Revolade Tablets Specified Drug-use Survey

Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)

This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).

Study Overview

• Status: Recruiting
• Conditions: Aplastic Anemia
• Intervention / Treatment: Other: eltrombopag

Detailed Description

The objective of this survey is to confirm the safety and efficacy of eltrombopag in ATG treatment naive pediatric patients with AA. Eltrombopag should be administered according to the dosage and administration specified in the latest version of the package insert. The observation period is 1 year (364 days) from the start of treatment with this product, regardless of whether treatment with eltrombopag is continued or not. However, if hematopoietic stem cell transplantation is performed within 1 year after the start of treatment with eltrombopag, the observation period shall be until the date of hematopoietic stem cell transplantation.

• Study Type: Observational
• Enrollment (Estimated): 10

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +81337978748; Email: novartis.email@novartis.com

Study Locations

• Japan
  • Aichi-ken
    • Nagoya, Aichi-ken, Japan, 453-8511
      • Recruiting
      • Novartis Investigative Site
    • Nagoya, Aichi-ken, Japan, 4668560
      • Recruiting
      • Novartis Investigative Site
    • Toyoake, Aichi-ken, Japan, 4701192
      • Recruiting
      • Novartis Investigative Site
  • Chiba
    • Chiba, Chiba, Japan, 260 8677
      • Recruiting
      • Novartis Investigative Site
  • Hyōgo
    • Amagasaki, Hyōgo, Japan, 660 8550
      • Recruiting
      • Novartis Investigative Site
    • Kobe, Hyōgo, Japan, 6500047
      • Recruiting
      • Novartis Investigative Site
  • Nagano
    • Matsumoto, Nagano, Japan, 3908621
      • Recruiting
      • Novartis Investigative Site
  • Okinawa
    • Shimajiri-Gun, Okinawa, Japan, 901-1303
      • Recruiting
      • Novartis Investigative Site
  • Osaka
    • Izumi, Osaka, Japan, 5941101
      • Recruiting
      • Novartis Investigative Site
    • Osaka, Osaka, Japan, 5340021
      • Recruiting
      • Novartis Investigative Site
    • Sakai, Osaka, Japan, 590-0197
      • Recruiting
      • Novartis Investigative Site
  • Saitama
    • Saitama, Saitama, Japan, 3308777
      • Recruiting
      • Novartis Investigative Site
  • Shiga
    • Ohtsu, Shiga, Japan, 5202192
      • Recruiting
      • Novartis Investigative Site
  • Tokyo
    • Chuo Ku, Tokyo, Japan, 1048560
      • Recruiting
      • Novartis Investigative Site
    • Setagaya-ku, Tokyo, Japan, 1578535
      • Recruiting
      • Novartis Investigative Site
    • Ōta-ku, Tokyo, Japan, 143 8541
      • Recruiting
      • Novartis Investigative Site
  • Wakayama
    • Wakayama, Wakayama, Japan, 641-8510
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The survey will be conducted in pediatric patients with Aplastic anemia (AA) who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA."

Description

Inclusion Criteria:

• Patients whose legally acceptable representative has given written consent for cooperation in this survey prior to enrollment in this survey
• Patients aged ≥ 6 years and < 18 years at the start of treatment with eltrombopag
• Pediatric patients with AA who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA"

Exclusion Criteria:

• Patients who have received ATG without concomitant use of eltrombopag
• Patients with congenital AA
• Patients with suspected or confirmed diagnosis of myelodysplastic syndrome (MDS) at the start of treatment with eltrombopag
• Patients who have received any drug products containing the same ingredient as eltrombopag (including investigational products)

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
eltrombopag; ATG treatment naïve pediatric patients with AA in routine clinical practice	Other: eltrombopag; This is an observational study. There is no treatment allocation. After confirming that patients are fulfilling the eligibility criteria, patients will be registered in this survey.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of serious adverse events	Occurrence of serious adverse events to be provided	Up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation	Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation to be provided	Up to 1 Year
Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)	Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)	Up to 1 year
Occurrence of clonal evolution	Occurrence of clonal evolution [cytogenetic abnormalities, myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), and paroxysmal nocturnal hemoglobinuria (PNH)]	Up to 1 year
Hematologic response status	Hematologic response status to be provided	Up to 1 year
Change over time from baseline in platelet count	Change over time from baseline in platelet count to be provided	Baseline, 1 year
Change over time from baseline in hemoglobin	Change over time from baseline in hemoglobin to be provided	Baseline, 1 year
Change over time from baseline in neutrophil count	Change over time from baseline in neutrophil count to be provided	Baseline, 1 year
Change over time from baseline in reticulocyte count	Change over time from baseline in reticulocyte count to be provided	Baseline, 1 year
Change over time from baseline in transfusion dependence	Change over time from baseline in transfusion dependence to be provided	Baseline, 1 year
Change over time from baseline in transfusion volume	Change over time from baseline in transfusion volume to be provided	Baseline, 1 year

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): July 17, 2024
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028

Study Registration Dates

• First Submitted: February 22, 2024
• First Submitted That Met QC Criteria: February 22, 2024
• First Posted (Actual): March 1, 2024

Study Record Updates

• Last Update Posted (Actual): February 19, 2026
• Last Update Submitted That Met QC Criteria: February 18, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: AA; NIS; eltrombopag; Pediatric Aplastic Anemia; Revolade Tablets; Drug-use Survey
• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Hemic and Lymphatic Diseases; Anemia, Aplastic; eltrombopag
• Other Study ID Numbers: CETB115G1401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO