Ignite Creation Date:
2025-12-24 @ 3:04 PM
Ignite Modification Date:
2026-01-01 @ 8:12 PM
Study NCT ID:
NCT07292259
Status:
RECRUITING
Last Update Posted:
2025-12-18
First Post:
2025-11-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D006918', 'term': 'Hydroxyurea'}, {'id': 'D013792', 'term': 'Thalidomide'}], 'ancestors': [{'id': 'D014508', 'term': 'Urea'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010797', 'term': 'Phthalimides'}, {'id': 'D010795', 'term': 'Phthalic Acids'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 150}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2025-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-05', 'studyFirstSubmitDate': '2025-11-13', 'studyFirstSubmitQcDate': '2025-12-05', 'lastUpdatePostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2025-12-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Red Blood Cell Transfusions after starting a combination of Thalidomide and hydroxyurea', 'timeFrame': '01 Year', 'description': 'The frequency of Red Blood cell transfusions before the start of intervention will be noted. At 03 months, 06 months, and 12 months of intervention (Thalidomide and hydroxyurea) number of Red Blood cell transfusions will be documented.'}], 'secondaryOutcomes': [{'measure': 'To document the spectrum and frequency of significant adverse drug reactions.', 'timeFrame': '01 Year', 'description': 'Monitoring and recording any adverse effects associated with thalidomide and hydroxyurea therapy at Day-90, Day-180, and Day-365.\n\nPatients will be contacted on the telephone weekly by the drug safety monitoring board and will be screened for any side effects of the intervention.'}, {'measure': 'To assess changes in spleen size during the intervention.', 'timeFrame': '01 Year', 'description': 'Assessment of spleen size in centimeters at Day-01, Day-90, Day-180, and Day-365 of intervention.'}, {'measure': 'Monitoring of Serum Ferritin levels in ng/ml during the time of intervention.', 'timeFrame': '01 Year', 'description': 'Measurement of Serum Ferritin Levels in ng/ml at Day-01, Day-90, Day-180, and Day-365.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Thalidomide, Hydoxyurea, Transfusion dependent thalassemia'], 'conditions': ['Transfusion Dependent Beta Thalassemia']}, 'referencesModule': {'references': [{'pmid': '34456732', 'type': 'RESULT', 'citation': 'Li X, Hu S, Liu Y, Huang J, Hong W, Xu L, Xu H, Fang J. Efficacy of Thalidomide Treatment in Children With Transfusion Dependent beta-Thalassemia: A Retrospective Clinical Study. Front Pharmacol. 2021 Aug 12;12:722502. doi: 10.3389/fphar.2021.722502. eCollection 2021.'}, {'pmid': '36699430', 'type': 'RESULT', 'citation': 'Garg A, Patel K, Shah K, Trivedi D, Raj A, Yadav R, Shah S. Safety and Efficacy of Thalidomide and Hydroxyurea Combination in Beta Thalassemia Patients. Indian J Hematol Blood Transfus. 2023 Jan;39(1):85-89. doi: 10.1007/s12288-022-01536-y. Epub 2022 Apr 21.'}, {'pmid': '28850716', 'type': 'RESULT', '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': '32425398', 'type': 'RESULT', 'citation': 'Nag A, Radhakrishnan VS, Kumar J, Bhave S, Mishra DK, Nair R, Chandy M. Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience. Indian J Hematol Blood Transfus. 2020 Apr;36(2):399-402. doi: 10.1007/s12288-020-01263-2. Epub 2020 Mar 2.'}, {'pmid': '35477175', 'type': 'RESULT', 'citation': 'Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.'}]}, 'descriptionModule': {'briefSummary': 'Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.\n\nIn Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.\n\nThe standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.', 'detailedDescription': 'Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.\n\nIn Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.\n\nThe standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.\n\nHydroxyurea (HU), an FDA-approved inducer of fetal hemoglobin (HbF), has shown promise in reducing or eliminating the need for frequent blood transfusions in β-thalassemia patients. However, a subset of patients exhibits limited responsiveness to HU, necessitating exploration of adjunct or alternative therapies. Thalidomide, an immune modulator, has demonstrated transfusion reduction by suppressing nuclear factor-κB induction, potentially increasing HbF levels.\n\nThe primary aim of this prospective study is to determine the efficacy of the combination of thalidomide and hydroxyurea in reducing transfusion frequency in patients with β-thalassemia Major. The secondary objectives are to document the spectrum of significant adverse drug reactions as well to document any alteration in the spleen size and serum ferritin levels.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '12 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n• Patients suffering from Transfusion Dependent β-thalassemia (TDBT) more than two years of age of either sex will be included in the study.