Clinical Experience of Thalidomide in Thalassemic Patients

NCT05132270 | Completed Phase 2 DMC

• 1 other identifier: CH-0420
• Study Type: interventional
• Target: 135
• 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. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study. Sample Size and Population This study included 135 Beta-thalassemia patients.

Conditions

Thalassemia, Beta

Outcome Measures

Primary Outcomes (5)

• Change in transfusion requirements

  Change in transfusion needs (measured as blood volume in ml) per month from baseline

  6 months on combination therapy

• Change in Hemoglobin levels

  Change in hemoglobin level of \>1g/dl from baseline

  6 months on combination therapy

• Change in the liver function test

  Change in SGPT in U/l from baseline

  6 months on combination therapy

• Change in liver function test

  Change in total, direct, and indirect bilirubin in mg/dl from baseline

  6 months on combination therapy

• Change in Kidney Function test

  Change in urea and creatinine levels in mg/dl from baseline

  6 months on combination therapy

Secondary Outcomes (2)

• Change in Spleen size

  6 months on combination therapy

• Change in liver size

  6 months 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 for 6 months.

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+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with clinical and genetic diagnosis of β-thalassemia major and intermedia
• Patients who showed partial response or a decline in response to hydroxyurea
• Patients who are not the 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

Related Publications (1)

• Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.

  PMID: 35477175 DERIVED

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 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: October 21, 2021
• First Posted: November 24, 2021
• Study Start: January 1, 2020
• Primary Completion: December 31, 2020
• Study Completion: January 31, 2021
• Last Updated: November 24, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)