PRGN-3006 Adoptive Cellular Therapy Relapsed or Refractory AML or High Risk MDS
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|ClinicalTrials.gov Identifier: NCT03927261|
Recruitment Status : Recruiting
First Posted : April 25, 2019
Last Update Posted : May 21, 2019
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndromes Acute Myeloid Leukemia||Drug: PRGN-3006 T Cells||Phase 1|
This is a single center, nonrandomized, investigator-initiated Phase 1/1b safety and tolerability study. The safety and tolerability of PRGN-3006 T cells will be assessed following intravenous administration of escalating doses in patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndromes (MDS).
This study will enroll in two phases: an initial dose escalation phase followed by a dose expansion phase.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||56 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/1b Safety Study of PRGN-3006 Adoptive Cellular Therapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndrome|
|Actual Study Start Date :||May 17, 2019|
|Estimated Primary Completion Date :||May 1, 2022|
|Estimated Study Completion Date :||May 1, 2025|
Experimental: Dose Escalation and Dose Expansion of PRGN-3006
Participants will be treated in dose escalation phase to identify the safety and maximum tolerated dose (MTD) of PRGN-3006.
Drug: PRGN-3006 T Cells
Participants will receive a single intravenous (IV) administration of PRGN-3006 T Cells, and will be monitored for safety in the clinic for at least 7 days following infusion, and for safety, efficacy and correlative endpoints up to 12 months following infusion.
- Number of Participants who Experience Dose Limiting Toxicities (DLTs) [ Time Frame: Up to Day 42 ]
A dose limiting toxicity (DLT) is any of the following and is determined by the investigator to be related to study drug:
- Aplastic bone marrow, defined by marrow cellularity <5% at Day 28 in the absence of residual disease and confirmed at Day 42.
- Treatment-emergent CRS of Grade 4 that does not resolve to Grade ≤2 within 72 hours, despite optimal treatment;
- Treatment-emergent CRS of Grade 3 that does not resolve to Grade ≤2 within 2 weeks, despite optimal treatment;
- Treatment-related Grade 4-5 allergic reactions
- Treatment-related non-reversible Grade 3 (< 14 days), or any Grade 4-5 autoimmune reactions related to the study cell infusion.
- Central neurologic toxicity Grade ≥3 lasting more than 14 days
- Grade 5 Cytokine Release Syndrome (CRS)
- Tumor Lysis Syndrome ≥ IV (Cairo and Bishop, 2004) that does not resolve within 7 days
- Number of Participants who Experience Treatment Related Adverse Events (AEs) [ Time Frame: Up to 12 months post treatment ]Systemic toxicity in general and hematologic toxicity in specific will be assessed through the capture of AEs at each study visit and through laboratory assessments throughout the study. The severity of the AEs will be assessed using the Common Terminology Criteria for Adverse Events (CTCAE) v 5.0 scale.
- Disease Progression in AML Participants [ Time Frame: Up to 15 years ]Proportion of AML patients achieving partial response (PR), complete response (CR), and/or morphologic leukemia free state (MLFS) by ELN Response Criteria in AML. CRh (complete PRGN-3006 T cells in patients with AML/MDS remission with partial hematological recovery) will also be captured, defined as <5% of blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts
- Disease Response in MDS Patients [ Time Frame: Up to 15 years ]Proportion of MDS patients achieving a response (CR, PR or Marrow CR) as defined in International Working Group (IWG) 2006 Criteria.
- Rate of Absolute Neutrophil Count Recovery [ Time Frame: Day 28 ]Rate of Absolute Neutrophil count recovery (>0.5 x 10^9/L)
- Absolute Lymphocyte Count (ALC) [ Time Frame: Baseline ]ALC including CD4/CD8 subsets by flow cytometry at baseline (at apheresis) in patients who have successful versus failed PRGN-3006 production.
- Number of PRGN-3006 T Cells [ Time Frame: Up to 12 months post treatment ]Number of PRGN-3006 T Cells present in patients treated with PRGN-3006
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): NCT03927261
|United States, Florida|
|H Lee Moffitt Cancer Center and Research Institute||Recruiting|
|Tampa, Florida, United States, 33612|
|Contact: Jeffrey Edelman 813-745-1040 Jeffrey.Edelman@moffitt.org|
|Principal Investigator: David A Sallman, MD|
|Sub-Investigator: Marco Davilla, MD, PhD|
|Sub-Investigator: Rami Komrokji, MD|
|Sub-Investigator: Jeffrey Lancet, MD|
|Sub-Investigator: Alan List, MD|
|Sub-Investigator: Kathy McGraw, PhD|
|Sub-Investigator: Eric Padron, MD|
|Sub-Investigator: Kendra Sweet, MD|
|Principal Investigator:||David A Sallman, MD||H. Lee Moffitt Cancer Center and Research Institute|