Solid Tumor, ChildhoodPhase 1CCPO011NCT04485416
Pilot Trial of Eltrombopag in Patients Undergoing Chemotherapy for Malignant Solid Tumors
A Phase 1 study testing Eltrombopag in people with Solid Tumor, Childhood and Solid Tumor, run by University of California, Davis at 1 site in United States.
Trial summary
Primary Objective: To assess safety of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors. Secondary Objectives: To assess the efficacy of eltrombopag in increasing platelet count up to 2 weeks after completion of chemotherapy in pediatric patients undergoing intensive… Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Solid Tumor, Childhood, Solid Tumor
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Jul 2021to 20 Jan 2025
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must meet all of the following criteria to be eligible for study entry.
- Persons aged ≥ 1 to ≤18 years of age.
- Histologically confirmed solid tumors (including rhabdomyosarcoma, Ewings sarcoma, osteosarcoma, non- rhabdomyosarcoma soft tissue sarcoma, peripheral nerve sheath tumor, desmoplastic small round cell tumor, hepatoblastoma, hepatocellular carcinoma, renal cell carcinoma, higher grade neuroblastoma, brain tumors (e.g. medulloblastoma), and other rare solid tumors.
- Currently receiving cancer directed therapy for solid tumor or scheduled to start receiving cancer directed therapy for solid tumor within 60 days.
- Karnofsky Performance Status (KPS) performance status of 80% or greater.
- Life expectancy ≥ 6 months.
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- Ability to swallow liquid solution/suspensions or tablets/capsules
- Platelet count < 150,000µL
- Blood chemistry levels defined by:
- Serum creatinine less than or equal to 2.5 × the upper limit of normal (ULN) range
- Total bilirubin level less than or equal to 1.5 × the upper limit of normal (ULN) range
- AST and ALT < 3 x upper limit of normal (ULN)
- INR and aPTT less than or equal to 1.5 × ULN (for patients on anticoagulation they must be receiving a stable dose for at least 1 week prior to first treatment)
- Ability to understand and willingness to sign an informed consent form; or Parent/Guardian with ability to understand and willingness to sign an informed consent form.
- Ability to adhere to the study visit schedule and other protocol requirements.
Who cannot join
- Patients who meet any of the following criteria will be excluded from study entry.
- Patients with known with hematologic malignancy diagnosis.
- Contraindications to receiving chemotherapy.
- Patients with history of thromboembolic disease or history of thrombophilic risk factors.
- History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study such as uncontrolled or significant cardiac disease, including any of the following:
- Recent myocardial infarction (within last 6 months),
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- Uncontrolled congestive heart failure,
- Unstable angina (within last 6 months),
- Clinically significant (symptomatic) cardiac arrhythmias (e.g., sustained ventricular tachycardia, and clinically significant second or third degree AV block without a pacemaker.)
- Long QT syndrome, family history of idiopathic sudden death, congenital long QT syndrome or additional risk factors for cardiac repolarization abnormality, as determined by the investigator.
- Impaired cardiac function, defined as:
- Corrected QTc >450 msec using Fridericia correction (QTcF) on the screening ECG (using triplicate ECGs),
- Other clinically significant cardio-vascular disease (e.g., uncontrolled hypertension, history of labile hypertension),
- History of known structural abnormalities (e.g. cardiomyopathy).
- Pregnant or lactating women.
- Subjects with liver enzymes 5x upper limit of normal or liver cirrhosis (as determined by the investigator).
- Patients with known history of HIV positivity.
- Patient with known active or uncontrolled infections not responding to appropriate therapy.
- History of alcohol/drug abuse.
- Concurrent participation in an investigational study within 30 days prior to enrollment or within 8 days (> than 5-half-lives)of the investigational product, whichever is longer. Note: parallel enrollment in a disease registry is permitted.
- Known thrombophilic risk factors or history of thromboembolic disease. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
- Known immediate or delayed hypersensitivity reaction to eltrombopag or its excipient.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study treatment. Basic contraception methods include:
- Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
- Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
- Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject.
- Barrier methods of contraception: Condom or Occlusive cap.
- Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception. In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
- Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1.
- Sexually active males unless they use a condom during intercourse while taking the drug during treatment, and for 8 days (> 5 half-lives ) after stopping eltrombopag and for 5 half-lives after the last dose of chemotherapy treatment and should not father a child in this period. A condom is required to be used also by vasectomized men as well as during intercourse with a male partner in order to prevent delivery of the drug via semen.
