Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia

NCT07673302 | Not Yet Recruiting

• 1 other identifier: Riphah/IIMC/IRB/26/1048
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients. The main objectives of this study are:

• To determine the therapeutic efficacy of HbF inducers (combination therapy: thalidomide and hydroxyurea) on hemoglobin level and blood transfusion in beta thalassemia patients.
• To determine the safety of HbF inducers (combination therapy: thalidomide and hydroxyurea) in beta thalassemia patients
• To determine effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on quality of life of beta thalassemia patients

Conditions

Thalassemia

Outcome Measures

Primary Outcomes (2)

• Improvement in the hemoglobin level

  To evaluate the Improvement in Hemoglobin level, response criteria are defined as follows: Major response, an elevation in total Hb level of ≥2 g/dL Minor response, an elevation in total Hb level of 1 to 2 g/dL, or Hb not substantially increased (\<1 g/dL) but the patients achieve Hb\>7 g/L No response, an elevation in total Hb level of \<1 g/dL

  6 months

• Decrease in transfusion requirement

  To evaluate the decrease in transfusion requirement, response criteria are defined as follows: Major response: reduce transfusion requirements by ≥25% after 6 months of therapy compared to baseline. Minor response: reduce transfusion requirement by \<25 % after 6 months of therapy, No response: same blood transfusion requirement as baseline

  6 months

Secondary Outcomes (4)

• Serum Bilirubin levels

  1 year

• Change in ALT levels

  1 year

• Change in serum creatinine levels

  1 year

• Assess Quality of life by using EORTC QLQ-C30

  6 months

Study Arms (2)

Blood transfusion Group

NO INTERVENTION

Control group will be on regular blood transfusion and iron chelation therapy

HbF inducers

EXPERIMENTAL

All patients will receive thalidomide and hydroxyurea. Low dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg/kg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurred. The starting dose of hydroxyurea will be 10-20 mg/kg per day.

Drug: Thalidomide; Drug: Hydroxy Urea

Interventions

Thalidomide DRUG

Tab Thalidomide 0.5 to 4 mg/kg orally every day for 12 months

Also known as: Thalido

HbF inducers

Hydroxy Urea DRUG

Tab Hydroxyurea 10-20 mg/kg per day for 12 months

Also known as: Hydrea

HbF inducers

Eligibility Criteria

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

You may qualify if:

• Confirmed diagnosis of Beta thalassemia Major (BTM) ascertained by Hemoglobin Electrophoresis or HPLC report performed pre-transfusion or genetic testing profile (comprising PCR or HBB gene sequencing) suggestive of β-thalassemia syndrome.
• All ages and both genders will be included
• Written informed consent

You may not qualify if:

• Pregnancy or unwilling to follow contraception or planning conception (Enrolled female patients will be strictly advised to avoid pregnancy during the study period and until 6 months after thalidomide withdrawal.
• Hemoglobinopathies other than beta thalassemia
• History of neurological problems
• Inability to regularly follow up

Sponsors & Collaborators

• Riphah International University: lead

Study Sites (1)

Riphah International University

Rawalpindi, Punjab Province, Pakistan

Location

Related Publications (5)

• Chen JM, Zhu WJ, Liu J, Wang GZ, Chen XQ, Tan Y, Xu WW, Qu LW, Li JY, Yang HJ, Huang L, Cai N, Wang WD, Huang K, Xu JQ, Li GH, He S, Luo TY, Huang Y, Liu SH, Wu WQ, Lu QY, Zhou MG, Chen SY, Li RL, Hu ML, Huang Y, Wei JH, Li JM, Chen SJ, Zhou GB. Safety and efficacy of thalidomide in patients with transfusion-dependent beta-thalassemia: a randomized clinical trial. Signal Transduct Target Ther. 2021 Nov 18;6(1):405. doi: 10.1038/s41392-021-00811-0.

  PMID: 34795208 BACKGROUND

• Ali Z, Ismail M, Rehman IU, Rani GF, Ali M, Khan MTM. Long-term clinical efficacy and safety of thalidomide in patients with transfusion-dependent beta-thalassemia: results from Thal-Thalido study. Sci Rep. 2023 Aug 21;13(1):13592. doi: 10.1038/s41598-023-40849-4.

  PMID: 37604857 BACKGROUND

• Tuo Y, Li Y, Li Y, Ma J, Yang X, Wu S, Jin J, He Z. Global, regional, and national burden of thalassemia, 1990-2021: a systematic analysis for the global burden of disease study 2021. EClinicalMedicine. 2024 May 6;72:102619. doi: 10.1016/j.eclinm.2024.102619. eCollection 2024 Jun.

  PMID: 38745964 BACKGROUND

• Lu Y, Wei Z, Yang G, Lai Y, Liu R. Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ss-Thalassemia: A Meta-Analysis. Front Pharmacol. 2022 Jan 11;12:814302. doi: 10.3389/fphar.2021.814302. eCollection 2021.

  PMID: 35087410 BACKGROUND

• Shah S, Sheth R, Shah K, Patel K. Safety and effectiveness of thalidomide and hydroxyurea combination in beta-thalassaemia intermedia and major: a retrospective pilot study. Br J Haematol. 2020 Feb;188(3):e18-e21. doi: 10.1111/bjh.16272. Epub 2019 Nov 11. No abstract available.

  PMID: 31710694 BACKGROUND

MeSH Terms

Conditions

Thalassemia

Interventions

Thalidomide; Hydroxyurea

Condition Hierarchy (Ancestors)

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

• Fariha Sardar, MBBS, FCPS

  Riphah International University, Rwp

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Fariha Sardar, MBBS, FCPS

CONTACT

+923335517877; doc.fariha@yahoo.com

Fariha Sardar, MBBS, FCPS

CONTACT

+923350941494; afzaal2kmt20@gamil.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is two-arm comparative study. One group is interventional group in which all patients will receive thalidomide and hydroxyurea. Low dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg/kg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurred. The starting dose of hydroxyurea will be 10-20 mg/kg per day. Second group will be control group on Blood transfusion.

Study Record Dates

• First Submitted: June 23, 2026
• First Posted: June 29, 2026
• Study Start: June 20, 2026
• Primary Completion (Estimated): June 19, 2027
• Study Completion (Estimated): June 19, 2027
• Last Updated: June 29, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)