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

NCT03183375 | Unknown Phase 2 DMC

• 1 other identifier: ERB0002
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

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).

Conditions

Thalassemia, Beta

Outcome Measures

Primary Outcomes (1)

• No. of participants who become responders or partial responders

  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.

  6 months

Secondary Outcomes (3)

• The compliance of Hydroxyurea in study participants.

  6 months

• Number of participants with abnormal/deranged laboratory values

  6 months

• No. of participants with severe adverse events related to the intervention

  6 months

Study Arms (2)

Hydroxyurea arm

EXPERIMENTAL

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

Standard arm

NO INTERVENTION

this arm will only receive the standard treatment which is blood transfusion and iron chelation

Interventions

Hydroxyurea DRUG

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)

Hydroxyurea arm

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Dr.Saqib Hussain Ansari: lead

Study Sites (1)

Omair Sana Foundation

Karachi, Sindh, 75300, Pakistan

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Interventions

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)

Urea; Amides; Organic Chemicals

Study Officials

• Saqib H Ansari, MBBS, DCH, DPGN, Mphil, PhD

  Omair Sana Foundation

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Saqib H Ansari, MBBS, DCH, DPGN, Mphil, PhD

CONTACT

+93002118018; muddasirsaqib@yahoo.com

Salima M Khowaja, BScN, MSBE

CONTACT

+93322696554; sohanisalima@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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.

Study Record Dates

• First Submitted: June 5, 2017
• First Posted: June 12, 2017
• Study Start: August 21, 2017
• Primary Completion: April 30, 2018
• Study Completion: June 30, 2018
• Last Updated: September 20, 2017

Record last verified: 2017-09

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)