NeuroblastomaPhase 1NCT04106219
A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma
A Phase 1 study testing LY3295668 Erbumine, Topotecan and Cyclophosphamide in people with Neuroblastoma, run by Eli Lilly and Company at 17 sites in 9 countries.
Trial summary
The reason for this study is to see if the study drug LY3295668 erbumine is safe in participants with relapsed/refractory neuroblastoma. Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- LY3295668 Erbumine, Topotecan and Cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 17 sitesin 9 countries
- Study dates
- 11 Jun 2020to Aug 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 2 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must have relapsed/refractory neuroblastoma and have active disease in at least one site: bone, bone marrow or soft tissue. Participants must be able to submit an archival sample of tissue.
- Participants must be able to swallow capsules.
Who cannot join
- Participants must not have had an allogeneic hematopoietic stem cell, bone marrow, or solid organ transplant.
- Participants must not have untreated tumor that has spread to the brain or spinal cord.
- Participants must not have a serious active disease other than neuroblastoma.
- Participants must not have a condition affecting absorption.
- Participants must not have had prior aurora kinase inhibitor exposure.
- Participants must not have a known allergy to the study treatment.
- Participants must not have symptomatic human immunodeficiency virus (HIV) infection or symptomatic activated/reactivated hepatitis A, B, or C.
Trial locations and contacts
17 sites in 9 countries.
17 sites in 9 countries
United States7 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
University of Chicago - Comer Children's Hospital
Chicago, Illinois, 60637
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Childrens Hospital
Houston, Texas, 77030
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UCSF Medical Center at Mission Bay
San Francisco, California, 94158
France2 sites
Centre Leon Berard
Lyon, Auvergne-Rhône-Alpes, 69373 CEDEX 08
Institut Curie
Paris, 75248
Spain2 sites
Hospital Universitari Vall d'Hebron
Barcelona, Barcelona [Barcelona], 8035
Hospital Infantil Universitario Niño Jesús
Madrid, 28009
About this study
Official title: A Phase 1 Study of Aurora Kinase A Inhibitor LY3295668 Erbumine as a Single Agent and in Combination in Patients With Relapsed/Refractory Neuroblastoma
The reason for this study is to see if the study drug LY3295668 erbumine is safe in participants with relapsed/refractory neuroblastoma.
- Conditions
- Neuroblastoma
- Being tested
- LY3295668 Erbumine, Topotecan, Cyclophosphamide
- Keywords
- aurora kinase A, kinase, aurora A, aurora kinase inhibitor, aurora kinase A inhibitor, kinase inhibitor, AURKA, AurA, pediatric neuroblastoma, children
- Medical subject headings
- Neuroblastoma, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY3295668 ErbumineDrug
Administered orally.
TopotecanDrug
Administered IV.
CyclophosphamideDrug
Administered IV.
| Study group | Role | What they receive |
|---|---|---|
| LY3295668 Erbumine Escalation | Experimental | LY3295668 Erbumine given orally. Receives: Drug: LY3295668 Erbumine |
| LY3295668 Erbumine + Topotecan + Cyclophosphamide Escalation | Experimental | LY3295668 Erbumine given orally and topotecan and cyclophosphamide given intravenously (IV). Receives: Drug: LY3295668 Erbumine; Drug: Topotecan; Drug: Cyclophosphamide |
| LY3295668 Erbumine Expansion | Experimental | LY3295668 Erbumine given orally. Receives: Drug: LY3295668 Erbumine |
| LY3295668 Erbumine + Topotecan + Cyclophosphamide Expansion | Experimental | LY3295668 Erbumine given orally and topotecan and cyclophosphamide given IV. Receives: Drug: LY3295668 Erbumine; Drug: Topotecan; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Dose Limiting Toxicities (DLTs)
Number of Participants with DLTs
Measured: Baseline through Cycle 2 (28 Day Cycle)
Overall Response Rate (ORR): Percentage of Participants Who Achieve Complete Response (CR) or Partial Response (PR)
ORR
Measured: Baseline through Measured Progressive Disease (Estimated up to 5 Years)
Duration of Response (DoR)
DoR
Measured: Date of CR or PR to Date of Disease Progression or Death Due to Any Cause (Estimated up to 5 Years)
Secondary outcomes (5)
Pharmacokinetics (PK): Area Under the Concentration Time Curve (AUC) of LY3295668
PK: AUC of LY3295668
Measured: Cycle 1 Day 1 through Cycle 1 Day 15 (28 Day Cycles)
PK: AUC of LY3295668 in Combination with Topotecan and Cyclophosphamide
PK: AUC of LY3295668 in Combination with topotecan and cyclophosphamide
Measured: Cycle 1 Day 1 through Cycle 1 Day 15 (28 Day Cycles)
Best Overall Response (BOR): Percentage of Participants with CR, PR, Stable Disease (SD), or Progressive Disease (PD)
BOR
Measured: Baseline to Date of Objective Disease Progression (Estimated up to 5 Years)
Progression-Free Survival (PFS)
PFS
Measured: Baseline to Objective Progression or Death Due to Any Cause (Estimated up to 5 Years)
Overall Survival (OS)
OS
Measured: Baseline to Date of Death from Any Cause (Estimated up to 6 Years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2019
- Study start11 Jun 2020
- Primary completion13 Apr 2022
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 71 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- New Approaches to Neuroblastoma Therapy Consortium, Innovative Therapies for Children with Cancer in Europe (ITCC)
- 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
- NCT04106219
- Sponsor's protocol ID
- 17295
- Other IDs
- J1O-MC-JZHD, 2019-001042-18, 2019-01, ITCC-085, 2019
- Status last verified
- Aug 2026
- First submitted
- 25 Sep 2019
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of Aurora Kinase A Inhibitor LY3295668 Erbumine as a Single Agent and in Combination in Patients With Relapsed/Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT04106219
- Acronym / sponsor ID
- 17295
- Conditions
- Neuroblastoma
- Keywords
- aurora kinase A, kinase, aurora A, aurora kinase inhibitor, aurora kinase A inhibitor, kinase inhibitor, AURKA, AurA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 71 participants (estimated)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Collaborators
- New Approaches to Neuroblastoma Therapy Consortium, Innovative Therapies for Children with Cancer in Europe (ITCC)
- Study start
- 11 Jun 2020 (actual)
- Primary completion
- 13 Apr 2022 (actual)
- Study completion
- Aug 2027 (estimated)
- First posted
- 26 Sep 2019 (actual)
- 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 reason for this study is to see if the study drug LY3295668 erbumine is safe in participants with relapsed/refractory neuroblastoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| LY3295668 Erbumine Escalation | Experimental | Drug: LY3295668 ErbumineLY3295668 Erbumine given orally. |
| LY3295668 Erbumine + Topotecan + Cyclophosphamide Escalation | Experimental | Drug: LY3295668 Erbumine, Drug: Topotecan, Drug: CyclophosphamideLY3295668 Erbumine given orally and topotecan and cyclophosphamide given intravenously (IV). |
| LY3295668 Erbumine Expansion | Experimental | Drug: LY3295668 ErbumineLY3295668 Erbumine given orally. |
| LY3295668 Erbumine + Topotecan + Cyclophosphamide Expansion | Experimental | Drug: LY3295668 Erbumine, Drug: Topotecan, Drug: CyclophosphamideLY3295668 Erbumine given orally and topotecan and cyclophosphamide given IV. |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Participants must have relapsed/refractory neuroblastoma and have active disease in at least one site: bone, bone marrow or soft tissue. Participants must be able to submit an archival sample of tissue.
- Participants must be able to swallow capsules.
Key exclusion criteria (7 listed)
- Participants must not have had an allogeneic hematopoietic stem cell, bone marrow, or solid organ transplant.
- Participants must not have untreated tumor that has spread to the brain or spinal cord.
- Participants must not have a serious active disease other than neuroblastoma.
- Participants must not have a condition affecting absorption.
- Participants must not have had prior aurora kinase inhibitor exposure.
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 |
|---|---|---|
| LY3295668 ErbumineAdministered orally. | Drug | LY3295668 Erbumine + Topotecan + Cyclophosphamide Escalation, LY3295668 Erbumine + Topotecan + Cyclophosphamide Expansion, LY3295668 Erbumine Escalation, LY3295668 Erbumine Expansion |
| TopotecanAdministered IV. | Drug | LY3295668 Erbumine + Topotecan + Cyclophosphamide Escalation, LY3295668 Erbumine + Topotecan + Cyclophosphamide Expansion |
| CyclophosphamideAdministered IV. | Drug | LY3295668 Erbumine + Topotecan + Cyclophosphamide Escalation, LY3295668 Erbumine + Topotecan + Cyclophosphamide Expansion |
Primary outcome
Number of Participants with Dose Limiting Toxicities (DLTs)
Time frame: Baseline through Cycle 2 (28 Day Cycle)
Number of Participants with DLTs
Overall Response Rate (ORR): Percentage of Participants Who Achieve Complete Response (CR) or Partial Response (PR)
Time frame: Baseline through Measured Progressive Disease (Estimated up to 5 Years)
ORR
Duration of Response (DoR)
Time frame: Date of CR or PR to Date of Disease Progression or Death Due to Any Cause (Estimated up to 5 Years)
DoR
5 secondary outcomes
Pharmacokinetics (PK): Area Under the Concentration Time Curve (AUC) of LY3295668
Time frame: Cycle 1 Day 1 through Cycle 1 Day 15 (28 Day Cycles)
PK: AUC of LY3295668 in Combination with Topotecan and Cyclophosphamide
Time frame: Cycle 1 Day 1 through Cycle 1 Day 15 (28 Day Cycles)
Best Overall Response (BOR): Percentage of Participants with CR, PR, Stable Disease (SD), or Progressive Disease (PD)
Time frame: Baseline to Date of Objective Disease Progression (Estimated up to 5 Years)
Progression-Free Survival (PFS)
Time frame: Baseline to Objective Progression or Death Due to Any Cause (Estimated up to 5 Years)
Overall Survival (OS)
Time frame: Baseline to Date of Death from Any Cause (Estimated up to 6 Years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory NeuroblastomaLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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
17 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LY3295668 Erbumine, Topotecan and Cyclophosphamide in people with Neuroblastoma, run by Eli Lilly and Company at 17 sites in 9 countries.
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 2 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, France, Spain, Australia, Belgium and 4 more countries. 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of Participants with Dose Limiting Toxicities (DLTs) (time frame: Baseline through Cycle 2 (28 Day Cycle)). The study lists 3 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 21 Aug 2026; AzTrials last refreshed its copy on 7 Nov 2026.