Immune ThrombocytopeniaPhase 3NCT03939637
Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children
A Phase 3 study testing Eltrombopag, Steroids and IVIG in people with Immune Thrombocytopenia, run by Baylor College of Medicine at 27 sites in United States.
Trial summary
This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Eltrombopag, Steroids and IVIG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 122 participants
- Where
- 27 sitesUnited States
- Study dates
- 2 May 2019to 26 Feb 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Age: 1- <18 years
- Newly diagnosed ITP (<3 months from diagnosis (first abnormal platelet count), per international working group definition17)
- Platelets <30 x 10^9/L at screening
- Requires pharmacologic treatment from the perspective of the treating clinician.
- Need to treat is at the discretion of the investigator, but there should be clinical equipoise about the use of eltrombopag vs standard treatment options (patients should not, in the opinion of the investigator, require concomitant therapy at time of enrollment).
- Treatment options include one of three standard therapies, (IVIg, steroids, or Anti-D). For example, if patient has previously shown no response to IVIg or steroids and is Rh-negative, patient would not be eligible for study.
Show all 13Show fewer
- Patient population includes both:
- Upfront treatment: Patient within 10 days of ITP diagnosis who has not received previous treatment OR
- Treatment failure: Patients who have failed standard management (observation or treatment with one or more first-line agents)
- Failure of observation: no platelet recovery (>30 x 10^9/L) with observation >10 days from diagnosis, with need to treat
- Poor response to first-line agent (platelets remain <30 x10^9/L)
- Initial response to first-line agent, but response wanes and platelets fall below 30 x10^9/L
- Family willing and able to return for required lab studies
Who cannot join
- Severe bleeding: Buchanan Overall Grade 4 or 5 bleeding, or severe bleeding requiring emergent treatment at the discretion of the provider. (e.g., intracranial hemorrhage, pulmonary hemorrhage, bleeding with ongoing need for pRBC transfusion)
- Prior treatment with TPO-RA (eltrombopag or romiplostim)
- Known secondary ITP (due to lupus, CVID, ALPS)
- Known HIV (or history of HIV positivity) or Hepatitis C (screening not required if no clinical suspicion)
- Evans Syndrome: positive direct Coombs with evidence of active hemolysis (elevated lactate dehydrogenase (LDH) or reticulocyte count not attributable to recent treatment or bleeding)
- Any Malignancy
Show all 38Show fewer
- History of stem cell transplant or solid organ transplant
- aspartate aminotransferase (AST) or ALT >2 x upper limit of normal (ULN)
- Total bilirubin >1.5 × ULN
- Subjects with liver cirrhosis (as determined by the investigator)
- Creatinine >2.5 × ULN
- Known active or uncontrolled infections not responding to appropriate therapy
- On anticoagulation or anti-platelet agents
- Known thrombophilic risk factors. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
- Baseline ophthalmic problems that may potentiate cataract development
- Impaired cardiac function, such as:
- Known prolonged QTc, with corrected QTc >450 msec
- Other clinically significant cardio-vascular disease (e.g., uncontrolled hypertension, history of labile hypertension),
- History of known structural abnormalities (e.g. cardiomyopathy).
- 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),
- 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.
- Known immediate or delayed hypersensitivity reaction to eltrombopag or its excipient.
- Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. Women of childbearing potential (have achieved menarche) must have a negative serum or urine pregnancy test and agree to use basic methods of contraception (if sexually active) or maintain abstinence for the duration of the study. 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. For the UK: with spermicidal foam/gel/film/cream/ vaginal suppository
- 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.
- Male patients who are sexually active and do not agree to abstinence or to use a condom during intercourse while taking eltrombopag, and for 7 days after stopping treatment.
- History of alcohol/drug abuse
- Presence of a medical condition that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Concurrent participation in an investigational study within 30 days prior to enrollment or within 5-half-lives of the investigational product, whichever is longer. Note: parallel enrollment in a non-therapeutic trial such as disease registry or biology study is permitted.
- Other Eligibility Criteria Considerations All patients and/or their parents or legal guardians must sign a written informed consent (and assent when applicable)
- Patients and/or parents who are unable to read at a grade 2 level will be excluded from the patient-reported outcome component of the study, as will non-English speaking patients and/or parents when there is no availability of translated versions in their spoken language . They will not be excluded from all other aspects of the study
Trial locations and contacts
27 sites in 1 country.
27 sites in 1 country
United States27 sites
Alfac Cancer and Blood Disorder Center: Scottish Rite
Atlanta, Georgia, 30342
Children's Hospital Colorado
Aurora, Colorado, 80045
Children's of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Levine Cancer Institute
Charlotte, North Carolina, 28203
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida College of Medicine
Gainesville, Florida, 32610
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children-Indiana University
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
University of Wisconsin
Madison, Wisconsin, 53792
St. Jude Children's Hospital
Memphis, Tennessee, 38105
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Columbia University Irving Medical Center
New York, New York, 10032
Weill Cornell Medical College
New York, New York, 10065
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Children's Hospital of Orange County
Orange, California, 92868
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix CHildren's Hospital
Phoenix, Arizona, 85016
Randall Children's Hospital
Portland, Oregon, 97227
Oregon Health and Science University
Portland, Oregon, 97239
Hasbro Children's Hospital
Providence, Rhode Island, 02903
UCSF Benioff Children's Hospital
San Francisco, California, 94158
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase III Study of Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia in Children
This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
Read the detailed description
This is a prospective, open label, randomized, two-arm, multi-center Phase 3 trial.
Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard therapies. The primary objective is to determine if the proportion of patients with platelet response is significantly greater in patients treated with eltrombopag compared to those treated with standard therapies.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Eltrombopag, Steroids, IVIG, Rho(D) Immune Globulin
- Keywords
- ITP, Immune Thrombocytopenia, Newly Diagnosed Immune Thrombocytopenia, Newly Diagnosed ITP
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Starting dose for eltrombopag will be based on manufacturer recommendations, and drug will be titrated to effect per guidelines.
- Children 1 to 5 years: Initial: 25 mg once daily
- Children ≥6 years and Adolescents: Initial: 50 mg once daily (25 mg once daily for patients of East-Asian ethnicity [e.g., Chinese, Japanese, Korean, Taiwanese])
Dose should be titrated based on platelet response. Maximum dose: 75 mg once daily.
SteroidsDrug
Prednisone/Prednisolone 4mg/kg/day (Max 120 mg/day) x 4 day
IVIGDrug
IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy)
Rho(D) Immune GlobulinDrug
Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Patients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response. Receives: Drug: Eltrombopag |
| Standard first-line therapy | Active comparator | Subjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm.
Standard therapy will be administered as commercially available drug.
Investigator may choose amongst the following:
* IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy)
* Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days
* Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy) Receives: Drug: Steroids; Drug: IVIG; Drug: Rho(D) Immune Globulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients With a Platelet Response
To determine if the percentage of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments
Measured: 12 weeks
Secondary outcomes (11)
Bleeding Score
Poor bleeding score (binary) at 1, 2, 3, 4 weeks, 12 weeks, and 1 year after study enrollment defined as World Health Organization (WHO) Bleeding Scale ≥ 2 or Modified Buchanan Scale ≥ 3
Measured: 1 year
Cumulative Number of Rescue Therapies Required
The percentage of patients who received rescue therapy in the experimental (eltrombopag) arm vs the comparator (standard therapy) arm during the first 12 weeks of treatment
Measured: 12 weeks
Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study
Platelet response (binary), defined as ≥ 3 of 4 weeks with platelets >50 x109/L during weeks 6-12 of therapy, but patient required a rescue treatment during weeks 1-2 of study.
Measured: 12 weeks
Need for Treatment
No further need for treatment (binary) after 12 weeks or 6 months of study
Measured: 6 months
Treatment Response
Treatment response (binary endpoints) at 1 year defined as:
- CR is defined as platelet count >/= 150 x 10^9/L
- Primary Remission at 1 year is defined as CR at 1 year with no second-line agents required and >/= 3 months after discontinuing most recent platelet active medication
- Disease resolution at 1 year is defined as complete response (CR) at 1 year >/= 3 months after discontinuing most recent platelet active medication. May have received a second-line therapy, excluding rituximab or splenectomy.
- Disease stability at 1 year is defined as platelets >/= 50 x 10^9/L but <150 x 10^9/L >/= 3 months after discontinuing most recent platelet active medication.
Measured: 1 year
Number of 2nd Line Therapies
Number of 2nd-line therapies in weeks 13-52
Measured: 52 weeks
Regulatory T-Cells
Absolute change in percentage of CD4+25+Foxp3+ regulatory T cells from baseline at 12 weeks and 1 year
Measured: 1 year
KIT Scores
Change in parent proxy-reported Kids ITP tool (KIT) overall scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment
Measured: 1 year
Hockenberry Fatigue Scale-Parent
Total scale intensity ratings (continuous) from the Hockenberry Fatigue Scale-Parent (FS-P) at 1 week, 4 weeks, 12 weeks, and 1 year
Measured: 1 year
Blood Iron Values
Serum iron, total iron binding capacity (TIBC), transferrin saturation, ferritin, mean corpuscular volume (MCV), and hemoglobin at 12 weeks, 6 months, and 1 year after study enrollment
Measured: 1 year
Safety Evaluations
Safety evaluations as defined by:
- Abnormal liver function tests (LFTs):
ALT ≥ 3 x upper limit of normal (ULN) in patients with normal baseline ALT ≥ 3 x baseline or ≥ 5 x ULN (whichever is lower) in patients with abnormal baseline ALT ≥ 3 x ULN AND bilirubin ≥ 1.5 x ULN (>35% direct)
- Incidence of adverse events
- Incidence of serious adverse events
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2019
- Study start2 May 2019
- Primary completion17 Apr 2024
- Study completion26 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 122 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amanda Grimes, Baylor College of Medicine
- Investigators
- Amanda Grimes, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03939637
- Sponsor's protocol ID
- H-42131 ICON 3
- Other IDs
- CETB115JUS33T, ICON 3
- Status last verified
- Jul 2025
- First submitted
- 25 Apr 2019
- Last updated
- 29 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Shimano KA, Grimes AB, Kaicker S, Shah SJ, Gunn E, Bhat RV, Kochhar M, Rothman JA, Rose MJ, Briones M, Nakano TA, Lebensburger JD, Lambert MP, Fritch Lilla SA, Jesudas R, Lee-Miller CA, Thompson AA, Rifkin-Zenenberg S, Majumdar S, Crary SE, Hege K, Ford JB, Bies JJ, Fort J, Wynn TT, Hsieh L, Ruiz ME, Dinu B, Wong JMW, Kao PC, Kim TO, Arnold SD, Bennett CM, Despotovic JM, Klaassen RJ, Neufeld EJ, Neunert CE, London WB, Grace RF. Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial. JAMA. 2025 Nov 25;334(20):1816-1826. doi: 10.1001/jama.2025.18168.
PubMed 41123939Shimano KA, Grace RF, Despotovic JM, Neufeld EJ, Klaassen RJ, Bennett CM, Ma C, London WB, Neunert C. Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. BMJ Open. 2021 Aug 27;11(8):e044885. doi: 10.1136/bmjopen-2020-044885.
PubMed 34452956
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Study of Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia in Children
- ClinicalTrials.gov ID
- NCT03939637
- Acronym / sponsor ID
- H-42131 ICON 3
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP, Immune Thrombocytopenia, Newly Diagnosed Immune Thrombocytopenia, Newly Diagnosed ITP
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 122 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Boston Children's Hospital, University of California, San Francisco
- Study start
- 2 May 2019 (actual)
- Primary completion
- 17 Apr 2024 (actual)
- Study completion
- 26 Feb 2025 (actual)
- First posted
- 7 May 2019 (actual)
- Results
- Posted 29 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
Detailed description
This is a prospective, open label, randomized, two-arm, multi-center Phase 3 trial.
Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard therapies. The primary objective is to determine if the proportion of patients with platelet response is significantly greater in patients treated with eltrombopag compared to those treated with standard therapies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagPatients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response. |
| Standard first-line therapy | Active comparator | Drug: Steroids, Drug: IVIG, Drug: Rho(D) Immune GlobulinSubjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm. Standard therapy will be administered as commercially available drug. Investigator may choose amongst the following: * IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) * Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days * Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy) |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Age: 1- <18 years
- Newly diagnosed ITP (<3 months from diagnosis (first abnormal platelet count), per international working group definition17)
- Platelets <30 x 10^9/L at screening
- Requires pharmacologic treatment from the perspective of the treating clinician.
