Outcomes in Bone Marrow Aplasia.

NCT06493981 | Not Yet Recruiting Phase 3

• 1 other identifier: Noha Mahmoud Muhammed
• Study Type: interventional
• Target: 3
• Locations: 0 countries
• Sites: N/A

Brief Summary

Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and fatty infiltration of the bone marrow space. Underlying mechanisms include immune-mediated attack, telomere defects, and inherent HSC compartment insufficiency. These events may occur individually or in concert, mostly involving effector T cells Historical treatment has included the use of high-dose chemotherapy and allogeneic stem cell transplantation as well as lymphotoxic immunosuppressive therapy (IST) Thrombopoietin (TPO) regulates platelet production, maturation, and release through binding of c-mpl on megakaryocytes.

Conditions

Aplastic Anemia Idiopathic

Outcome Measures

Primary Outcomes (2)

• Determine hematological response after 6 months

  Change in CBC elements after 6 months of treatment

  6 months to 5 years

• Determine overall survival rate.

  Number if years estimated to survive after treatment

  5 years

Secondary Outcomes (5)

• Rate of relapse in patients deemed responsers at 6 months.

  Around 1 year

• Clonal evolution to myeloid malignancy or new chromosomal abnormality

  Around 2 years

• Change in serum iron and ferritin over time of treatment

  From 6 months to 5 years

• Eltrombopag efficacy in increasing platelet count

  After6 months

• Adverse effects raelated to treatment

  Within 2 years

Study Arms (1)

Cases of aplastic anemia recieving Eltrombopag

EXPERIMENTAL

Newely diagnosed bone marrow aplasia starting treatment with Eltrombopag in adose of 50-150mg / day

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

Treatment with eltrombopag in a dose of (50-150mg/d)

Also known as: Revolade, Versapenia

Cases of aplastic anemia recieving Eltrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \- Age \> 18. Newely diagnosed bone marrow aplasia Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. Patients started CSA plus Eltrombopag therapy Normal cardiac, hepatic \& renal functions

You may not qualify if:

• Hypersensitivity or contraindications to eltrombopag. Cardiovascular, pulmonary, hepatic, or renal diseases. History of malignancy. Pregnant, breastfeeding. Inherited bone marrow aplasia. Secondry bone marrow aplasia Previous thromboembolic events. Previous malignancies either solid or hematologic.

Sponsors & Collaborators

• Assiut University: lead

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Long term outcomes of eltrombopag in patients with bone marrow aplasia, Assiut university hospital insight.

Study Record Dates

• First Submitted: July 2, 2024
• First Posted: July 10, 2024
• Study Start: July 1, 2024
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): July 1, 2030
• Last Updated: July 10, 2024

Record last verified: 2024-06