Revolade Tablets Specified Drug-use Survey

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06287268
• Other study ID #: CETB115G1401
• Status: Not yet recruiting
• Start date: April 30, 2024
• Est. completion date: October 31, 2028

Study information

• Verified date: February 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: +81337978748
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 10
• Est. completion date: October 31, 2028
• Est. primary completion date: October 31, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

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)

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Intervention

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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence of serious adverse events	Occurrence of serious adverse events to be provided	Up to 1 year
Secondary	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
Secondary	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
Secondary	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
Secondary	Hematologic response status	Hematologic response status to be provided	Up to 1 year
Secondary	Change over time from baseline in platelet count	Change over time from baseline in platelet count to be provided	Baseline, 1 year
Secondary	Change over time from baseline in hemoglobin	Change over time from baseline in hemoglobin to be provided	Baseline, 1 year
Secondary	Change over time from baseline in neutrophil count	Change over time from baseline in neutrophil count to be provided	Baseline, 1 year
Secondary	Change over time from baseline in reticulocyte count	Change over time from baseline in reticulocyte count to be provided	Baseline, 1 year
Secondary	Change over time from baseline in transfusion dependence	Change over time from baseline in transfusion dependence to be provided	Baseline, 1 year
Secondary	Change over time from baseline in transfusion volume	Change over time from baseline in transfusion volume to be provided	Baseline, 1 year