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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/19/2021.

PRGN-3006 Adoptive Cellular Therapy Relapsed or Refractory AML or High Risk MDS

Clinicaltrials.gov identifier NCT03927261

Recruitment Status Recruiting

First Posted April 25, 2019

Last update posted September 23, 2020

Study Description

Brief summary:

This study is to determine the safety and best dose of PRGN-3006 T Cells

  • Condition or Disease:Myelodysplastic Syndromes
    Acute Myeloid Leukemia
  • Intervention/Treatment: Drug: PRGN-3006 T Cells
  • Phase: Phase 1
Detailed Description

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 Design
  • Study Type: Interventional
  • Estimated Enrollment: 56 participants
  • Intervention Model: Single Group Assignment
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • 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 2019
  • Estimated Primary Completion Date: May 2022
  • Estimated Study Completion Date: May 2025
Arms and interventions
Arm Intervention/treatment
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.
Outcome Measures
  • Primary Outcome Measures: 1. 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[2]) that does not resolve within 7 days
  • 2. 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.
  • Secondary Outcome Measures: 1. 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
  • 2. 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.
  • 3. Rate of Absolute Neutrophil Count Recovery [ Time Frame: Day 28 ]
    Rate of Absolute Neutrophil count recovery (>0.5 x 10^9/L)
  • 4. 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.
  • 5. 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
Eligibility Criteria
  • Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Criteria

Inclusion Criteria:

- Participants must be diagnosed with either relapsed or refractory AML or higher risk
MDS

- Absolute lymphocyte count ≥ 0.2 k/μL.

- Karnofsky performance status score ≥60%.

- Life expectancy ≥ 12 weeks from the time of enrollment.

- Pretreatment calculated or measured creatinine clearance (absolute value) of ≥ 40
mL/minute or Cr > 2x upper limit of normal (ULN).

- Serum bilirubin ≤ 2.0 mg/dL or total bilirubin ≤ 3.0 x IULN with direct bilirubin
within normal range in participants with well documented Gilbert's syndrome or
hemolysis or who require regular blood transfusions

- Alanine aminotransferase (AST) and aspartate aminotransferase (ALT) 45%.

- Participant does not require supplemental oxygen or mechanical ventilation AND has an
oxygen saturation by pulse oximetry of ≥ 92% or higher on room air.

- Negative serum pregnancy test. Note: Women of child-bearing potential and men must
agree to use adequate contraception prior to study entry and for at least 1 year
following study treatment (T cell infusion); should a woman participant or female
partner of a male participant become pregnant or suspect that she is pregnant while
participating on the trial, she should inform her treating physician immediately.

- Participant has a matched bone marrow donor and is otherwise able to receive a bone
marrow transplant (dose escalation part only)

- Participants who have undergone allo-SCT are eligible if they are at least 3 months
post SCT, have relapsed AML/MDS as defined above, are not on treatment or prophylaxis
for GVHD for at least 6 weeks before administration of CAR T cells, and have no active
GVHD.

- All participants must have the ability to understand and willingness to sign a written
informed consent.

Exclusion Criteria:

- Diagnosis of acute promyelocytic leukemia (APL M3): t(15;17)(q22;q12); (promyelocytic
leukemia [PML]/retinoic acid receptor [RAR] alpha [a]) and variants excluded.

- Participants with extramedullary disease as their sole site of relapsed AML.

- Known central nervous system (CNS) leukemic involvement that is refractory to
intrathecal chemotherapy and/or cranio-spinal radiation; participants with a history
of CNS disease that have been effectively treated to complete remission ( i.e. no
blasts in cerebrospinal fluid [CSF] by cytology and flow cytometry) will be eligible.

- Prior treatment with investigational CAR T therapy for any disease.

- Participants enrolled in another investigational therapy protocol for AML within 14
days or 5 half-lives of enrollment, whichever is shorter.

- Ongoing uncontrolled serious infection, symptomatic congestive heart failure, unstable
angina pectoris, uncontrolled cardiac arrhythmia, poorly controlled pulmonary disease
or psychiatric illness/social situations that would limit compliance with study
requirements.

- Human immunodeficiency virus (HIV) seropositivity, or active hepatitis B or C
infection based on testing performed within 28 days of enrollment.

- Participants requiring agents other than hydroxyurea to control blast counts within 14
days of study enrollment.

- Participants with presence of other active malignancy within 1 year of study entry;

- Participants with adequately resected basal or squamous cell carcinoma of the skin, or
adequately resected carcinoma in situ (e.g. cervix) may enroll irrespective of the
time of diagnosis.

- Pregnant and lactating women are excluded from this study

- History of allergic reactions attributed to compounds of similar chemical or
biological composition to cetuximab (anti-EGFR).

- Active autoimmune disease requiring systemic immunosuppressive therapy (i.e. >10mg of
prednisone daily or equivalent).

- Participant, who in the opinion of the investigator, may not be able to comply with
the safety monitoring requirements of the study.

Contacts and Locations
Contacts
Locations

United States, Florida
H Lee Moffitt Cancer Center and Research Institute
Tampa

Sponsors and Collaborators

H. Lee Moffitt Cancer Center and Research Institute

Precigen, Inc

Investigators

Principal Investigator: David A Sallman, MD H. Lee Moffitt Cancer Center and Research Institute

Principal Investigator: Nelli Bejanyan, MD Moffitt Cancer Center

More Information
  • Responsible Party: H. Lee Moffitt Cancer Center and Research Institute
  • ClinicalTrials.gov Identifier: NCT03927261 History of Changes
  • Other Study ID Numbers: MCC-19862
  • First Posted: April 25, 2019 Key Record Dates
  • Last Update Posted: September 23, 2020
  • Last Verified: September 2020
  • Individual Participant
    Data (IPD) Sharing
    Statement:
  • Plan to Share IPD: Undecided
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Keywords provided by H. Lee Moffitt Cancer Center and Research Institute: AML MDS
  • Additional relevant MeSH terms: Leukemia
    Leukemia, Myeloid
    Leukemia, Myeloid, Acute
    Preleukemia
    Myelodysplastic Syndromes