Clinical trial NCT03651102: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
Official title: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Thalassemia
Keywords: Thalidomide
Thalassemia
Lead sponsor: Blood Diseases Clinic
Sponsor class: OTHER
Collaborators: name: University of Peshawar
First posted: 2018-08-29
Enrollment: 654
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Haemoglobin Level
timeFrame: 6-24 months
measure: Response Rate (Excellent, Good, Partial and No response)
timeFrame: 6-24 months
measure: Adverse Effects
timeFrame: 6-24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Peshawar
country: Pakistan
facility: Blood Diseases Clinic
state: Khyber Pakhtukhwa
Study officials:
affiliation: Blood Diseases Clinic
name: Muhammad Tariq M Khan, MBBS, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Pharmacy, University of Peshawar
name: Zahid Ali, PharmD, MPhil
role: STUDY_DIRECTOR
References:
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.
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.
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. No abstract available.
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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.