- Any condition that would prohibit the understanding or rendering of informed consent.
- Any condition that in the opinion of the investigator would interfere with the patient's safety or compliance on trial.
- Severe infection within 4 weeks prior to enrollment that in the opinion of the investigator would interfere with patient safety or compliance on trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California Davis Health System, Comprehensive Cancer Center
Sacramento, California, 95817
About this study
Official title: Open Label Single Arm Prospective Pilot Trial of Eltrombopag in Patients 1 Year to 18 Years of Age Undergoing Intensive Chemotherapy for Malignant Solid Tumors (CCPO011)
Primary Objective: To assess safety of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors.
Secondary Objectives: To assess the efficacy of eltrombopag in increasing platelet count up to 2 weeks after completion of chemotherapy in pediatric patients undergoing intensive chemotherapy for malignant solid tumors.
Hypothesis: The hypothesis is that eltrombopag an oral thrombopoietin receptor agonist will increase the platelet count safely and efficaciously in children having chemotherapy induced thrombocytopenia while on therapy for solid tumors.
Read the detailed description
Eltrombopag is an orally administered, small molecule thrombopoietin receptor (TPO-R) agonist that stimulates platelet production by a mechanism similar, but not identical to endogenous TPO. Eltrombopag interacts with the transmembrane domain of the TPO-R (also known as C-MPL) leading to increased platelet production (Erickson-Miller, 2010; Sun et al, 2012).
Eltrombopag is indicated for the treatment of thrombocytopenia in adult and pediatric patients one year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Eltrombopag has also been approved for the treatment of thrombocytopenia in adult patients with chronic hepatitis C to allow for the initiation and maintenance of interferon-based therapy and for the treatment of cytopenias in adult patients with severe aplastic anemia (SAA) who have had insufficient responses to immunosuppressive therapy.
This is an open label, single center pilot trial of eltrombopag in pediatric patients receiving cancer directed therapy for solid tumors. The purpose of this study is to explore the platelet supportive care effects and safety of eltrombopag in pediatric patients (ages one to 18 years of age) undergoing intensive chemotherapy for malignant solid tumors. The primary endpoint will be to determine the safety of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors using CTCAE v5.0 criteria. The secondary endpoint will be to determine the efficacy of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors. These objectives will be assessed by evaluating drug-related toxicities, the platelet response in patients and the proportion of subjects receiving eltrombopag who are platelet transfusion-free during the time period of chemotherapy.
The study will enroll 10 subjects with histologically confirmed solid tumors, including but not limited to rhabdomyosarcoma, Ewing sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcoma, peripheral nerve sheath tumor, desmoplastic small round cell tumor, hepatoblastoma, hepatocellular carcinoma, renal cell carcinoma, higher grade neuroblastoma, medulloblastoma, or other rare malignant solid tumors.
The cancer directed therapy will be part of standard treatment for each patient and will consist of two to four cycles of chemotherapy (or as many as clinically indicated per physician discretion) to reduce tumor burden, followed by surgery in the majority of cases, and/or radiation in a minority of cases, or both in rare cases at various times in the course of treatment. After recovery from the surgery and/or radiation, chemotherapy and eltrombopag will be resumed until completion. Each cycle of chemotherapy will be approximately two to four weeks (14 to 28 days) in length with chemotherapy administered for one to five days per cycle. The duration of chemotherapy varies by regimen and underlying malignancy.
Patients will initiate eltrombopag on the first day following the completion of chemotherapy for each cycle (e.g., chemo is administered on Days 1-5, eltrombopag to start on Day 6). Eltrombopag will be administered daily and the dose will be age dependent (see Table 5). Children less than 6 years of age will receive a starting dose of 25 mg by mouth once daily, taken on empty stomach one hour before or two hours after a meal. For children greater than or equal to 6 years of age, the starting dose will be 75 mg by mouth once daily. Doses shall be reduced in patients of Asian ancestry (e.g., Japanese, Chinese, Taiwanese, or Korean): for those patients greater than or equal to 6 years of age, the starting dose will be 50 mg by mouth once daily and for those patients less than 6 years old, the starting dose will be 12.5 mg by mouth once daily. Eltrombopag will be continued until the platelets are at least 100,000/µL after the nadir.
