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

NCT06239389 | Completed Phase 2

• 1 other identifier: NIBD/IRB-236/21-2021.
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Beta-Thalassemia

Keywords

thalidomide,; hydroxyurea; hemoglobin F inducers; beta thalassemia

Outcome Measures

Primary Outcomes (15)

• 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

• 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

Study Arms (2)

hydroxyurea

ACTIVE COMPARATOR

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

EXPERIMENTAL

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

Interventions

Thalidomide 100mg DRUG

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.

Also known as: thalido

hydroxyurea

Hydroxyurea DRUG

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.

Also known as: Hydrea

Thalidomide

Eligibility Criteria

• Age: 10 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Institute of Blood and Marrow Transplant (NIBMT), Pakistan: lead

Study Sites (1)

National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi, Sindh, 75300, Pakistan

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Thalidomide; Hydroxyurea

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)

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

Study Officials

• Safia Mehmood khan, MPHIL

  National institute if blood diseases and bone marrow transplantation

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.

Study Record Dates

• First Submitted: December 23, 2023
• First Posted: February 2, 2024
• Study Start: September 1, 2021
• Primary Completion: December 1, 2021
• Study Completion: March 3, 2022
• Last Updated: March 12, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)