Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.

The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study.

The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency.

Test were done at baseline and after completion of study means after 06 months.

Study Overview

• Status: Completed
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Drug: Thalidomide 100mg; Drug: Hydroxyurea

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• Pakistan
  • Sindh
    • Karachi, Sindh, Pakistan, 75300
      • National Institute of Blood Diseases and Bone Marrow Transplantation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Know case of beta thalassemia age between 10-30 years

Exclusion Criteria:

• Patients on any other haemoglobin F inducer or erythropoietin.
• Patients with co-morbidities like cardiopulmonary and neurological disease
• Pregnant, lactating women.
• Patient did not willing to take contraceptive measures.
• Participants with history of thrombosis.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: hydroxyurea; Hydroxyurea is available in oral dosage form which is capsule. frequency: once a day duration: at every 24 hours dose: 15/mg/kg/day.	Drug: Thalidomide 100mg; Thalidomide is available in capsulated form in the strength of 100 mg and 50 mg. It was given according to the weight of the patient. Due to somnolence, it was advised to give at night only.; Other Names: thalido
Experimental: Thalidomide; Thalidomide is available in oral dosage form which is capsule. frequency: once a day at night duration: at every 24 hours dose:50 mg / day (in patients >10-13 years) while the adult dose was 100 mg /day (age >13 Years)	Drug: Hydroxyurea; Hydroxyurea is available in capsulated form under the brand of hydrea. Its available strength is 500 mg . Dosing depends upon weight of patient, that is 15mg/kg/day. so patients unable to take full capsule due to low weight, was advised to take hydroxyurea syrup.; Other Names: Hydrea

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
comparison of Lactate dehydrogenase baseline and after the completion of study.	Lactate dehydrogenase	06 months
comparison of tranfusion frequency baseline and after the completion of study.	Transfusion interval	06 months
comparison of hemoglobin	Hemoglobin	06 months
comparison of red blood cells	Red blood cells	06 months
comparison of Mean corpuscular volume	Mean corpuscular volume	06 months
comparison of Mean Corpuscular Hemoglobin	Mean Corpuscular Hemoglobin	06 months
comparison of Mean Corpuscular Hemoglobin concentration	Mean Corpuscular Hemoglobin concentration	06 months
comparison of Indirect bilirubin	Indirect bilirubin	06 months
comparison of Total Bilirubin	Total Bilirubin	06 months
comparison of Serum glutamic pyruvic transaminase	Serum glutamic pyruvic transaminase	06 months
comparison of glutamic-oxaloacetic transaminase	glutamic-oxaloacetic transaminase	06 months
comparison of white blood cells	white blood cells	06 months
comparison of platelets	Platelets	06 months
comparison of urea	Urea	06 months
comparison of creatinine	Creatinine	06 months

Collaborators and Investigators

• Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
• Investigators: Principal Investigator: Safia Mehmood khan, MPHIL, National institute if blood diseases and bone marrow transplantation

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2021
• Primary Completion (Actual): December 1, 2021
• Study Completion (Actual): March 3, 2022

Study Registration Dates

• First Submitted: December 23, 2023
• First Submitted That Met QC Criteria: January 25, 2024
• First Posted (Actual): February 2, 2024

Study Record Updates

• Last Update Posted (Actual): March 12, 2024
• Last Update Submitted That Met QC Criteria: March 7, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: hydroxyurea; beta thalassemia; thalidomide,; hemoglobin F inducers
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Nucleic Acid Synthesis Inhibitors; Enzyme Inhibitors; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Anti-Bacterial Agents; Leprostatic Agents; Antisickling Agents; Thalidomide; Hydroxyurea
• Other Study ID Numbers: NIBD/IRB-236/21-2021.

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No