NCT03183375

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

Status unknown Phase 2 Last updated 20 September 2017

What this trial tests

Phase 2 trial testing Hydroxyurea in Thalassemia, Beta in 100 participants. Status unknown.

Timeline

21 August 2017

Primary endpoint
30 April 2018

30 June 2018

Quick facts

Lead sponsor	Dr.Saqib Hussain Ansari
Phase	Phase 2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	100
Start date	21 August 2017
Primary completion	30 April 2018
Estimated completion	30 June 2018
Sites	1 location across Pakistan

Drugs / interventions tested

• Hydroxyurea — full drug profile →

Conditions studied

• Thalassemia, Beta — all drugs for Thalassemia, Beta →

Sponsor

Dr.Saqib Hussain Ansari

Who can join

6 Months and older, any sex, with Thalassemia, Beta. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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).

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. A comprehensive review of hydroxyurea for β-haemoglobinopathies: the role revisited during COVID-19 pandemic.
   Yasara N, Premawardhena A, Mettananda S. · · 2021 · cited 23× · PMID 33648529 · DOI 10.1186/s13023-021-01757-w
2. Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia.
   Ansari SH, Lassi ZS, Khowaja SM, Adil SO, et al · · 2019 · cited 18× · PMID 30882896 · DOI 10.1002/14651858.cd012064.pub2
3. Pharmacogenomics of Drugs Used in β-Thalassemia and Sickle-Cell Disease: From Basic Research to Clinical Applications.
   Gambari R, Waziri AD, Goonasekera H, Peprah E. · · 2024 · cited 14× · PMID 38673849 · DOI 10.3390/ijms25084263
4. Combined approaches for increasing fetal hemoglobin (HbF) and <i>de novo</i> production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients: treatment with HbF inducers and CRISPR-Cas9 based genome editing.
   Finotti A, Gambari R. · · 2023 · cited 6× · PMID 37529398 · DOI 10.3389/fgeed.2023.1204536
5. New Synthetic Isoxazole Derivatives Acting as Potent Inducers of Fetal Hemoglobin in Erythroid Precursor Cells Isolated from β-Thalassemic Patients.
   Zuccato C, Cosenza LC, Tupini C, Finotti A, et al · · 2023 · cited 3× · PMID 38202591 · DOI 10.3390/molecules29010008