[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05371964":3,"trial-entities:NCT05371964":191,"trial-summary:NCT05371964":194},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":25,"interventions":28,"primary_outcomes":42,"secondary_outcomes":54,"sex":101,"minimum_age":102,"maximum_age":17,"healthy_volunteers":15,"eligibility_criteria":103,"std_ages":135,"locations":138,"central_contacts":184,"overall_officials":185,"references":189,"see_also_links":190},"NCT05371964","MYF1001","A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Participants With Myelofibrosis","An Open Label, Phase 1\u002F1b Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Patients With Myelofibrosis","ACTIVE_NOT_RECRUITING","2028-08","2026-08","2026-08-26","2022-05-04","Geron Corporation","INDUSTRY",false,"The purpose of the study is to identify the recommended Part 2 dose (R2PD) of imetelstat sodium in combination with ruxolitinib in participants with myelofibrosis (MF) in Part 1, and to evaluate the safety and preliminary clinical activity of the R2PD of imetelstat sodium in combination with ruxolitinib in participants with MF in Part 2.",null,[19],"Myelofibrosis",[],"INTERVENTIONAL","TREATMENT",[24],"PHASE1",{"count":26,"type":27},30,"ACTUAL",[29,38],{"type":30,"name":31,"description":32,"armGroupLabels":33,"otherNames":36},"DRUG","Imetelstat sodium","Imetelstat sodium will be administered as intravenous (IV) every 28 days.",[34,35],"Part 1: Imetelstat sodium + Ruxolitinib","Part 2: Imetelstat sodium + Ruxolitinib",[37],"GRN163L",{"type":30,"name":39,"description":40,"armGroupLabels":41},"Ruxolitinib","Ruxolitinib will be administered, orally (PO), twice daily (BID) in cohort B as the standard of care per local prescribing guidelines.",[34,35],[43,46,50],{"measure":44,"timeFrame":45},"Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and\u002For Study Treatment","28 days after first dose",{"measure":47,"description":48,"timeFrame":49},"Part 2: Number of Participants With Treatment-emergent Adverse Event (AE)","Safety will be assessed based on incidence and severity (according to Common Terminology Criteria for Adverse Events) of treatment emergent adverse events from the first dose of study treatment until 30 days after completion of treatment.","First dose of study treatment until 30 days after the last dose of study treatment (up to approximately 5 years)",{"measure":51,"description":52,"timeFrame":53},"Part 2: Symptom Response Rate at Week 24","Symptom response rate is defined as percentage of participants with \\>=50% reduction in the Total Symptom Score (TSS) measured by the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 e-diary at 24 week compared to baseline.","Week 24",[55,58,61,63,65,67,71,74,77,80,84,87,91,95,98],{"measure":56,"timeFrame":57},"Part 1: Pharmacokinetic Profile of Ruxolitinib (Maximum Observed Plasma Concentration [Cmax]","From first dose of Ruxolitinib treatment up to approximately 5 years",{"measure":59,"timeFrame":60},"Part 1: Pharmacokinetic Profile of Ruxolitinib Time to Reach Maximum Plasma Concentration [Tmax])","From first dose of imetelstat treatment up to approximately 5 years",{"measure":62,"timeFrame":60},"Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Sodium Maximum Observed Plasma Concentration [Cmax]",{"measure":64,"timeFrame":60},"Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Time to Reach Maximum Plasma Concentration [Tmax])",{"measure":66,"timeFrame":60},"Part 1 and Part 2: Percentage of Participants with Anti-imetelstat Antibodies",{"measure":68,"description":69,"timeFrame":70},"Part 1: Symptom Response at Week 24","Symptom response rate is defined as percentage of participants with \\>50% reduction in the TSS measured by the MFSAF v4.0 e-diary at 24 week compared to baseline.","Baseline, Week 24",{"measure":72,"description":73,"timeFrame":70},"Part 1 and Part 2: Absolute Change From Baseline in TSS at Week 24","Absolute change is defined as change in total symptom score from baseline to week 24 as measured by the MFSAF v4.0 e-diary.",{"measure":75,"description":76,"timeFrame":70},"Part 1 and Part 2: Average Absolute Change in TSS Over 24 weeks","Average absolute change in TSS is defined as change in the average of absolute change in total symptom score from week 1(baseline) to week 24 as measured by the MFSAF v4.0 e-diary.",{"measure":78,"description":79,"timeFrame":53},"Part 1 and Part 2: Spleen Response at Week 24","Spleen response is the proportion of participants who achieve a reduction in spleen volume of ≥35% from baseline confirmed by magnetic resonance imaging (MRI) or computed tomography (CT).",{"measure":81,"description":82,"timeFrame":83},"Part 1 and Part 2: Progression Free Survival (PFS)","The time interval from start of study treatment date to the first date of disease progression or death from any cause, whichever occurs first.","From start of study treatment date to the disease progression or death (up to approximately 5 years)",{"measure":85,"timeFrame":86},"Part 1 and Part 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), Clinical Improvement (CI) Per the Modified 2013 International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria.","From first dose to end of the treatment (up to approximately 5 years)",{"measure":88,"description":89,"timeFrame":90},"Part 1 and Part 2: Time to Response","Time to response was defined as the duration from first dose of study treatment to the earliest date that a response is first documented. The response CI\u002FCR\u002FPR was assessed by IWG-MRT criteria.","From first dose of study treatment to the earliest date that a response was first documented (Up to approximately 5 years)",{"measure":92,"description":93,"timeFrame":94},"Part 1 and Part 2: Duration of Response (DOR) Per IWG-MRT Criteria","DOR measured from time of initial response (CR\u002FPR\u002FCI) until documented PD or death whichever occurs first.","From time of initial response to PD or death whichever occurs first (up to approximately 5 years)",{"measure":96,"description":97,"timeFrame":86},"Part 1 and Part 2: Reduction of Bone Marrow Fibrosis","Reduction of bone marrow fibrosis is defined as the percentage of participants with a post-baseline bone marrow fibrosis degree smaller than the baseline fibrosis degree prior to start of subsequent anticancer therapy.",{"measure":99,"description":100,"timeFrame":86},"Part 1 and Part 2: Time to Progression to Acute Myeloid Leukemia (AML)","Time to progression to AML, defined as the time interval from the start of study treatment date to the first date of documented progression to AML or death from any cause, whichever occurs first.","ALL","18 Years",{"inclusion":104,"exclusion":119,"raw_text":134},[105,106,107,108,109,110,111,112,113,114,115,116,117,118],"Diagnosis of primary myelofibrosis (PMF) according to the revised World Health Organization (WHO) criteria or post-essential thrombocythemia-MF or post-polycythemia vera according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria.","Dynamic International Prognostic Scoring System (DIPSS) intermediate-1, intermediate-2 or high-risk MF.","Candidate for ruxolitinib treatment:","Part 1 participants: On ruxolitinib treatment for at least 12 weeks with at least 4 consecutive weeks immediately prior to enrollment at a stable dose.","Part 2 participants: Candidate for ruxolitinib treatment as assessed by the investigator and has not previously been treated with a JAK inhibitor (Cohort A) OR currently receiving ruxolitinib per standard of care for at least 12 weeks with at least 4 consecutive weeks at a stable dose prior to enrollment (Cohort B). Note that the study will no longer recruit participants into Cohort A.","Active symptoms of MF on the MFSAF v4.0 demonstrated by:","Part 1 participants only: At least 2 symptoms with a score ≥ 1","Part 2 participants only: At least 2 symptoms with a score of ≥ 3, or a total score of at least 10.","Ineligible for or unwilling to undergo hematopoietic stem cell transplant at time of study entry.","Hematology laboratory test values within protocol defined limits.","Biochemical laboratory test values within protocol defined limits.","Eastern Cooperative Oncology Group Performance Status score of 0, 1, or 2.","Participants should follow protocol defined contraceptives procedures.","A woman of childbearing potential must have a negative serum or urine pregnancy test at screening.",[120,121,122,123,124,125,126,127,128,129,130,131,132,133],"Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%.","Prior treatment with JAK inhibitor (except for participants being dosed optimized on ruxolitinib treatment prior to screening and enrollment in part 1 or Part 2 Cohort B).","Known allergies, hypersensitivity, or intolerance to imetelstat or ruxolitinib or excipients.","Prior treatment with imetelstat.","Major surgery within 28 days prior to enrollment.","Any investigational drug regardless of class or mechanism of action, hydroxyurea, chemotherapy, (except for ruxolitinib for participants being dose optimized prior to enrollment), immunomodulatory or immunosuppressive therapy, corticosteroids \\>30 mg\u002Fday prednisone or equivalent ≤14 days prior to enrollment.","Prior history of hematopoietic stem cell transplant.","Diagnosis or treatment for malignancy other than MF, except:","Malignancy treated with curative intent and with no known active disease present for ≥3 years before enrollment.","Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease.","Adequately treated cervical carcinoma in situ without evidence of disease.","Clinically significant cardiovascular disease.","Known history of human immunodeficiency virus (HIV) or any uncontrolled active systemic infection requiring IV antibiotics.","Active systemic hepatitis infection requiring treatment or any known acute or chronic liver disease unless related to MF. Carriers of hepatitis virus are permitted to enter the study.","Inclusion Criteria:\n\n* Diagnosis of primary myelofibrosis (PMF) according to the revised World Health Organization (WHO) criteria or post-essential thrombocythemia-MF or post-polycythemia vera according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria.\n* Dynamic International Prognostic Scoring System (DIPSS) intermediate-1, intermediate-2 or high-risk MF.\n* Candidate for ruxolitinib treatment:\n\n  * Part 1 participants: On ruxolitinib treatment for at least 12 weeks with at least 4 consecutive weeks immediately prior to enrollment at a stable dose.