\n\nExclusion Criteria:\n\n* Age less than two years.\n* Patients having cardiac, hepatic, pulmonary, renal or neurological dysfunction or history of thrombosis.\n* Both male and female participants of childbearing potential will be excluded due to the teratogenicity of thalidomide.'}, 'identificationModule': {'nctId': 'NCT07292259', 'acronym': 'Thal-H', 'briefTitle': 'Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia', 'organization': {'class': 'OTHER', 'fullName': 'Pakistan Blood and Marrow Transplant (PBMT) Group'}, 'officialTitle': 'The Efficacy of Combined Thalidomide and Hydroxyurea Therapy in Transfusion Dependent β-thalassemia (TDBT), Phase II Trial', 'orgStudyIdInfo': {'id': 'PBMT-1'}, 'secondaryIdInfos': [{'id': '6871', 'type': 'OTHER', 'domain': 'AFBMTC'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT', 'description': '* The trial include Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day).\n* To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily.', 'interventionNames': ['Drug: ADDITION OF THALIDOMIDE AND HYDROXYUREA']}], 'interventions': [{'name': 'ADDITION OF THALIDOMIDE AND HYDROXYUREA', 'type': 'DRUG', 'otherNames': ['Hydrea and Thalidomide'], 'description': 'The intervention includes Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day).\n\n* To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily.\n* Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload.', 'armGroupLabels': ['A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT']}]}, 'contactsLocationsModule': {'locations': [{'zip': '46000', 'city': 'Islamabad', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'Armed Forces Bone Marrow Transplant Center', 'geoPoint': {'lat': 29.7426, 'lon': 73.14353}}, {'zip': '44000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'Armed Forces Bone Marrow Transplant Center', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'contacts': [{'name': 'Tariq Khattak, FCPS', 'role': 'CONTACT', 'email': 'drtariqazamktk2629@gmail.com', 'phone': '+923215196104'}, {'name': 'Tariq Ghafoor, FCPS,FRCP', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'TariqAzam khattak, FCPS', 'role': 'SUB_INVESTIGATOR'}, {'name': 'HaiderNasir Awan, FCPS', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Aamir Aslam Awan, FCPS', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'AFBMTC (Clinical Trial and Research Cell)', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'AFBMTC (CT&RC), CMH Medical Complex', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'AFBMTC (CT&RC), Medical Complex', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'contacts': [{'name': 'Tariq Ghafoor, FCPS', 'role': 'CONTACT', 'email': 'drtariqghafoor@gmail.com', 'phone': '+92-300-8519006'}, {'name': 'Tariq Khattak, FCPS', 'role': 'CONTACT', 'email': 'drtariqazamktk2629@gmail.com', 'phone': '+923215196104'}, {'name': 'Tariq Ghafoor, FCPS,FRCP', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Tariq AzamKhattak, FCPS', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Aamir AslamAwan, FCPS', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Haider NisarAwan, FCPS', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'AFBMTC, CMH Medical Complex', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '46000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'contacts': [{'name': 'Ammad Akram, MBBS,FCPS', 'role': 'CONTACT', 'email': 'ammadakramch@gmail.com', 'phone': '03216332942'}], 'facility': 'Armed Forces Bone Marrow Transplant Center', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}, {'zip': '60000', 'city': 'Rawalpindi', 'state': 'Punjab Province', 'status': 'RECRUITING', 'country': 'Pakistan', 'facility': 'Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan', 'geoPoint': {'lat': 33.59733, 'lon': 73.0479}}], 'centralContacts': [{'name': 'Tariq Ghafoor, FCPS,FRCP', 'role': 'CONTACT', 'email': 'drtariqghafoor@gmail.com', 'phone': '+923008519006'}, {'name': 'Tariq Khattak, FCPS', 'role': 'CONTACT', 'email': 'drtariqazamktk2629@gmail.com', 'phone': '+923215196104'}], 'overallOfficials': [{'name': 'Tariq Ghafoor, FCPS,FRCP', 'role': 'STUDY_DIRECTOR', 'affiliation': 'National Institute of Blood and Marrow Transplant (NIBMT), Pakistan'}]}, 'ipdSharingStatementModule': {'url': 'https://pbmt.org.pk/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'timeFrame': 'Six months after completion of study', 'ipdSharing': 'YES', 'description': 'The investigators may share de-identified IPD upon reasonable request, beginning 9 months after publication and available for up to 24 months, following review and data-sharing agreement.', 'accessCriteria': 'Contact with PI'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pakistan Blood and Marrow Transplant (PBMT) Group', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Director AFBMTC', 'investigatorFullName': 'Professor Tariq Ghafoor', 'investigatorAffiliation': 'Pakistan Blood and Marrow Transplant (PBMT) Group'}}}}