Clinical trial NCT06287268: Revolade Tablets Specified Drug-use Survey
Official title: Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Aplastic Anemia
Keywords: NIS
Pediatric Aplastic Anemia
AA
Revolade Tablets
eltrombopag
Drug-use Survey
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-03-01
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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."

Brief 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).

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Occurrence of serious adverse events
timeFrame: Up to 1 year
Secondary outcomes:
measure: Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation
timeFrame: Up to 1 Year
measure: Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)
timeFrame: Up to 1 year
measure: Occurrence of clonal evolution
timeFrame: Up to 1 year
measure: Hematologic response status
timeFrame: Up to 1 year
measure: Change over time from baseline in platelet count
timeFrame: Baseline, 1 year
measure: Change over time from baseline in hemoglobin
timeFrame: Baseline, 1 year
measure: Change over time from baseline in neutrophil count
timeFrame: Baseline, 1 year
measure: Change over time from baseline in reticulocyte count
timeFrame: Baseline, 1 year
measure: Change over time from baseline in transfusion dependence
timeFrame: Baseline, 1 year
measure: Change over time from baseline in transfusion volume
timeFrame: Baseline, 1 year
Other outcomes:
Locations:
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Toyoake city
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Amagasaki city
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Kobe
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Matsumoto
country: Japan
facility: Novartis Investigative Site
state: Nagano
city: Shimajiri-Gun
country: Japan
facility: Novartis Investigative Site
state: Okinawa
city: Izumi-city
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Osaka Sayama
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Ohtsu
country: Japan
facility: Novartis Investigative Site
state: Shiga
city: Chuo ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Ota-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Setagaya-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Osaka
country: Japan
facility: Novartis Investigative Site
city: Saitama
country: Japan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: