Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients

NCT06153784 | Completed Phase 2 DMC

• 1 other identifier: CH-0428
• Study Type: interventional
• Target: 603
• Locations: 1 country
• Sites: 1

Brief Summary

Objectives Primary objective:

• To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients. Secondary objective:
• To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy. A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide was started. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during, and end of the study. The assessment of treatment outcomes was conducted at the 1-year, 2-year, and 3-year follow-up points during the combination therapy period, categorizing patients as either "good responders," "responders," or "non-responders."

Conditions

Thalassemia, Beta

Outcome Measures

Primary Outcomes (3)

• Response at different time intervals

  Frequency of good responder, responder, and non-responder.

  1-3 years on combination therapy

• Change in laboratory levels

  Mean changes in hemoglobin, platelets, leukocytes, urea, creatinine, and ferritin level from baseline

  1-3 years on combination therapy

• Change in the liver and spleen size

  Mean changes in the liver and spleen size from baseline

  1-3 years on combination therapy

Secondary Outcomes (1)

• XmnI polymorphism

  1-3 years on combination therapy

Study Arms (1)

Combination of hydroxyurea and thalidomide

EXPERIMENTAL

Hydroxyurea was continued at a dose of 10-20 mg/kg/day for 6 months and then thalidomide was added orally at a dose of 2-5mg/kg/day.

Drug: Hydroxyurea and Thalidomide

Interventions

Hydroxyurea and Thalidomide DRUG

Evaluation of hydroxyurea and thalidomide combination use in beta-thalassemia patients

Combination of hydroxyurea and thalidomide

Eligibility Criteria

• Age: 2 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with clinical and genetic diagnoses of β-thalassemia major and intermedia
• Patients who showed partial response or a decline in response to hydroxyurea
• Patients who are not candidates for the bone marrow transplant procedure

You may not qualify if:

• Married Patients
• Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
• Patients allergic to the drug ingredients
• Patients with mental disorders
• Patients who are enrolled in other clinical trials
• Patients with a history of venous or arterial thrombosis

Sponsors & Collaborators

• Children's Hospital Karachi: lead

Study Sites (1)

Children's Hospital Karachi

Karachi, Sindh, Pakistan

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Hydroxyurea; Thalidomide

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Saqib H Ansari, Phd

  Children's Hospital Karachi Sindh, Pakistan

  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

Study Record Dates

• First Submitted: November 23, 2023
• First Posted: December 1, 2023
• Study Start: July 7, 2020
• Primary Completion: July 30, 2023
• Study Completion: July 30, 2023
• Last Updated: December 1, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)