Ignite Creation Date:
2025-12-25 @ 3:46 AM
Ignite Modification Date:
2026-02-22 @ 4:56 AM
Study NCT ID:
NCT03651102
Status:
COMPLETED
Last Update Posted:
2022-03-25
First Post:
2018-08-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013789', 'term': 'Thalassemia'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D013792', 'term': 'Thalidomide'}, {'id': 'D006918', 'term': 'Hydroxyurea'}], 'ancestors': [{'id': 'D010797', 'term': 'Phthalimides'}, {'id': 'D010795', 'term': 'Phthalic Acids'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010881', 'term': 'Piperidones'}, {'id': 'D010880', 'term': 'Piperidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D054833', 'term': 'Isoindoles'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D014508', 'term': 'Urea'}, {'id': 'D000577', 'term': 'Amides'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 654}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-03', 'completionDateStruct': {'date': '2022-03-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-03-10', 'studyFirstSubmitDate': '2018-08-24', 'studyFirstSubmitQcDate': '2018-08-27', 'lastUpdatePostDateStruct': {'date': '2022-03-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-08-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-11-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Haemoglobin Level', 'timeFrame': '6-24 months', 'description': 'mg/dL'}, {'measure': 'Response Rate (Excellent, Good, Partial and No response)', 'timeFrame': '6-24 months'}, {'measure': 'Adverse Effects', 'timeFrame': '6-24 months'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Thalidomide', 'Thalassemia'], 'conditions': ['Thalassemia']}, 'referencesModule': {'references': [{'pmid': '28718348', 'type': 'BACKGROUND', '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.'}, {'pmid': '29931453', 'type': 'BACKGROUND', '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.'}, {'pmid': '20104280', 'type': 'BACKGROUND', '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.'}, {'pmid': '26810455', 'type': 'BACKGROUND', '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.'}, {'pmid': '18439858', 'type': 'BACKGROUND', '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.'}, {'pmid': '28850716', 'type': 'BACKGROUND', '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.'}, {'pmid': '24505535', 'type': 'BACKGROUND', '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.'}, {'pmid': '20104272', 'type': 'BACKGROUND', '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.'}, {'pmid': '37604857', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': "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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Transfusion dependent thalassemia patients refractory to Hydroxyurea\n\nExclusion Criteria:\n\n* Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery'}, 'identificationModule': {'nctId': 'NCT03651102', 'acronym': 'Thal-Thalido', 'briefTitle': 'Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia', 'organization': {'class': 'OTHER', 'fullName': 'Blood Diseases Clinic'}, 'officialTitle': 'Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan', 'orgStudyIdInfo': {'id': 'TDT'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Thalidomide in combination with hydroxyurea', 'description': '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.', 'interventionNames': ['Drug: Thalidomide and Hydroxyurea']}], 'interventions': [{'name': 'Thalidomide and Hydroxyurea', 'type': 'DRUG', 'description': 'Dosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day', 'armGroupLabels': ['Thalidomide in combination with hydroxyurea']}]}, 'contactsLocationsModule': {'locations': [{'zip': '0000', 'city': 'Peshawar', 'state': 'Khyber Pakhtukhwa', 'country': 'Pakistan', 'facility': 'Blood Diseases Clinic', 'geoPoint': {'lat': 34.008, 'lon': 71.57849}}], 'overallOfficials': [{'name': 'Muhammad Tariq M Khan, MBBS, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Blood Diseases Clinic'}, {'name': 'Zahid Ali, PharmD, MPhil', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Department of Pharmacy, University of Peshawar'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Blood Diseases Clinic', 'class': 'OTHER'}, 'collaborators': [{'name': 'University of Peshawar', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Consultant Haematologist; Research Associate', 'investigatorFullName': 'Muhammad Tariq Masood Khan', 'investigatorAffiliation': 'Blood Diseases Clinic'}}}}