation to primary tumor;≥2 weeks prior to study entry for focal irradiation for symptomatic metastatic sites; ≥3 months for craniospinal XRT, or ≥24 weeks if ≥50% radiation of pelvis; minimum of 6 weeks must have elapsed if other substantial bone marrow radiation has been received.
- Stem cell transplant: allowed if they have recovered from all acute toxicity and adequate bone marrow reserve is demonstrated. At least 8 weeks must have elapsed since autologous stem cell transplantation or ≥3 months for allogenic transplantation. Participants must be off all immunosuppressive therapy and have no evidence of active graft-versus-host disease.
- Have adequate organ function, including:
- Bone marrow: Peripheral absolute neutrophil count (ANC) ≥500/cubic millimeter (mm^3) and platelet count ≥100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to enrollment).
- Hepatic: Bilirubin (sum of conjugated + unconjugated)≤1.5 × upper limit of normal (ULN) for age. Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.0 times ULN for age. Serum albumin ≥2 grams/deciliter (g/dL).
- Renal: Creatinine clearance or radioisotope glomerular filtration rate (GFR)
- ≥70 milliliters/minute/1.73 square meters (mL/min/1.73 m^2), or a serum creatinine based on age/gender per the Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control and Prevention (CDC).
- Neurologic: Participants with seizure disorders may be enrolled if receiving nonenzyme-inducing anticonvulsants and if the symptoms are well controlled. They must have a stable neurologic status for at least 1 week prior to enrollment in the study.
- Must be able to swallow powder or a capsule.
- Have the presence of either measurable or nonmeasurable disease.
Who cannot join
- Have received treatment within 21 days of the initial dose of study drug with an experimental agent for noncancer indications that has not received regulatory approval for any indication.
- Receiving corticosteroids and have not been on a stable or decreasing dose of corticosteroid for the prior 7 days.
- Receiving enzyme-inducing anticonvulsants.
- Have serious preexisting medical conditions.
- Have current hematologic malignancies or acute or chronic leukemia.
- Have a known active fungal, bacterial, and/or known viral infection, including human immunodeficiency virus (HIV) or viral (A, B, or C) hepatitis (screening is not required).
- Have a second primary malignancy that, in the judgment of the investigator and sponsor, may affect the interpretation of results.
- Have ≥Grade 2 QT prolongation, that is, QTc interval of >480 milliseconds (msec) on screening electrocardiography (ECG).
Trial locations and contacts
4 sites in 1 country.
United States4 sites
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Aurora, Colorado, 80045
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Cincinnati, Ohio, 45229
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Portland, Oregon, 97239
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
San Diego, California, 92123
About this study
Official title: A Phase 1 Trial of LY2940680 in Pediatric Patients With Recurrent or Refractory Rhabdomyosarcoma or Medulloblastoma
The purpose of this study is to find a recommended dose level of LY2940680 that can be safely given to children with medulloblastoma or rhabdomyosarcoma that has returned or doesn't respond to initial treatment. The study will also explore the changes in a cancer marker levels. Finally, the study will help document any antitumor activity.
- Conditions
- Medulloblastoma, Childhood, Rhabdomyosarcoma
- Being tested
- LY2940680
- Keywords
- recurrent medulloblastoma
- Medical subject headings
- Medulloblastoma, Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY2940680Drug
Capsule administered orally.
LY2940680Drug
Powder administered orally (may be sprinkled on soft food).
| Study group | Role | What they receive |
|---|---|---|
| Part A: LY2940680 (Dose Escalation) | Experimental | LY2940680 administered orally once daily at escalating doses (92.5 milligrams per square meter [mg/m^2] up to 370 mg/m^2) for two 28 day cycles. Lower dose levels (23 mg/m^2 and 46 mg/m^2) may also be explored, if necessary. Participants receiving benefit may continue until disease progression, unacceptable toxicity, or discontinuation. Receives: Drug: LY2940680 |
| Part B: LY2940680 (Dose Confirmation) | Experimental | LY2940680 administered orally once daily for two 28 day cycles. Dose based on Part A. Participants receiving benefit may continue until disease progression, unacceptable toxicity, or discontinuation. Receives: Drug: LY2940680 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose of LY2940680
Measured: Baseline to Part A Completion (estimated as 12 months)
Secondary outcomes (4)
Pharmacokinetics: Area Under the Concentration - Time Curve (AUC) of LY2940680 and LSN2941091
Measured: Predose up to 24 hours Postdose
Pharmacokinetics: Maximum Concentration (Cmax) of LY2940680 and LSN2941091
Measured: Predose up to 24 hours Postdose
Pharmacokinetics: Time of Maximal Concentration (Tmax) of LY2940680 and LSN2941091
Measured: Predose up to 24 hours Postdose
Number of Participants with Tumor Response
Measured: Baseline to Study Completion (estimated as 44 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2012 (estimated)
- Study startJul 2013
- Primary completionJul 2016 (estimated)
- Study completionJul 2016 (estimated)
Why it stopped: Trial stopped early for poor accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Medulloblastoma, ChildhoodStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01697514
- Sponsor's protocol ID
- 14492
- Other IDs
- I4J-MC-HHBD
- Status last verified
- Aug 2013
- First submitted
- 28 Sep 2012
- Last updated
- 27 Aug 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Trial of LY2940680 in Pediatric Patients With Recurrent or Refractory Rhabdomyosarcoma or Medulloblastoma
- ClinicalTrials.gov ID
- NCT01697514
- Acronym / sponsor ID
- 14492
- Conditions
- Medulloblastoma, Childhood, Rhabdomyosarcoma
- Keywords
- recurrent medulloblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Trial stopped early for poor accrual.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Study start
- Jul 2013
- Primary completion
- Jul 2016 (estimated)
- Study completion
- Jul 2016 (estimated)
- First posted
- 2 Oct 2012 (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.
The purpose of this study is to find a recommended dose level of LY2940680 that can be safely given to children with medulloblastoma or rhabdomyosarcoma that has returned or doesn't respond to initial treatment. The study will also explore the changes in a cancer marker levels. Finally, the study will help document any antitumor activity.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part A: LY2940680 (Dose Escalation) | Experimental | Drug: LY2940680LY2940680 administered orally once daily at escalating doses (92.5 milligrams per square meter [mg/m^2] up to 370 mg/m^2) for two 28 day cycles. Lower dose levels (23 mg/m^2 and 46 mg/m^2) may also be explored, if necessary. Participants receiving benefit may continue until disease progression, unacceptable toxicity, or discontinuation. |
| Part B: LY2940680 (Dose Confirmation) | Experimental | Drug: LY2940680LY2940680 administered orally once daily for two 28 day cycles. Dose based on Part A. Participants receiving benefit may continue until disease progression, unacceptable toxicity, or discontinuation. |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (20 listed)
- For Part A: Have a diagnosis of recurrent or refractory rhabdomyosarcoma or medulloblastoma and have had histologic verification of malignancy at original diagnosis or relapse.
- For Part B: Have a diagnosis of recurrent or refractory medulloblastoma and have had histologic verification of malignancy at original diagnosis or relapse.
- 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.
- Karnofsky score must be at least 50% for participants >16 years of age, and Lansky score must be at least 50% for participants 16 years of age or less. Participants who are unable to walk because of paralysis, but who are in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
- Have fully recovered from the acute toxic effects of all prior anticancer chemotherapy.
Key exclusion criteria (8 listed)
- Have received treatment within 21 days of the initial dose of study drug with an experimental agent for noncancer indications that has not received regulatory approval for any indication.
- Receiving corticosteroids and have not been on a stable or decreasing dose of corticosteroid for the prior 7 days.
- Receiving enzyme-inducing anticonvulsants.
- Have serious preexisting medical conditions.
- Have current hematologic malignancies or acute or chronic leukemia.
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 |
|---|---|---|
| LY2940680Capsule administered orally. | Drug | Part A: LY2940680 (Dose Escalation), Part B: LY2940680 (Dose Confirmation) |
| LY2940680Powder administered orally (may be sprinkled on soft food). | Drug | Part A: LY2940680 (Dose Escalation), Part B: LY2940680 (Dose Confirmation) |
Primary outcome
Maximum Tolerated Dose of LY2940680
Time frame: Baseline to Part A Completion (estimated as 12 months)
4 secondary outcomes
Pharmacokinetics: Area Under the Concentration - Time Curve (AUC) of LY2940680 and LSN2941091
Time frame: Predose up to 24 hours Postdose
Pharmacokinetics: Maximum Concentration (Cmax) of LY2940680 and LSN2941091
Time frame: Predose up to 24 hours Postdose
Pharmacokinetics: Time of Maximal Concentration (Tmax) of LY2940680 and LSN2941091
Time frame: Predose up to 24 hours Postdose
Number of Participants with Tumor Response
Time frame: Baseline to Study Completion (estimated as 44 months)
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 27 Aug 2013.
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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.
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) study director
Eli Lilly and Company
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LY2940680 in people with Medulloblastoma, Childhood and Rhabdomyosarcoma, run by Eli Lilly and Company at 4 sites in United States.
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 20 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 27 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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?
Maximum Tolerated Dose of LY2940680 (time frame: Baseline to Part A Completion (estimated as 12 months)). 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 27 Aug 2013.