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The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan

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ClinicalTrials.gov Identifier: NCT03183375
Recruitment Status : Recruiting
First Posted : June 12, 2017
Last Update Posted : September 20, 2017
Sponsor:
Information provided by (Responsible Party):
Dr.Saqib Hussain Ansari, Omair Sana Foundation

Brief Summary:

Objectives

Primary objectives:

  • To determine the efficacy of Hydroxyurea in the study participants.
  • Hypothesis: The study will result in either maintenance or rise in hemoglobin as compared to the control treatment.

Secondary objectives:

  • To determine the compliance of Hydroxyurea in study participants.
  • To determine the safety of Hydroxyurea in the study participants. Design and Outcomes

An open label randomized controlled trial to test the efficacy and safety of Hydroxyurea on beta thalassemia major patients. It is a six months study. Findings of physical examination, vital sign variables, laboratory variables and ultrasound at baseline, during and end of the study will be listed. Schedule of intervention is mentioned in section 6.1. later in the protocol.

Interventions and Duration Hydroxyurea will be given to the participants in intervention arm along with the standard treatment if thalassemia (blood transfusion and iron chelation therapy) and the control arm will receive the standard treatment (blood transfusion and iron chelation therapy) only. Each participant will be followed up for 6 months after initiating the intervention. Intervention will be given for 6 months or until the participant withdraws from the study or due to any reason, the investigator stops the intervention.

Sample Size and Population This pilot study will be done on 100 patients initially. Stratified randomization will be done on the basis of presence of Xmn polymorphism. And the study population will be assigned to intervention or control arm randomly through a computer software (randomizer.org).


Condition or disease Intervention/treatment Phase
Thalassemia, Beta Drug: Hydroxyurea Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: One is intervention arm which will be given investigational drug along with standard treatment and one will be control arm which will be given standard treatment only. Both groups will be assigned to only one treatment parallelly.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Randomized Controlled Trial to Evaluate the Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
Actual Study Start Date : August 21, 2017
Estimated Primary Completion Date : April 30, 2018
Estimated Study Completion Date : June 30, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia
Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: Hydroxyurea arm
This arm will be given investigational drug that is Hydroxyurea .This intervention will be given along with the standard treatment that is blood transfusion and iron chelation.
Drug: Hydroxyurea
Hydroxyurea (starting from 10mg/kg/day with increasing dose by 2mg/kg/day until the desired response is achieved. The maximum dose given will be 20mg/kg/day)

No Intervention: Standard arm
this arm will only receive the standard treatment which is blood transfusion and iron chelation



Primary Outcome Measures :
  1. No. of participants who become responders or partial responders [ Time Frame: 6 months ]
    The responders will be those who become transfusion independent (in those who were transfusion dependent before Hydroxyurea) or those who maintain or show 1-2 g/dl increase in Hb from baseline and partial responders will be those whose transfusion requirement decreases by atleast 50% as compared to baseline.


Secondary Outcome Measures :
  1. The compliance of Hydroxyurea in study participants. [ Time Frame: 6 months ]
    Compliance will be defined as actual number of tablets used/taken divided by the estimated number of tablets to be used/taken.

  2. Number of participants with abnormal/deranged laboratory values [ Time Frame: 6 months ]

    Participants with at least 1 of the following deranged laboratory values:

    • Deranged renal function (creatinine >2, Urea above normal range),
    • Liver function (SGPT 100 times more from baseline)
    • Neutropenia (neutrophil <1000)
    • Platelets < 50, 000
    • Deranged renal function (creatinine >2, Urea above normal range),
    • Liver function (SGPT 100 times more from baseline)
    • Neutropenia (neutrophil <1000)
    • Platelets < 50, 000 Deranged renal function (creatinine >2, Urea above normal range), Liver function (SGPT 100 times more from baseline),Neutropenia (neutrophil <1000),Platelets < 50, 000

  3. No. of participants with severe adverse events related to the intervention [ Time Frame: 6 months ]


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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with homozygous beta thalassemia major diagnosed on the basis of genetic mutation
  • Beta thalassemia intermedia (patients carrying homozygous, heterozygous or compound heterozygous beta thalassemia genes) (HbS, HbE)
  • Those patients who do not have baseline HbE suggestive of beta thalassemia, genetic mutations will be performed to confirm the diagnosis.
  • Age: 6 months and onwards
  • Gender: Either
  • Able to understand study procedures and to comply with them for the entire length of the study.
  • Provide written informed consent if aged 18 years and above and if minor that is below 18 years (Parental consent will be taken)

Exclusion Criteria:

  • Chronic liver disease, renal failure, history of stroke
  • Participants who have developed immune hemolytic anemia
  • Spleenomegaly (liver and spleen >5 cm below coastal margin
  • If allergic or sensitive to Hydroxyurea or its ingredients
  • Patients on immunosuppressants including Azathiopine or any other drug causing bone marrow suppression, Hepatitis C treatment that may cause red cell suppression and NSAIDS
  • Inability or unwillingness of individual or legal guardian/representative to give written informed consent.

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): NCT03183375


Contacts
Contact: Saqib H Ansari, MBBS, DCH, DPGN, Mphil, PhD +93002118018 muddasirsaqib@yahoo.com
Contact: Salima M Khowaja, BScN, MSBE +93322696554 sohanisalima@gmail.com

Locations
Pakistan
Omair Sana Foundation Recruiting
Karachi, Sindh, Pakistan, 75300
Contact: Saqib H Ansari, MBBS, DCH, DPGN, Mphil, PhD    03002118018    muddasirsaqib@yahoo.com   
Contact: Salima M Ali, BScN, MS    03322696554    sohanisalima@gmail.com   
Sponsors and Collaborators
Dr.Saqib Hussain Ansari
Investigators
Principal Investigator: Saqib H Ansari, MBBS, DCH, DPGN, Mphil, PhD Omair Sana Foundation

Responsible Party: Dr.Saqib Hussain Ansari, Principal Investigator, Omair Sana Foundation
ClinicalTrials.gov Identifier: NCT03183375     History of Changes
Other Study ID Numbers: ERB0002
First Posted: June 12, 2017    Key Record Dates
Last Update Posted: September 20, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors