Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia

NCT03651102 | Completed Phase 2 DMC

• 1 other identifier: TDT
• Study Type: interventional
• Target: 654
• Locations: 1 country
• Sites: 1

Brief Summary

Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.

Conditions

Thalassemia

Keywords

Thalidomide; Thalassemia

Outcome Measures

Primary Outcomes (3)

• Haemoglobin Level

  mg/dL

  6-24 months

• Response Rate (Excellent, Good, Partial and No response)

  6-24 months

• Adverse Effects

  6-24 months

Study Arms (1)

Thalidomide in combination with hydroxyurea

EXPERIMENTAL

All the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day.

Drug: Thalidomide and Hydroxyurea

Interventions

Thalidomide and Hydroxyurea DRUG

Dosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day

Thalidomide in combination with hydroxyurea

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Transfusion dependent thalassemia patients refractory to Hydroxyurea

You may not qualify if:

• Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery

Sponsors & Collaborators

• Blood Diseases Clinic: lead
• University of Peshawar: collaborator

Study Sites (1)

Blood Diseases Clinic

Peshawar, Khyber Pakhtukhwa, 0000, Pakistan

Location

Related Publications (9)

• Li Y, Ren Q, Zhou Y, Li P, Lin W, Yin X. Thalidomide has a significant effect in patients with thalassemia intermedia. Hematology. 2018 Jan;23(1):50-54. doi: 10.1080/10245332.2017.1354427. Epub 2017 Jul 18.

  PMID: 28718348 BACKGROUND

• Ren Q, Zhou YL, Wang L, Chen YS, Ma YN, Li PP, Yin XL. Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia. Ann Hematol. 2018 Oct;97(10):1933-1939. doi: 10.1007/s00277-018-3395-5. Epub 2018 Jun 22.

  PMID: 29931453 BACKGROUND

• Masera N, Tavecchia L, Capra M, Cazzaniga G, Vimercati C, Pozzi L, Biondi A, Masera G. Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy. Blood Transfus. 2010 Jan;8(1):63-5. doi: 10.2450/2009.0102-09. No abstract available.

  PMID: 20104280 BACKGROUND

• Ricchi P, Costantini S, Spasiano A, De Dominicis G, Di Matola T, Cinque P, Ammirabile M, Marsella M, Filosa A. The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with Non-Transfusion-Dependent Thalassemia. Blood Cells Mol Dis. 2016 Mar;57:97-9. doi: 10.1016/j.bcmd.2016.01.003. Epub 2016 Jan 16.

  PMID: 26810455 BACKGROUND

• Aguilar-Lopez LB, Delgado-Lamas JL, Rubio-Jurado B, Perea FJ, Ibarra B. Thalidomide therapy in a patient with thalassemia major. Blood Cells Mol Dis. 2008 Jul-Aug;41(1):136-7. doi: 10.1016/j.bcmd.2008.03.001. Epub 2008 Apr 24. No abstract available.

  PMID: 18439858 BACKGROUND

• Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.

  PMID: 28850716 BACKGROUND

• Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K. Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of beta-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res. 2013;7(3):47-54.

  PMID: 24505535 BACKGROUND

• Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 2010 Jan;8(1):5-7. doi: 10.2450/2009.0137-09. No abstract available.

  PMID: 20104272 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 DERIVED

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

• Muhammad Tariq M Khan, MBBS, PhD

  Blood Diseases Clinic

  PRINCIPAL INVESTIGATOR

• Zahid Ali, PharmD, MPhil

  Department of Pharmacy, University of Peshawar

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Consultant Haematologist; Research Associate

Study Record Dates

• First Submitted: August 24, 2018
• First Posted: August 29, 2018
• Study Start: January 1, 2018
• Primary Completion: November 30, 2021
• Study Completion: March 1, 2022
• Last Updated: March 25, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)