Subjects will be recruited from the UC Davis Comprehensive Cancer Center or when they are admitted to UC Davis Children's Hospital.
- Conditions
- Solid Tumor, Childhood, Solid Tumor
- Being tested
- Eltrombopag
- Keywords
- chemotherapy, pediatric
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Promacta
Eltrombopag is an orally administered small-molecule nonpeptide TPO-R agonist.
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | Subjects will receive eltrombopag Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety objectives
For safety our end point is liver enzymes ALT, AST up to 5 x upper limit of normal (ULN) in <80% of patients.
Measured: Through follow up after end of treatment
Secondary outcomes (1)
Efficacy objectives
For efficacy the end point is 6 of 10 patients respond to the study treatment by an increase in platelet counts by 20,000/μL and are able to proceed with their next cycle of chemotherapy.
Measured: 2 weeks after completion of chemotherapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2020
- Study start16 Jul 2021
- Primary completion20 Jan 2025
- Study completion20 Jan 2025
Why it stopped: PI discretion; suspended while CAPA is reviewed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Solid Tumor, ChildhoodStudies of the same condition, open and under way.
- More trials run by University of California, DavisThe 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anjali Pawar, University of California, Davis
- Investigators
- Anjali Pawar, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04485416
- Sponsor's protocol ID
- 1602666
- Other IDs
- 150124, CCPO011
- Acronym
- CCPO011
- Status last verified
- Oct 2026
- First submitted
- 21 Jul 2020
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Geng W, Kearney S, Nelson S. Upfront eltrombopag monotherapy induces stable hematologic remission in pediatric patients with nonsevere idiopathic aplastic anemia. Pediatr Blood Cancer. 2018 Oct;65(10):e27290. doi: 10.1002/pbc.27290. Epub 2018 Jun 22.
PubMed 29932285Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.
PubMed 28423296Soff GA, Miao Y, Bendheim G, Batista J, Mones JV, Parameswaran R, Wilkins CR, Devlin SM, Abou-Alfa GK, Cercek A, Kemeny NE, Sarasohn DM, Mantha S. Romiplostim Treatment of Chemotherapy-Induced Thrombocytopenia. J Clin Oncol. 2019 Nov 1;37(31):2892-2898. doi: 10.1200/JCO.18.01931. Epub 2019 Sep 23.
PubMed 31545663Li S, Wu R, Wang B, Fu L, Zhu G, Zhou X, Ma J, Zhang L, Qin M. Eltrombopag for Delayed Platelet Recovery and Secondary Thrombocytopenia Following Allogeneic Stem Cell Transplantation in Children. J Pediatr Hematol Oncol. 2019 Jan;41(1):38-41. doi: 10.1097/MPH.0000000000001263.
PubMed 30080752Erickson-Miller CL, Kirchner J, Aivado M, May R, Payne P, Chadderton A. Reduced proliferation of non-megakaryocytic acute myelogenous leukemia and other leukemia and lymphoma cell lines in response to eltrombopag. Leuk Res. 2010 Sep;34(9):1224-31. doi: 10.1016/j.leukres.2010.02.005. Epub 2010 Mar 3.
PubMed 20202683Sun H, Tsai Y, Nowak I, Liesveld J, Chen Y. Eltrombopag, a thrombopoietin receptor agonist, enhances human umbilical cord blood hematopoietic stem/primitive progenitor cell expansion and promotes multi-lineage hematopoiesis. Stem Cell Res. 2012 Sep;9(2):77-86. doi: 10.1016/j.scr.2012.05.001. Epub 2012 May 14.
PubMed 22683680Ballmaier M, Germeshausen M, Krukemeier S, Welte K. Thrombopoietin is essential for the maintenance of normal hematopoiesis in humans: development of aplastic anemia in patients with congenital amegakaryocytic thrombocytopenia. Ann N Y Acad Sci. 2003 May;996:17-25. doi: 10.1111/j.1749-6632.2003.tb03228.x.
PubMed 12799278Zeigler FC, de Sauvage F, Widmer HR, Keller GA, Donahue C, Schreiber RD, Malloy B, Hass P, Eaton D, Matthews W. In vitro megakaryocytopoietic and thrombopoietic activity of c-mpl ligand (TPO) on purified murine hematopoietic stem cells. Blood. 1994 Dec 15;84(12):4045-52.
