NCT06045052

Brief Summary

An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
8

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2020

Geographic Reach
1 country

1 active site

Status
completed

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 Start

First participant enrolled

December 2, 2020

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 19, 2023

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 29, 2023

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

September 13, 2023

Completed
8 days until next milestone

First Posted

Study publicly available on registry

September 21, 2023

Completed
Last Updated

September 21, 2023

Status Verified

September 1, 2023

Enrollment Period

2.5 years

First QC Date

September 13, 2023

Last Update Submit

September 20, 2023

Conditions

Fanconi Anemia

Keywords

Fanconi Anemia

Outcome Measures

Primary Outcomes (1)

  • Assessing the efficacy of eltrombopag on hematopoiesis

    An analysis of the proportion of patients with complete response and/or partial response after 6 months of treatment in the absence of transfusions or rescue therapy will be performed.

    6 months

Secondary Outcomes (3)

  • Evaluation of the incidence of clonal evolution

    6 and 12 months.

  • Identification and tracking of adverse reactions associated with eltrombopag treatment in patients with Fanconi anemia graded

    3, 6, 9 ans 12 months.

  • Determination of the proportion of grafted cells

    12 months

Study Arms (1)

Eltrombopag

EXPERIMENTAL

Eltrombopag will be administered orally once daily for 24 weeks (6 months) and the dose will be adjusted according to race, age and weight. Patients who achieve at least partial remission may continue treatment for up to one year.

Drug: Eltrombopag

Interventions

EltrombopagDRUG

Eltrombopag tablets by mouth once daily for 24 weeks (6 months) with the dose depending on race, age and weight.

Also known as: Revolade®
Eltrombopag

Eligibility Criteria

Age4 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patients with a diagnosis of Fanconi anemia. Diagnosis will be defined by the presence of biallelic mutations in genes known to cause the disease and/or by positive chromosome breakage analysis of lymphocytes and/or skin fibroblasts (for cases of mosaicism).
  • Age from 4 to ≤ 17 years.
  • Lansky index \> 80%.
  • Informed consent provided in accordance with current legislation.
  • Presenting with one or more of one of the following clinically significant cytopenias:

You may not qualify if:

  • Patients with HLA-matched related donor or unrelated donor with a 12/12 match who is immediately available.
  • Baseline creatinine greater than 2.5 times the upper limit of normality.
  • GOT/AST or GPT/ALT more than three times the upper limit of normality. Direct bilirubin greater than 1.5 times the upper limit of normality.
  • Patients who are already receiving treatment with some drug for bone marrow failure may be included as long as the dose administered remains stable for at least two months. In the event that such treatment requires an increase in dose during the study, the patient must withdraw from the trial. Patients who have already started Revolade® treatment in the previous two months may also be included, and the blood counts and baseline bone marrow studies performed at the start of treatment will be used.
  • Women of postpubertal age and therefore at risk of pregnancy should have a negative serum or urine pregnancy test at the screening visit and agree to use a contraceptive method throughout the treatment period and for at least one month after.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital Infantil Universitario Niño Jesús (HIUNJ)

Madrid, 28009, Spain

Location

Related Publications (1)

  • Iriondo J, Zubicaray J, Rio P, Catala A, Solsona M, Sanz A, Gomez A, Sebastian E, de la Cruz A, Galan A, Navarro S, Larcher L, de Andoin NG, Uriz JJ, Vagace JM, Gonzalez de Pablo J, Pujol MR, Nicoletti E, Surralles J, Martin-Prado S, Schwartz JD, Soulier J, Bueren JA, Sevilla J. Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV. Eur J Haematol. 2025 Oct;115(4):403-412. doi: 10.1111/ejh.70007. Epub 2025 Jul 16.

    PMID: 40665878DERIVED

MeSH Terms

Conditions

Fanconi Anemia

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Julián Sevilla Navarro, MD, PhD

    Hospital Infantil Universitario Niño Jesús (HIUNJ)

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Dr. Julián Sevilla Navarro, MD, PhD

Study Record Dates

First Submitted

September 13, 2023

First Posted

September 21, 2023

Study Start

December 2, 2020

Primary Completion

June 19, 2023

Study Completion

August 29, 2023

Last Updated

September 21, 2023

Record last verified: 2023-09

Data Sharing

IPD Sharing
Will share

Locations

ES(1)