A Study of Romiplostim to Prevent Low Platelet Counts in Children and Young Adults Receiving Chemotherapy for Solid Tumors

NCT04671901 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 20-467
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 7

Brief Summary

The purpose of this study is to find out whether romiplostim can help prevent low platelet counts caused by N8 or EFT chemotherapy, reduce the number of platelet transfusions required during chemotherapy, and prevent treatment delays due to low platelet counts.

Conditions

Solid Tumor; Solid Tumor, Childhood; Solid Carcinoma

Keywords

romiplostim; primary solid tumor; Solid tumor; Solid tumor, childhood; Childhood solid tumor; Pediatric Solid Tumors; 20-467; Memorial Sloan Kettering Cancer Center

Outcome Measures

Primary Outcomes (1)

• Total Number of Platelet Transfusions During the Studied Portions of the EFT or D9803 Cycles

  The primary purpose of this study is to evaluate whether romiplostim administration can decrease the total number of platelet transfusions required during the treatment courses of EFT or D9803 when compared to the benchmark rate.

  up to 6 months

Study Arms (1)

Pediatric Participants

EXPERIMENTAL

Male and female patients aged 1-21 years with a primary solid tumor undergoing treatment with the pre-defined chemotherapy regimens of EFT, MAP or D9803.

Drug: Romiplostim

Interventions

Romiplostim DRUG

Participants will receive weekly doses of romiplostim, beginning with cycle 4. The initial romiplostim dose will be 10 mcg/kg, and subsequent doses w ill vary based on chemotherapy regimen for a target of platelet count \> 75,000- 200,000/mcL. Participants will continue romiplostim until completion of MAP or D9803, as defined above. Maximum romiplostim dose is 10 mcg/kg. Participants will be followed until 6 months after the last dose of romiplostim.

Pediatric Participants

Eligibility Criteria

• Age: 1 Year - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Documented diagnosis of a primary solid tumor. Patients must have histological verification of malignancy at MSKCC.
• Male and female patients aged 1-21 years with a primary solid tumor undergoing treatment with the pre-defined chemotherapy regimens of EFT, MAP, D9803. Prior to enrollment patient could have been undergoing induction therapy with a similarly myelosuppressive regimen as long as they will be continuing with EFT, MAP, D9803 at the time of study enrollment.
• Patients undergoing treatment with MAP chemotherapy w ho have had ≥ 1 platelet transfusion during induction stage of treatment.
• Total Bilirubin (sum of conjugated + unconjugated) ≤ 3 times institutional upper limit of normal (ULN) for age and ALT/AST ≤ 3 times institutional ULN for age.
• Normal cardiac function:
• Shortening fraction greater than or equal to 28% by echocardiogram OR Left ventricular ejection fraction (LVEF) greater than or equal to 50% on technetium- 99m pertechnetate radionuclide cineangiography (MUGA) or echocardiogram.
• Screening ECG with corrected QT (QTc) interval of \< 470 msec.
• Timing of cardiac assessment: We will utilize the most recent EKG/ECHO when assessing cardiac function. See section 9.0 for additional details.
• Adequate renal function, defined as an estimated Creatinine Clearance or GFR \>40ml/min or an normal creatine for age (see below)
• Serum Creatinine by age:
• Age (years) \<6: Maximum Serum Creatinine (mg/dL), Male 0.8, Female 0.8 Age (years) 6 to \<10: Maximum Serum Creatinine (mg/dL), Male 1, Female 1 Age (years) 10 to \<13: Maximum Serum Creatinine (mg/dL), Male 1.2, Female 1.2 Age (years) 13 to \<16: Maximum Serum Creatinine (mg/dL), Male 1.5, Female 1.4 Age (years) \>16: Maximum Serum Creatinine (mg/dL), Male 1.7, Female 1.4
• These threshold creatine values were derived from the Scwartz formula estimating GFR, utilizing child length and statured published by the CDC.

You may not qualify if:

• Patients with history of hematologic malignancies or allogenic/autogenic stem cell transplant.
• Patients with a currently known predisposition to a myeloid stem cell disorder, myeloid leukemia, and/or bone marrow failure syndrome including, but not limited to:
• Aplastic anemia
• Ataxia telangiectasia
• Bloom syndrome
• Congenital amegakaryocytic thrombocytopenia
• Cyclic neutropenia
• Diamond Blackfan anemia
• Dyskeratosis congenita
• Familial AML/MDS syndromes (including ANKRD26, CEBPA, DDX41, ETV6, GATA2, RUNX1, SRP72)
• Fanconi anemia
• Kostmann disease
• Li-Fraumeni syndrome
• Neurofibromatosis
• Nijmegen breakage syndrome

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead

Study Sites (7)

Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

Basking Ridge, New Jersey, 07920, United States

Location

Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

Middletown, New Jersey, 07748, United States

Location

Memorial Sloan Kettering Bergen (Limited Protocol Activities)

Montvale, New Jersey, 07645, United States

Location

Memorial Sloan Kettering Cancer Center @ Suffolk - Commack (Limited Protocol Activities)

Commack, New York, 11725, United States

Location

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, 10604, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Memorial Sloan Kettering Nassau (Limited Protocol Activities)

Uniondale, New York, 11553, United States

Location

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Interventions

romiplostim

Results Point of Contact

• Title: Dr. Michael Ortiz MD
• Organization: Memorial Sloan Kettering Cancer Center

ortizm2@mskcc.org

212-639-6057

Study Officials

• Michael Ortiz, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2020
• First Posted: December 17, 2020
• Study Start: December 10, 2020
• Primary Completion: March 29, 2023
• Study Completion: March 29, 2023
• Last Updated: May 28, 2025
• Results First Posted: November 18, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will share

Locations

US (7)