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NCT03651102 Clinical Trial

Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia

Thalassemia Clinical Trial

— Thal-Thalido  

Official title:
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT03651102
Other study ID # TDT
Secondary ID
Status Completed
Phase Phase 2/Phase 3
First received
Last updated
Start date January 1, 2018
Est. completion date March 1, 2022

Study information
Verified date March 2022
Source Blood Diseases Clinic
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

Recruitment information / eligibility
Status Completed
Enrollment 654
Est. completion date March 1, 2022
Est. primary completion date November 30, 2021
Accepts healthy volunteers No
Gender All
Age group 1 Year and older
Eligibility Inclusion Criteria: - Transfusion dependent thalassemia patients refractory to Hydroxyurea Exclusion Criteria: - Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery

Study Design

Related Conditions & MeSH terms

Intervention

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

Locations
Country Name City State
Pakistan Blood Diseases Clinic Peshawar Khyber Pakhtukhwa

Sponsors (2)
Lead Sponsor Collaborator
Blood Diseases Clinic University of Peshawar

Country where clinical trial is conducted

Pakistan, 

References & Publications (8)

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. — View Citation

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

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

Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 2010 Jan;8(1):5-7. doi: 10.2450/2009.0137-09. Review. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Outcome
Type Measure Description Time frame Safety issue
Primary Haemoglobin Level mg/dL 6-24 months
Primary Response Rate (Excellent, Good, Partial and No response) 6-24 months
Primary Adverse Effects 6-24 months
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