\n  * Part 2 participants: Candidate for ruxolitinib treatment as assessed by the investigator and has not previously been treated with a JAK inhibitor (Cohort A) OR currently receiving ruxolitinib per standard of care for at least 12 weeks with at least 4 consecutive weeks at a stable dose prior to enrollment (Cohort B). Note that the study will no longer recruit participants into Cohort A.\n* Active symptoms of MF on the MFSAF v4.0 demonstrated by:\n\n  * Part 1 participants only: At least 2 symptoms with a score ≥ 1\n  * Part 2 participants only: At least 2 symptoms with a score of ≥ 3, or a total score of at least 10.\n* Ineligible for or unwilling to undergo hematopoietic stem cell transplant at time of study entry.\n* Hematology laboratory test values within protocol defined limits.\n* Biochemical laboratory test values within protocol defined limits.\n* Eastern Cooperative Oncology Group Performance Status score of 0, 1, or 2.\n* Participants should follow protocol defined contraceptives procedures.\n* A woman of childbearing potential must have a negative serum or urine pregnancy test at screening.\n\nExclusion Criteria:\n\n* Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%.\n* Prior treatment with JAK inhibitor (except for participants being dosed optimized on ruxolitinib treatment prior to screening and enrollment in part 1 or Part 2 Cohort B).\n* Known allergies, hypersensitivity, or intolerance to imetelstat or ruxolitinib or excipients.\n* Prior treatment with imetelstat.\n* Major surgery within 28 days prior to enrollment.\n* Any investigational drug regardless of class or mechanism of action, hydroxyurea, chemotherapy, (except for ruxolitinib for participants being dose optimized prior to enrollment), immunomodulatory or immunosuppressive therapy, corticosteroids \\>30 mg\u002Fday prednisone or equivalent ≤14 days prior to enrollment.\n* Prior history of hematopoietic stem cell transplant.\n* Diagnosis or treatment for malignancy other than MF, except:\n\n  * Malignancy treated with curative intent and with no known active disease present for ≥3 years before enrollment.\n  * Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease.\n  * Adequately treated cervical carcinoma in situ without evidence of disease.\n* Clinically significant cardiovascular disease.\n* Known history of human immunodeficiency virus (HIV) or any uncontrolled active systemic infection requiring IV antibiotics.\n* Active systemic hepatitis infection requiring treatment or any known acute or chronic liver disease unless related to MF. Carriers of hepatitis virus are permitted to enter the study.",[136,137],"ADULT","OLDER_ADULT",[139,148,154,162,169,176],{"facility":140,"city":141,"state":142,"zip":143,"country":144,"geoPoint":145},"City of Hope","Duarte","California","91010","United States",{"lat":146,"lon":147},34.13945,-117.97729,{"facility":140,"city":149,"state":142,"zip":150,"country":144,"geoPoint":151},"Irvine","92618",{"lat":152,"lon":153},33.66946,-117.82311,{"facility":155,"city":156,"state":157,"zip":158,"country":144,"geoPoint":159},"University of Miami","Coral Gables","Florida","33146",{"lat":160,"lon":161},25.72149,-80.26838,{"facility":163,"city":164,"state":157,"zip":165,"country":144,"geoPoint":166},"H. Lee Moffitt Cancer Center and Research Institute, Inc.","Tampa","33612",{"lat":167,"lon":168},27.94752,-82.45843,{"facility":170,"city":171,"state":171,"zip":172,"country":144,"geoPoint":173},"Icahn School of Medicine at Mount Sinai","New York","10029",{"lat":174,"lon":175},40.71427,-74.00597,{"facility":177,"city":178,"state":179,"zip":180,"country":144,"geoPoint":181},"Fred Hutchinson Cancer Center","Seattle","Washington","98109",{"lat":182,"lon":183},47.60621,-122.33207,[],[186],{"name":187,"affiliation":13,"role":188},"Michelle Mudge-Riley, DO","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":192,"biomarkers":193},[19],[],{"nct_id":4,"found":195,"summary":196,"prompt_version":206},true,{"design":197,"status":198,"heading":199,"summary":200,"follow_up":201,"word_count":202,"commitments":203,"compensation":204,"drugs_mentioned":205},"This interventional study plans to enroll 36 participants. It aims to find the right dose of imetelstat sodium to combine with ruxolitinib, and then evaluate the safety and activity of that combination.","completed","A Study of Imetelstat with Ruxolitinib for Myelofibrosis","This study is looking at the safety and effects of combining two drugs, imetelstat sodium and ruxolitinib, for people with myelofibrosis (a bone marrow disorder). Researchers want to find the best dose of imetelstat sodium to use with ruxolitinib. They will also check for any side effects and see how well the combination works to improve symptoms. You may be able to join if you are 18 or older and have been diagnosed with primary myelofibrosis, or myelofibrosis that developed after essential thrombocythemia or polycythemia vera. The study will measure side effects and how many participants have symptom improvement at 24 weeks. The current status of this study is unclear.","Participants will be followed for treatment-emergent adverse events until 30 days after the last dose of study treatment, for up to approximately 5 years.",110,"Imetelstat sodium will be given through a vein every 28 days. Ruxolitinib will be taken by mouth twice daily. The study will monitor you for adverse events from the first dose until 30 days after your last dose, for up to approximately 5 years.","Not stated in the trial record.",[31,39],"v2"]