Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs)

NCT04197596 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: NYMC 590
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 7

Brief Summary

BK cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be safe and effective in decreasing specific viral load in children, adolescents and young adults (CAYA) with refractory BK infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) or with primary immunodeficiencies (PID).

Conditions

Viral Infection; Primary Immune Deficiency Disorder

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Patients will be monitored for adverse events following each CTL infusion

  12 weeks

Study Arms (1)

BK CTL

EXPERIMENTAL

Eligible patients with refractory BK infection will receive up to 20 infusions of BK CTLs that are donor derived.

Biological: BK CTL

Interventions

BK CTL BIOLOGICAL

Patient with refractory BK infection and a HLA Matched Related Donors: BK specific CTLs (2.5 x 104 CD3/kg) infused intravenously on day 0 and may be additionally reinfused at a minimum of every two weeks (depending on safety and efficacy) for a maximum of twenty total infusions (maximum 12.5 x 104 CD3/kg). Patients with refractory BK virus and a HLA Mismatched Related Donors: BK specific CTLs (0.5x104 CD3/kg) infused intravenously on day 0 and may additionally be reinfused at a minimum of every two weeks (depending on safety and efficacy) for a maximum of five total infusions (maximum 2.5 x 104 CD3/kg).

BK CTL

Eligibility Criteria

• Age: 1 Month - 79 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with refractory BK infection post allogeneic HSCT, post solid organ transplantation or with primary immunodeficiencies with either
• Increasing urine and/or plasma BK RT-PCR DNA (by 1 log) after 7 days or persistent quantitative qRT-PCR DNA copies after 14 days despite two weeks of appropriate anti-viral therapy AND/OR
• Medical intolerance to anti-viral therapies including:
• renal toxicity with cidofovir or other \> grade 2 toxicities secondary to cidofovir And/or
• known resistance to cidofovir 1.2. Consent: Written informed consent given (by patient or legal representative) prior to any study-related procedures.
• Performance Status \> 30% (Lansky \< 16 yrs and Karnofsky \> 16 yrs) 1.4 Age: 0.1 to 79.99 years 1.5 Females of childbearing potential with a negative urine pregnancy test

You may not qualify if:

• Patient with acute GVHD \> grade 2 or extensive chronic GVHD at the time of BK CTL infusion
• Patient receiving steroids (\>0.5 mg/kg prednisone equivalent) at the time of BK CTL infusion
• Patient treated with donor lymphocyte infusion (DLI) within 4 weeks prior to BK CTL infusion
• Thymoglobulin (ATG) or Alemtuzumab within 30 days
• Patient with poor performance status determined by Karnofsky (patients \>16 years) or Lansky (patients ≤16 years) score ≤30%
• Concomitant enrollment in another experimental clinical trial investigating the treatment of refractory BK infection.
• Any medical condition which could compromise participation in the study according to the investigator's assessment
• Known HIV infection
• Female patient of childbearing age who is pregnant or breast-feeding or not willing to use an effective method of birth control during study treatment.
• Known hypersensitivity to iron dextran
• Patients unwilling or unable to comply with the protocol or unable to give informed consent.
• Known human anti-mouse antibodies

Sponsors & Collaborators

• New York Medical College: lead
• Children's Hospital of Philadelphia: collaborator
• Medical College of Wisconsin: collaborator
• Nationwide Children's Hospital: collaborator
• Johns Hopkins University: collaborator
• University of California, San Francisco: collaborator

Study Sites (7)

University of California San Francisco

San Francisco, California, 94158, United States

RECRUITING

Johns Hopkins

Baltimore, Maryland, 21287, United States

RECRUITING

Washington University

St Louis, Missouri, 63130, United States

RECRUITING

New York Medical College

Vallhala, New York, 10595, United States

RECRUITING

Nationwide Children's Hosptial

Columbus, Ohio, 43205, United States

RECRUITING

Children's Hospital of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

MeSH Terms

Conditions

Virus Diseases; Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Infections; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Study Officials

• Mitchell S Cairo, MD

  New York Medical College

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mitchell S Cairo, MD

CONTACT

9145942150; mitchell_cairo@nymc.edu

Lauren Harrison

CONTACT

16172857844; lauren_harrison@nymc.edu

Study Design

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

Study Record Dates

• First Submitted: December 11, 2019
• First Posted: December 13, 2019
• Study Start: July 1, 2020
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: April 15, 2026

Record last verified: 2026-04

Locations

US (7)