- Need to treat is at the discretion of the investigator, but there should be clinical equipoise about the use of eltrombopag vs standard treatment options (patients should not, in the opinion of the investigator, require concomitant therapy at time of enrollment).
Key exclusion criteria (38 listed)
- Severe bleeding: Buchanan Overall Grade 4 or 5 bleeding, or severe bleeding requiring emergent treatment at the discretion of the provider. (e.g., intracranial hemorrhage, pulmonary hemorrhage, bleeding with ongoing need for pRBC transfusion)
- Prior treatment with TPO-RA (eltrombopag or romiplostim)
- Known secondary ITP (due to lupus, CVID, ALPS)
- Known HIV (or history of HIV positivity) or Hepatitis C (screening not required if no clinical suspicion)
- Evans Syndrome: positive direct Coombs with evidence of active hemolysis (elevated lactate dehydrogenase (LDH) or reticulocyte count not attributable to recent treatment or bleeding)
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 |
|---|---|---|
| EltrombopagStarting dose for eltrombopag will be based on manufacturer recommendations, and drug will be titrated to effect per guidelines. * Children 1 to 5 years: Initial: 25 mg once daily * Children ≥6 years and Adolescents: Initial: 50 mg once daily (25 mg once daily for patients of East-Asian ethnicity [e.g., Chinese, Japanese, Korean, Taiwanese]) Dose should be titrated based on platelet response. Maximum dose: 75 mg once daily. | Drug | Eltrombopag |
| SteroidsPrednisone/Prednisolone 4mg/kg/day (Max 120 mg/day) x 4 day | Drug | Standard first-line therapy |
| IVIGIVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) | Drug | Standard first-line therapy |
| Rho(D) Immune GlobulinAnti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy) | Drug | Standard first-line therapy |
Primary outcome
Proportion of Patients With a Platelet Response
Time frame: 12 weeks
To determine if the percentage of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments
11 secondary outcomes
Bleeding Score
Time frame: 1 year
Cumulative Number of Rescue Therapies Required
Time frame: 12 weeks
Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study
Time frame: 12 weeks
Need for Treatment
Time frame: 6 months
Treatment Response
Time frame: 1 year
Number of 2nd Line Therapies
Time frame: 52 weeks
Regulatory T-Cells
Time frame: 1 year
KIT Scores
Time frame: 1 year
Hockenberry Fatigue Scale-Parent
Time frame: 1 year
Blood Iron Values
Time frame: 1 year
Safety Evaluations
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41123939Shimano KA, Grimes AB, Kaicker S, Shah SJ, Gunn E, Bhat RV, Kochhar M, Rothman JA, Rose MJ, Briones M, Nakano TA, Lebensburger JD, Lambert MP, Fritch Lilla SA, Jesudas R, Lee-Miller CA, Thompson AA, Rifkin-Zenenberg S, Majumdar S, Crary SE, Hege K, Ford JB, Bies JJ, Fort J, Wynn TT, Hsieh L, Ruiz ME, Dinu B, Wong JMW, Kao PC, Kim TO, Arnold SD, Bennett CM, Despotovic JM, Klaassen RJ, Neufeld EJ, Neunert CE, London WB, Grace RF. Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial. JAMA. 2025 Nov 25;334(20):1816-1826. doi: 10.1001/jama.2025.18168.
- Derived from this study: PubMed 34452956Shimano KA, Grace RF, Despotovic JM, Neufeld EJ, Klaassen RJ, Bennett CM, Ma C, London WB, Neunert C. Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. BMJ Open. 2021 Aug 27;11(8):e044885. doi: 10.1136/bmjopen-2020-044885.
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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.
Amanda Grimes, MD principal investigator
Baylor College of Medicine
27 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eltrombopag, Steroids and IVIG in people with Immune Thrombocytopenia, run by Baylor College of Medicine at 27 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 13 inclusion and 38 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: finished, as of the sponsor's last update on 29 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Proportion of Patients With a Platelet Response (time frame: 12 weeks). 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 NCT03939637, which also lists 2 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 29 Jul 2025.