PubMed 7527664Qian H, Buza-Vidas N, Hyland CD, Jensen CT, Antonchuk J, Mansson R, Thoren LA, Ekblom M, Alexander WS, Jacobsen SE. Critical role of thrombopoietin in maintaining adult quiescent hematopoietic stem cells. Cell Stem Cell. 2007 Dec 13;1(6):671-84. doi: 10.1016/j.stem.2007.10.008. Epub 2007 Nov 20.
PubMed 18371408Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. doi: 10.1634/stemcells.2008-0366.
PubMed 19038790Alexander WS, Roberts AW, Nicola NA, Li R, Metcalf D. Deficiencies in progenitor cells of multiple hematopoietic lineages and defective megakaryocytopoiesis in mice lacking the thrombopoietic receptor c-Mpl. Blood. 1996 Mar 15;87(6):2162-70.
PubMed 8630375Kimura S, Roberts AW, Metcalf D, Alexander WS. Hematopoietic stem cell deficiencies in mice lacking c-Mpl, the receptor for thrombopoietin. Proc Natl Acad Sci U S A. 1998 Feb 3;95(3):1195-200. doi: 10.1073/pnas.95.3.1195.
PubMed 9448308Kellum A, Jagiello-Gruszfeld A, Bondarenko IN, Patwardhan R, Messam C, Mostafa Kamel Y. A randomized, double-blind, placebo-controlled, dose ranging study to assess the efficacy and safety of eltrombopag in patients receiving carboplatin/paclitaxel for advanced solid tumors. Curr Med Res Opin. 2010 Oct;26(10):2339-46. doi: 10.1185/03007995.2010.510051.
PubMed 20735290Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. doi: 10.1056/NEJMoa1200931.
PubMed 22762314
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Single Arm Prospective Pilot Trial of Eltrombopag in Patients 1 Year to 18 Years of Age Undergoing Intensive Chemotherapy for Malignant Solid Tumors (CCPO011)
- ClinicalTrials.gov ID
- NCT04485416
- Acronym / sponsor ID
- CCPO011 / 1602666
- Conditions
- Solid Tumor, Childhood, Solid Tumor
- Keywords
- chemotherapy, pediatric
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PI discretion; suspended while CAPA is reviewed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Study start
- 16 Jul 2021 (actual)
- Primary completion
- 20 Jan 2025 (actual)
- Study completion
- 20 Jan 2025 (actual)
- First posted
- 24 Jul 2020 (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.
Primary Objective: To assess safety of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors.
Secondary Objectives: To assess the efficacy of eltrombopag in increasing platelet count up to 2 weeks after completion of chemotherapy in pediatric patients undergoing intensive chemotherapy for malignant solid tumors.
Hypothesis: The hypothesis is that eltrombopag an oral thrombopoietin receptor agonist will increase the platelet count safely and efficaciously in children having chemotherapy induced thrombocytopenia while on therapy for solid tumors.
Detailed description
Eltrombopag is an orally administered, small molecule thrombopoietin receptor (TPO-R) agonist that stimulates platelet production by a mechanism similar, but not identical to endogenous TPO. Eltrombopag interacts with the transmembrane domain of the TPO-R (also known as C-MPL) leading to increased platelet production (Erickson-Miller, 2010; Sun et al, 2012).
Eltrombopag is indicated for the treatment of thrombocytopenia in adult and pediatric patients one year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Eltrombopag has also been approved for the treatment of thrombocytopenia in adult patients with chronic hepatitis C to allow for the initiation and maintenance of interferon-based therapy and for the treatment of cytopenias in adult patients with severe aplastic anemia (SAA) who have had insufficient responses to immunosuppressive therapy.
This is an open label, single center pilot trial of eltrombopag in pediatric patients receiving cancer directed therapy for solid tumors. The purpose of this study is to explore the platelet supportive care effects and safety of eltrombopag in pediatric patients (ages one to 18 years of age) undergoing intensive chemotherapy for malignant solid tumors. The primary endpoint will be to determine the safety of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors using CTCAE v5.0 criteria. The secondary endpoint will be to determine the efficacy of eltrombopag in pediatric patients undergoing intensive chemotherapy for malignant solid tumors. These objectives will be assessed by evaluating drug-related toxicities, the platelet response in patients and the proportion of subjects receiving eltrombopag who are platelet transfusion-free during the time period of chemotherapy.
