NCT06287268

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).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
30mo left

Started Jul 2024

Longer than P75 for all trials

Geographic Reach
1 country

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress42%
Jul 2024Oct 2028

First Submitted

Initial submission to the registry

February 22, 2024

Completed
8 days until next milestone

First Posted

Study publicly available on registry

March 1, 2024

Completed
5 months until next milestone

Study Start

First participant enrolled

July 17, 2024

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2028

Last Updated

February 19, 2026

Status Verified

February 1, 2026

Enrollment Period

4.3 years

First QC Date

February 22, 2024

Last Update Submit

February 18, 2026

Conditions

Keywords

NISPediatric Aplastic AnemiaAARevolade TabletseltrombopagDrug-use Survey

Outcome Measures

Primary Outcomes (1)

  • Occurrence of serious adverse events

    Occurrence of serious adverse events to be provided

    Up to 1 year

Secondary Outcomes (10)

  • Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation

    Up to 1 Year

  • 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

    Up to 1 year

  • Hematologic response status

    Up to 1 year

  • Change over time from baseline in platelet count

    Baseline, 1 year

  • +5 more secondary outcomes

Study Arms (1)

eltrombopag

ATG treatment naïve pediatric patients with AA in routine clinical practice

Other: eltrombopag

Interventions

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.

eltrombopag

Eligibility Criteria

Age6 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-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."

You may qualify if:

  • 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"

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Novartis Investigative Site

Nagoya, Aichi-ken, 453-8511, Japan

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 4668560, Japan

RECRUITING

Novartis Investigative Site

Toyoake, Aichi-ken, 4701192, Japan

RECRUITING

Novartis Investigative Site

Chiba, Chiba, 260 8677, Japan

RECRUITING

Novartis Investigative Site

Amagasaki, Hyōgo, 660 8550, Japan

RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, 6500047, Japan

RECRUITING

Novartis Investigative Site

Matsumoto, Nagano, 3908621, Japan

RECRUITING

Novartis Investigative Site

Shimajiri-Gun, Okinawa, 901-1303, Japan

RECRUITING

Novartis Investigative Site

Izumi, Osaka, 5941101, Japan

RECRUITING

Novartis Investigative Site

Osaka, Osaka, 5340021, Japan

RECRUITING

Novartis Investigative Site

Sakai, Osaka, 590-0197, Japan

RECRUITING

Novartis Investigative Site

Saitama, Saitama, 3308777, Japan

RECRUITING

Novartis Investigative Site

Ohtsu, Shiga, 5202192, Japan

RECRUITING

Novartis Investigative Site

Chuo Ku, Tokyo, 1048560, Japan

RECRUITING

Novartis Investigative Site

Ōta-ku, Tokyo, 143 8541, Japan

RECRUITING

Novartis Investigative Site

Setagaya-ku, Tokyo, 1578535, Japan

RECRUITING

Novartis Investigative Site

Wakayama, Wakayama, 641-8510, Japan

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

Novartis Pharmaceuticals

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 22, 2024

First Posted

March 1, 2024

Study Start

July 17, 2024

Primary Completion (Estimated)

October 31, 2028

Study Completion (Estimated)

October 31, 2028

Last Updated

February 19, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations