MPN-RC 118 AVID200 in Myelofibrosis
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|ClinicalTrials.gov Identifier: NCT03895112|
Recruitment Status : Recruiting
First Posted : March 29, 2019
Last Update Posted : June 2, 2020
Increased levels of TGF-β1 were detected in serum, plasma and BM and positively correlated with both grade of BMF and extent of leukemic cell infiltration in the marrow. TGF-β likely plays a dual role in promoting myelofibrosis and myeloproliferation, both of which are the bone marrow morphologic hallmark of MF. AVID200 is a drug that targets TGF-β1 and TGF-β3. The study team hypothesizes that inhibiting the TGF-β signaling pathway in MF will decrease the fibrogenic stimuli leading to myelofibrosis and concomitantly interrupt myeloproliferation and restore normal hematopoiesis.
This is a first in human, open-label, multicenter, Phase I/Ib trial of AVID200. Patients must have intermediate-2 or higher primary myelofibrosis (PMF), post-essential thrombocythemia or polycythemia-vera related MF (Post ET/PV MF). This study will enroll up to 24 patients. AVID200 is delivered by IV infusion on day 1 of each 3 week cycle.
|Condition or disease||Intervention/treatment||Phase|
|Primary Myelofibrosis Post-essential Thrombocythemia Myelofibrosis Post-polycythemia Vera Myelofibrosis Post ET MF Post PV MF||Drug: AVID200||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study of AVID200 in Patients With Myelofibrosis (Myeloproliferative Neoplasms Research Consortium [MPN-RC] 118)|
|Actual Study Start Date :||February 15, 2019|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||March 2023|
intravenous in dose cohorts of 70mg/m2 or 180 mg/m2
dose cohorts of 21-day cycles
- Maximally tolerated dose (MTD) of AVID200 [ Time Frame: After 6 cycles (Each cycle is 21 days) ]Cohorts of 3 patients with a maximum evaluable sample size of 12 patients and a target toxicity rate of 30% to estimate the MTD. Each cycle is 21 days.
- Number of patients with response eligibility for Phase 1b [ Time Frame: After 6 cycles ]Subjects attaining at least a CI (clinical improvement) by IWG/ELN criteria, or a decrease in bone marrow fibrosis by ≥1 grade with otherwise stable disease, will be allowed to continue AVID200 in the extension phase of the trial.
- IWG/ELN criteria [ Time Frame: After 6 cycles and after 12 cycles (Each cycle is 21 days) ]response by IWG/ELN criteria at the end of Cycle 6 and Cycle 12. The IWG/ELN criteria: CR (complete remission), PR (partial remission), Clinical improvement, Anemia response, Spleen response, Symptoms response, PD (progressive disease), SD (stable disease), Relapse, Cytogenetic remission, and Molecular remission
- Bone marrow fibrosis grade [ Time Frame: up to 37 days after 13 cycles (Each cycle is 21 days) ]bone marrow fibrosis grade at the end of Cycle 6 and 12. Bone marrow fibrosis (MF) is graded as MF-0 to MF-3, with higher number indicating more disease.
- Myelofibrosis Symptom Assessment Form (MFSAF) [ Time Frame: up to 37 days after 13 cycles (Each cycle is 21 days) ]MF-SAFv4.0,each of the items are scored 0 to 10, with total summation from 0 to 100, with higher score indicating more symptoms.
- EORTC QLQ-C30 [ Time Frame: up to 37 days after 13 cycles (Each cycle is 21 days) ]EORTC QLQ-C30, 28 item scored from 1 (not at all) to 4 (very much), and 2 items scored 1 (very poor) to 7 (excellent). Total score from 28 - 126, with higher score indicating poorer health
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03895112
|Contact: John Mascarenhas, MDfirstname.lastname@example.org|
|Contact: Lonette Sandyemail@example.com|
|United States, New York|
|Icahn School of Medicine at Mount Sinai||Recruiting|
|New York, New York, United States, 10029|
|Contact: Lonette Sandy 212-241-4546 firstname.lastname@example.org|
|Principal Investigator: John Mascarenhas, MD|
|Study Chair:||John Mascarenhas, MD||Icahn School of Medicine at Mount Sinai|
|Study Chair:||Ruben Mesa, MD||Mays Cancer Center at UT Health|
|Study Director:||Ronald Hoffman, MD||Icahn School of Medicine at Mount Sinai|