The study will enroll 10 subjects with histologically confirmed solid tumors, including but not limited to rhabdomyosarcoma, Ewing sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcoma, peripheral nerve sheath tumor, desmoplastic small round cell tumor, hepatoblastoma, hepatocellular carcinoma, renal cell carcinoma, higher grade neuroblastoma, medulloblastoma, or other rare malignant solid tumors.
The cancer directed therapy will be part of standard treatment for each patient and will consist of two to four cycles of chemotherapy (or as many as clinically indicated per physician discretion) to reduce tumor burden, followed by surgery in the majority of cases, and/or radiation in a minority of cases, or both in rare cases at various times in the course of treatment. After recovery from the surgery and/or radiation, chemotherapy and eltrombopag will be resumed until completion. Each cycle of chemotherapy will be approximately two to four weeks (14 to 28 days) in length with chemotherapy administered for one to five days per cycle. The duration of chemotherapy varies by regimen and underlying malignancy.
Patients will initiate eltrombopag on the first day following the completion of chemotherapy for each cycle (e.g., chemo is administered on Days 1-5, eltrombopag to start on Day 6). Eltrombopag will be administered daily and the dose will be age dependent (see Table 5). Children less than 6 years of age will receive a starting dose of 25 mg by mouth once daily, taken on empty stomach one hour before or two hours after a meal. For children greater than or equal to 6 years of age, the starting dose will be 75 mg by mouth once daily. Doses shall be reduced in patients of Asian ancestry (e.g., Japanese, Chinese, Taiwanese, or Korean): for those patients greater than or equal to 6 years of age, the starting dose will be 50 mg by mouth once daily and for those patients less than 6 years old, the starting dose will be 12.5 mg by mouth once daily. Eltrombopag will be continued until the platelets are at least 100,000/µL after the nadir.
Subjects will be recruited from the UC Davis Comprehensive Cancer Center or when they are admitted to UC Davis Children's Hospital.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Drug: EltrombopagSubjects will receive eltrombopag |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Patients must meet all of the following criteria to be eligible for study entry.
- Persons aged ≥ 1 to ≤18 years of age.
- Histologically confirmed solid tumors (including rhabdomyosarcoma, Ewings sarcoma, osteosarcoma, non- rhabdomyosarcoma soft tissue sarcoma, peripheral nerve sheath tumor, desmoplastic small round cell tumor, hepatoblastoma, hepatocellular carcinoma, renal cell carcinoma, higher grade neuroblastoma, brain tumors (e.g. medulloblastoma), and other rare solid tumors.
- Currently receiving cancer directed therapy for solid tumor or scheduled to start receiving cancer directed therapy for solid tumor within 60 days.
- Karnofsky Performance Status (KPS) performance status of 80% or greater.
Key exclusion criteria (33 listed)
- Patients who meet any of the following criteria will be excluded from study entry.
- Patients with known with hematologic malignancy diagnosis.
- Contraindications to receiving chemotherapy.
- Patients with history of thromboembolic disease or history of thrombophilic risk factors.
- History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study such as uncontrolled or significant cardiac disease, including any of the following:
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 |
|---|---|---|
| EltrombopagAlso: PromactaEltrombopag is an orally administered small-molecule nonpeptide TPO-R agonist. | Drug | Treatment group |
Primary outcome
Safety objectives
Time frame: Through follow up after end of treatment
For safety our end point is liver enzymes ALT, AST up to 5 x upper limit of normal (ULN) in <80% of patients.
1 secondary outcome
Efficacy objectives
Time frame: 2 weeks after completion of chemotherapy
Results and safety
The study was stopped early (reason given: PI discretion; suspended while CAPA is reviewed). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Background: PubMed 29932285Geng W, Kearney S, Nelson S. Upfront eltrombopag monotherapy induces stable hematologic remission in pediatric patients with nonsevere idiopathic aplastic anemia. Pediatr Blood Cancer. 2018 Oct;65(10):e27290. doi: 10.1002/pbc.27290. Epub 2018 Jun 22.
- Background: PubMed 28423296Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.
