[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100644722":3},{"organization":4,"outcomesModule":7,"designInfo":31,"detailedDescription":38,"studyPopulation":30,"armGroups":39,"interventions":51,"overallOfficials":65,"centralContacts":70,"locations":78,"responsibleParty":95,"collaborators":30,"id":97,"slug":98,"hasResults":99,"nctId":100,"briefTitle":101,"officialTitle":102,"acronym":30,"eligibilityCriteria":103,"healthyVolunteers":99,"sex":104,"minAge":30,"maxAge":30,"enrollmentInfo":105,"targetDuration":30,"studyType":108,"phases":109,"briefSummary":111,"conditions":112,"keywords":30,"overallStatus":114,"whyStopped":30,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":119,"completionDateStruct":121,"leadSponsor":123,"locationsCount":124},{"fullName":5,"class":6},"Riphah International University","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":16,"otherOutcomes":30},[9,13],{"measure":10,"description":11,"timeFrame":12},"Improvement in the hemoglobin level","To evaluate the Improvement in Hemoglobin level, response criteria are defined as follows:\n\nMajor response, an elevation in total Hb level of ≥2 g\u002FdL Minor response, an elevation in total Hb level of 1 to 2 g\u002FdL, or Hb not substantially increased (\\\u003C1 g\u002FdL) but the patients achieve Hb\\>7 g\u002FL No response, an elevation in total Hb level of \\\u003C1 g\u002FdL","6 months",{"measure":14,"description":15,"timeFrame":12},"Decrease in transfusion requirement","To evaluate the decrease in transfusion requirement, response criteria are defined as follows:\n\nMajor response: reduce transfusion requirements by ≥25% after 6 months of therapy compared to baseline.\n\nMinor response: reduce transfusion requirement by \\\u003C25 % after 6 months of therapy,\n\nNo response: same blood transfusion requirement as baseline",[17,21,24,27],{"measure":18,"description":19,"timeFrame":20},"Serum Bilirubin levels","0.2- 1.1 mg\u002FdL Normal \\>1.1- 3 mg\u002FdL Mild derangement \\>3 mg\u002FdL Severe derangement","1 year",{"measure":22,"description":23,"timeFrame":20},"Change in ALT levels","ALT levels range:\n\n0- 42 U\u002FL Normal \\>42-126 U\u002FL Mild severity \\>126- 420 U\u002FL Moderate severity \\>420 U\u002FL severe",{"measure":25,"description":26,"timeFrame":20},"Change in serum creatinine levels","Serum creatinine 0.2 - 1.1 mg\u002FdL Normal \\>1.1- 1.5 mg\u002FdL Mild severity \\>1.5- 3.0 mg\u002FdL Moderate severity \\>3 mg\u002FdL Severe",{"measure":28,"description":29,"timeFrame":12},"Assess Quality of life by using EORTC QLQ-C30","EORTC QLQ C-30 URDU version, a self-administered questionnaire, will be filled by each participant at baseline and 6 months.\n\nFunctional\u002FGlobal:\n\n100 = perfect function. 0 = no function. 10-point change = clinically meaningful.\n\nSymptoms: 0 = no symptoms. 100 = maximum symptoms 10-point increase = worse symptom burden.",null,{"allocation":32,"interventionModel":33,"interventionModelDescription":34,"primaryPurpose":35,"observationalModel":30,"timePerspective":30,"maskingInfo":36},"NON_RANDOMIZED","PARALLEL","This is two-arm comparative study. One group is interventional group in which all patients will receive thalidomide and hydroxyurea. Low dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg\u002Fkg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurred. The starting dose of hydroxyurea will be 10-20 mg\u002Fkg per day. Second group will be control group on Blood transfusion.","TREATMENT",{"masking":37,"maskingDescription":30,"whoMasked":30},"NONE","This is a two-arm comparative study. One group is the interventional group, in which all patients will receive thalidomide and hydroxyurea. Low-dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg\u002Fkg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurs. The starting dose of hydroxyurea will be 10-20 mg\u002Fkg per day. The second group will be the control group for blood transfusion.\n\nIn the intervention group, 114 confirmed diagnoses of beta thalassemia ascertained by Hemoglobin Electrophoresis or HPLC report performed pre-transfusion or genetic testing profile (comprising PCR or HBB gene sequencing) suggestive of β-thalassemia syndrome will be included. To assess the therapeutic efficacy of HbF inducers (combination therapy: thalidomide and hydroxyurea), the number of blood transfusions and hemoglobin level will be assessed as per the given schedule.\n\n. Adverse events will be recorded at each follow-up to assess the safety of the therapy. The patient\u002Fguardian\u002Fparent will be specifically asked about paresthesia, rash, constipation, unexplained infections, bleeding symptoms, headache, syncope, focal weakness, and behavioral changes. All participants will be asked to report any adverse reactions and will be questioned about adverse events during the study visit. EORTC QLQ C-30 URDU version, a self-administered questionnaire, will be filled by each participant at baseline and 6 months.",[40,44],{"label":41,"type":42,"description":43,"interventionNames":30},"Blood transfusion Group","NO_INTERVENTION","Control group will be on regular blood transfusion and iron chelation therapy",{"label":45,"type":46,"description":47,"interventionNames":48},"HbF inducers","EXPERIMENTAL","All patients will receive thalidomide and hydroxyurea. Low dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg\u002Fkg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurred. The starting dose of hydroxyurea will be 10-20 mg\u002Fkg per day.",[49,50],"Drug: Thalidomide","Drug: Hydroxy Urea",[52,59],{"type":53,"name":54,"description":55,"armGroupLabels":56,"otherNames":57},"DRUG","Thalidomide","Tab Thalidomide 0.5 to 4 mg\u002Fkg orally every day for 12 months",[45],[58],"Thalido",{"type":53,"name":60,"description":61,"armGroupLabels":62,"otherNames":63},"Hydroxy Urea","Tab Hydroxyurea 10-20 mg\u002Fkg per day for 12 months",[45],[64],"Hydrea",[66],{"name":67,"affiliation":68,"role":69},"Fariha Sardar, MBBS, FCPS","Riphah International University, Rwp","PRINCIPAL_INVESTIGATOR",[71,75],{"name":67,"role":72,"phone":73,"phoneExt":30,"email":74},"CONTACT","+923335517877","doc.fariha@yahoo.com",{"name":67,"role":72,"phone":76,"phoneExt":30,"email":77},"+923350941494","afzaal2kmt20@gamil.com",[79],{"facility":5,"status":30,"city":80,"state":81,"zip":30,"country":82,"countryCode":83,"cosmosGeoPoint":84,"geoPoint":89,"contacts":90},"Rawalpindi","Punjab Province","Pakistan","PK",{"type":85,"coordinates":86},"Point",[87,88],73.0479,33.59733,{"lat":88,"lon":87},[91,93],{"name":92,"role":72,"phone":73,"phoneExt":30,"email":74},"Fariha Sadar, MBBS, FCPS",{"name":94,"role":72,"phone":76,"phoneExt":30,"email":77},"Fariha Saradr, MBBS, FCPS",{"type":96,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100644722","safety-and-efficacy-of-hemoglobin-f-inducers-in-patients-with-beta-thalassemia-100644722",false,"NCT07673302","Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia","Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia: a Prospective 12 Months Study","Inclusion criteria:\n\n* Confirmed diagnosis of Beta thalassemia Major (BTM) ascertained by Hemoglobin Electrophoresis or HPLC report performed pre-transfusion or genetic testing profile (comprising PCR or HBB gene sequencing) suggestive of β-thalassemia syndrome.\n* All ages and both genders will be included\n* Written informed consent\n\nExclusion criteria:\n\n* Pregnancy or unwilling to follow contraception or planning conception (Enrolled female patients will be strictly advised to avoid pregnancy during the study period and until 6 months after thalidomide withdrawal.\n* Hemoglobinopathies other than beta thalassemia\n* History of neurological problems\n* Inability to regularly follow up","ALL",{"count":106,"type":107},240,"ESTIMATED","INTERVENTIONAL",[110],"NA","The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients. The main objectives of this study are:\n\n* To determine the therapeutic efficacy of HbF inducers (combination therapy: thalidomide and hydroxyurea) on hemoglobin level and blood transfusion in beta thalassemia patients.\n* To determine the safety of HbF inducers (combination therapy: thalidomide and hydroxyurea) in beta thalassemia patients\n* To determine effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on quality of life of beta thalassemia patients",[113],"Thalassemia","NOT_YET_RECRUITING","2026-06-23",{"date":117,"type":118},"2026-06-29","ACTUAL",{"date":120,"type":107},"2026-06-20",{"date":122,"type":107},"2027-06-19",{"name":5,"class":6},1]