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Clinical Experience of Thalidomide in Thalassemic Patients

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ClinicalTrials.gov Identifier: NCT05132270
Recruitment Status : Completed
First Posted : November 24, 2021
Last Update Posted : November 24, 2021
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Karachi

Study Description
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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.


Condition or disease Intervention/treatment Phase
Thalassemia, Beta Drug: hydroxyurea and thalidomide Phase 2 Phase 3

Detailed Description:

The purpose of the study was to evaluate the efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. The participants were evaluated on the basis of eligibility criteria, consent was obtained and baseline investigations were performed on the screening visit. Participants were continued on Hydroxyurea (10-20mg/kg/day) for the first six months and then for the next six months thalidomide (2-5 mg/kg/day) was added to the intervention. Aspirin was also added (2-4mg/kg/day).

Efficacy:

Maintenance or rise in Hemoglobin (Hb) levels and changes in transfusion frequency before and after the use of combination therapy was used to evaluate the efficacy of the combination therapy. Good responders were the patients who were on transfusion and went off-transfusion after the combination therapy or individuals who were already off transfusion and after combination therapy demonstrated an increase in Hb of at least 1gm/dl. Responders were those who remained on transfusion on combination therapy but displayed a 50% reduction in blood requirements. Whereas, non-responders, were individuals who were off-transfusion before combination therapy and had improvement in Hb of <1gm/dl, or those who remained on transfusion during combination therapy and did not experience transfusion reduction of at least 50%.

Safety:

Safety of the drug was evaluated on the basis of the following parameters and intervention was discontinued or put on hold if:

  • Creatinine >1.1mg/dl, Urea >43mg/dl),
  • Liver function (SGPT >35mg/L)
  • Absolute Neutrophil counts<2*109/L
  • Platelets < 100*109/L
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 135 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Combination of Hydroxyurea and Low-dose Thalidomide on Hemoglobin Synthesis in Thalassemia Patients
Actual Study Start Date : January 1, 2020
Actual Primary Completion Date : December 31, 2020
Actual Study Completion Date : January 31, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Combination of hydroxyurea and thalidomide
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
Evaluation of hydroxyurea and thalidomide combination use in beta-thalassemia patients


Outcome Measures
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Primary Outcome Measures :
  1. Change in transfusion requirements [ Time Frame: 6 months on combination therapy ]
    Change in transfusion needs (measured as blood volume in ml) per month from baseline

  2. Change in Hemoglobin levels [ Time Frame: 6 months on combination therapy ]
    Change in hemoglobin level of >1g/dl from baseline

  3. Change in the liver function test [ Time Frame: 6 months on combination therapy ]
    Change in SGPT in U/l from baseline

  4. Change in liver function test [ Time Frame: 6 months on combination therapy ]
    Change in total, direct, and indirect bilirubin in mg/dl from baseline

  5. Change in Kidney Function test [ Time Frame: 6 months on combination therapy ]
    Change in urea and creatinine levels in mg/dl from baseline


Secondary Outcome Measures :
  1. Change in Spleen size [ Time Frame: 6 months on combination therapy ]
    Change in size of spleen (in centimeters) from baseline

  2. Change in liver size [ Time Frame: 6 months on combination therapy ]
    Change in size of liver (in centimeters) from baseline


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  1. Married Patients
  2. Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
  3. Patients allergic to the drug ingredients
  4. Patients with mental disorders
  5. Patients who are enrolled in other clinical trials
  6. Patients with a history of venous or arterial thrombosis
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05132270


Locations
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Pakistan
Children's Hospital Karachi
Karachi, Sindh, Pakistan
Sponsors and Collaborators
Children's Hospital Karachi
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Children's Hospital Karachi
ClinicalTrials.gov Identifier: NCT05132270    
Other Study ID Numbers: CH-0420
First Posted: November 24, 2021    Key Record Dates
Last Update Posted: November 24, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Thalidomide
Hydroxyurea
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors