A Safety Study of Human Cord Blood Derived, Culture-expanded, Natural Killer Cell (PNK-007) Infusion With or Without Subcutaneous Recombinant Human Interleukin-2 (rhIL-2) Following Autologous Stem Cell Transplant for Multiple Myeloma (MM)

NCT02955550 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: CCT-PNK-007-MM-001
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 7

Brief Summary

This study will find the highest acceptable treatment dose and timing of infusion of cord blood, culture expanded natural killer (NK) cells, a kind of immune cell, in patients with multiple myeloma. The NK cells will be given at varying days post autologous stem cell transplant. rhIL-2 is administered after treatment to help the NK cells expand in the body. The safety of this treatment will be studied and researchers want to learn if NK cells will help in treating multiple myeloma.

Conditions

Multiple Myeloma

Keywords

Multiple Myeloma; Plasma Cell Myeloma; Plasma Cell Neoplasm; Relapsed/refractory Myeloma; PNK-007; Recombinant Human IL-2 (RHIL-2); Human IL-2; Bone Marrow Transplant

Outcome Measures

Primary Outcomes (4)

• Dose-Limiting Toxicity (DLT)

  Number and severity of adverse events within 28 days of administration

  Up to 28 days

• Maximum Tolerated Dose (MTD)

  The maximum dose safely administered for the treatment of patients with multiple myeloma

  Up to 28 days

• Dose Timing After Autologous Stem Cell Transplant

  The optimal dose timing safely administered for the treatment of patients with multiple myeloma post ASCT

  Up to 28 days

• Adverse Events (AEs)

  Number and severity of adverse events within 12 months of administration

  Up to 12 months

Secondary Outcomes (1)

• Response Rate

  Up to day 100

Study Arms (1)

PNK-007 and rhIL-2

EXPERIMENTAL

Melphalan per institutional practices (within Day -5 to 01), ASCT (Day 0), PNK-007 at varying dose levels (within Day 7 to Day 14) and rhIL-2 every other day starting day of PNK-007 administration.

Drug: rhIL-2; Biological: PNK-007

Interventions

rhIL-2 DRUG

Human recombinant Interleukin-2

PNK-007 and rhIL-2

PNK-007 BIOLOGICAL

PNK-007 is a culture-expanded cell population derived from human cord blood hematopoietic stem/progenitor cells.

PNK-007 and rhIL-2

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must satisfy the following criteria to be enrolled in the study:
• Subject has eligible disease status:
• Newly diagnosed and are undergoing induction therapy prior to undergoing first Autologous stem cell transplant (ASCT) or
• Myeloma patients with prior relapse undergoing first ASCT. or
• Myeloma patients with relapsed disease after first ASCT who are undergoing second ASCT. Subjects must have achieved at least a partial response (PR) prior to proceeding to ASCT.
• Subject is \> 18 and ≤ 70 years of age at the time of signing the informed consent form (ICF).
• Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
• Subject is willing and able to adhere to the study schedule and other protocol requirements.
• Performance status of Karnofsky performance status ≥ 70% or Eastern Cooperative Oncology Group (ECOG) \< 2
• Ability to be off immunosuppressive drugs for at least 3 days prior to the PNK-007 cell infusion. Steroids at the equivalent of no more than 5 mg prednisone per day are permissible.
• Be a candidate for ASCT based on institutional practices.
• Subjects must have autologous peripheral blood stem cell graft available in storage for additional transplant in the event of engraftment failure.
• Female of childbearing potential (FCBP) must:
• Have two negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after the end of study treatment. This applies even if the subject practices true abstinence\* from heterosexual contact.
• Either commit to true abstinence\* from heterosexual contact (which must be reviewed at applicable study visits and source documented) or agree to use, and be able to comply with, effective contraception without interruption, during the study therapy (including dose interruptions), and for 28 days after discontinuation of PNK-007.

You may not qualify if:

• The presence of any of the following will exclude a subject from enrollment:
• Subject has plasma cell leukemia.
• Subject has non-secretory myeloma.
• Subject has previously undergone allogeneic stem cell transplant.
• Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
• Subject has any condition including the presence of laboratory abnormalities which places the subject at unacceptable risk if he or she were to participate in the study.
• Subject has any condition that confounds the ability to interpret data from the study.
• Subject has a body weight exceeding 120 kg.
• Subject has aspartate aminotransferase (AST), alanine aminotransferase (ALT), or alkaline phosphatase ≥ 2.5 x the upper limit of normal (ULN) at screening.
• Estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m2 at screening calculated using the Modification of Diet in Renal Disease Study equation.
• Subject has a bilirubin level \> 2 mg/dL (unless subject has known Gilbert's disease) at screening.
• Subject has had prior treatment with biologic antineoplastic agents no less than 7 days before PNK-007 infusion and at least 5 half-lives. For agents that have known AEs occurring beyond 7 days after administration (ie, monoclonal antibodies), this period must be extended beyond the time during which acute AEs are known to occur.
• Subject is pregnant or breastfeeding.
• Subject has new or progressive pulmonary infiltrates or pleural effusion large enough to be detected by chest x-ray or computed tomography (CT) scan.
• Subject has active autoimmune disease other than controlled connective tissue disorder or those who are not on active therapy.

Sponsors & Collaborators

• Celularity Incorporated: lead

Study Sites (7)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington Univ School of Medicine Siteman Cancer Center

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198-6805, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Mt. Sinai School of Medicine

New York, New York, 10029, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Multiple Myeloma; Neoplasms, Plasma Cell

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Solveig Ericson, MD

  Celularity Incorporated

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2016
• First Posted: November 4, 2016
• Study Start: January 5, 2017
• Primary Completion: July 10, 2018
• Study Completion: June 4, 2019
• Last Updated: July 22, 2020

Record last verified: 2020-07

Locations

US (7)