- Background: PubMed 31545663Soff GA, Miao Y, Bendheim G, Batista J, Mones JV, Parameswaran R, Wilkins CR, Devlin SM, Abou-Alfa GK, Cercek A, Kemeny NE, Sarasohn DM, Mantha S. Romiplostim Treatment of Chemotherapy-Induced Thrombocytopenia. J Clin Oncol. 2019 Nov 1;37(31):2892-2898. doi: 10.1200/JCO.18.01931. Epub 2019 Sep 23.
- Background: PubMed 30080752Li S, Wu R, Wang B, Fu L, Zhu G, Zhou X, Ma J, Zhang L, Qin M. Eltrombopag for Delayed Platelet Recovery and Secondary Thrombocytopenia Following Allogeneic Stem Cell Transplantation in Children. J Pediatr Hematol Oncol. 2019 Jan;41(1):38-41. doi: 10.1097/MPH.0000000000001263.
- Background: PubMed 20202683Erickson-Miller CL, Kirchner J, Aivado M, May R, Payne P, Chadderton A. Reduced proliferation of non-megakaryocytic acute myelogenous leukemia and other leukemia and lymphoma cell lines in response to eltrombopag. Leuk Res. 2010 Sep;34(9):1224-31. doi: 10.1016/j.leukres.2010.02.005. Epub 2010 Mar 3.
- Background: PubMed 22683680Sun H, Tsai Y, Nowak I, Liesveld J, Chen Y. Eltrombopag, a thrombopoietin receptor agonist, enhances human umbilical cord blood hematopoietic stem/primitive progenitor cell expansion and promotes multi-lineage hematopoiesis. Stem Cell Res. 2012 Sep;9(2):77-86. doi: 10.1016/j.scr.2012.05.001. Epub 2012 May 14.
- Background: PubMed 12799278Ballmaier M, Germeshausen M, Krukemeier S, Welte K. Thrombopoietin is essential for the maintenance of normal hematopoiesis in humans: development of aplastic anemia in patients with congenital amegakaryocytic thrombocytopenia. Ann N Y Acad Sci. 2003 May;996:17-25. doi: 10.1111/j.1749-6632.2003.tb03228.x.
- Background: PubMed 7527664Zeigler FC, de Sauvage F, Widmer HR, Keller GA, Donahue C, Schreiber RD, Malloy B, Hass P, Eaton D, Matthews W. In vitro megakaryocytopoietic and thrombopoietic activity of c-mpl ligand (TPO) on purified murine hematopoietic stem cells. Blood. 1994 Dec 15;84(12):4045-52.
- Background: PubMed 18371408Qian H, Buza-Vidas N, Hyland CD, Jensen CT, Antonchuk J, Mansson R, Thoren LA, Ekblom M, Alexander WS, Jacobsen SE. Critical role of thrombopoietin in maintaining adult quiescent hematopoietic stem cells. Cell Stem Cell. 2007 Dec 13;1(6):671-84. doi: 10.1016/j.stem.2007.10.008. Epub 2007 Nov 20.
- Background: PubMed 19038790Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. doi: 10.1634/stemcells.2008-0366.
- Background: PubMed 8630375Alexander WS, Roberts AW, Nicola NA, Li R, Metcalf D. Deficiencies in progenitor cells of multiple hematopoietic lineages and defective megakaryocytopoiesis in mice lacking the thrombopoietic receptor c-Mpl. Blood. 1996 Mar 15;87(6):2162-70.
- Background: PubMed 9448308Kimura S, Roberts AW, Metcalf D, Alexander WS. Hematopoietic stem cell deficiencies in mice lacking c-Mpl, the receptor for thrombopoietin. Proc Natl Acad Sci U S A. 1998 Feb 3;95(3):1195-200. doi: 10.1073/pnas.95.3.1195.
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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.
Anjali Pawar, MD principal investigator
University of California, Davis
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Eltrombopag in people with Solid Tumor, Childhood and Solid Tumor, run by University of California, Davis at 1 site 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 15 inclusion and 33 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: stopped early, as of the sponsor's last update on 9 Oct 2026. 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 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 stopped early (reason given: PI discretion; suspended while CAPA is reviewed). Results are not available in the source record.
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?
Safety objectives (time frame: Through follow up after end of treatment). 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 NCT04485416, which also lists 14 linked publications.
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 9 Oct 2026.