NCT01570283

Brief Summary

The subjects eligible for this trial have a type of blood cell cancer, other blood disease or a genetic disease for which they will receive a stem cell transplant. The donor of the stem cells will be either the subject's brother or sister, or another relative, or a closely matched unrelated donor. The Investigators are asking subjects to participate in this study which tests if blood cells from the subject's donor that have been grown in a special way, can prevent or be a effective treatment for early infection by five viruses - Epstein Barr virus (EBV), cytomegalovirus (CMV), adenovirus, BK virus (BKV) and human herpes virus 6 (HHV6). The Investigators have grown T cells from the subject's stem cell donor in the laboratory in a way that will train them to recognize the viruses and control them when the T cells are given after a transplant. This treatment with specially trained T cells (also called cytotoxic T cells or "CTLs") has had activity against three of these viruses (CMV, EBV and Adenovirus) in previous studies. In this study the Investigators want to see if they increase the number of viruses that can be targeted to include BKV and HHV6 using a simple and fast approach to make the cells. The Investigators want to see if they can use a kind of white blood cell called T lymphocytes (or T cells) to prevent and treat adenovirus, CMV, EBV, BKV and HHV6 in the early stages of reactivation or infection.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Sep 2012

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 27, 2012

Completed
8 days until next milestone

First Posted

Study publicly available on registry

April 4, 2012

Completed
5 months until next milestone

Study Start

First participant enrolled

September 1, 2012

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2016

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2017

Completed
1.7 years until next milestone

Results Posted

Study results publicly available

May 30, 2019

Completed
Last Updated

May 30, 2019

Status Verified

April 1, 2019

Enrollment Period

3.9 years

First QC Date

March 27, 2012

Results QC Date

December 3, 2018

Last Update Submit

May 9, 2019

Conditions

Keywords

post allogeneic hematopoietic stem cell transplantcytotoxic T lymphocytesCytomegalovirusCMVadenovirusEpstein-Barr virusEBVHuman polyomavirus type IBK virusHuman herpesvirus 6HHV6

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With a DLT

    DLT is defined as acute GvHD grades III-IV within 42 days of the last dose of CTLs, # of patients with Grade 3-5 infusion-related adverse events within 30 days of the last dose of CTLs, and # of patients with Grade 4-5 non-hematological adverse events within 30 days of the last dose of CTLs. GVHD grade III-IV scoring is based on the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) GVHD scoring stamp or equivalent. Grade 3-5 infusion-related adverse events and Grade 4-5 non-hematological adverse events are graded by the NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 4.X.

    42 days

Secondary Outcomes (3)

  • Percentage of Patients Who Have a Response in Anti-viral Activity

    42 days

  • Percentage Change of Viral Load From Baseline to Follow-up

    3 months

  • Median Peak Frequency of Specific T Cells Post-infusion

    3 months

Study Arms (6)

Multivirus-specific T cells 5*10^6 mCTLs/m2 for Prophylaxis

EXPERIMENTAL

Cohort 1 prophylaxis: Participants were administrated 5\*10\^6 mCTLs/m multivirusspecific T cells intravenously for prophylaxis of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 1

Multivirus-specific T cells 5*10^6 mCTLs/m2 for Treatment

EXPERIMENTAL

Cohort 1 treatment: Participants were administrated 5\*10\^6 mCTLs/m multivirusspecific T cells intravenously for treatment of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 1

Multivirus-specific T cells 1*10^7 mCTLs/m2 for Prophylaxis

EXPERIMENTAL

Cohort 2 prophylaxis: Participants were administrated 1\*10\^7 mCTLs/m multivirusspecific T cells intravenously for prophylaxis of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 2

Multivirus-specific T cells 1*10^7 mCTLs/m2 for Treatment

EXPERIMENTAL

Cohort 2 treatment: Participants were administrated 1\*10\^7 mCTLs/m multivirusspecific T cells intravenously for treatment of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 2

Multivirus-specific T Cells 2*10^7 mCTLs/m2 for Prophylaxis

EXPERIMENTAL

Cohort 3 prophylaxis: Participants were administrated 2\*10\^7 mCTLs/m multivirusspecific T cells intravenously for prophylaxis of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 3

Multivirus-specific T Cells 2*10^7 mCTLs/m2 for Treatment

EXPERIMENTAL

Cohort 3 treatment: Participants were administrated 2\*10\^7 mCTLs/m multivirusspecific T cells intravenously for treatment of EBV, CMV, Adenovirus, HHV6 and BK virus infections post allogeneic stem cell transplant.

Biological: Multivirus-specific T cells Dose Level 3

Interventions

The feasibility and safety of 3 different dose levels will be evaluated and will determine the maximum tolerated dose (MTD) level. Dose Level One: 5x10\^6 mCTLs/m2 There may be an option of administering 2 additional doses (at the same level the patient was receiving), 28 days after the first dose, in subjects that have a partial response after one dose or who receive other therapy that may affect the persistence or function of the infused CTL.

Multivirus-specific T cells 5*10^6 mCTLs/m2 for ProphylaxisMultivirus-specific T cells 5*10^6 mCTLs/m2 for Treatment

The feasibility and safety of 3 different dose levels will be evaluated and will determine the maximum tolerated dose (MTD) level. Dose Level Two: 1x10\^7 mCTLs/m2 There may be an option of administering 2 additional doses (at the same level the patient was receiving), 28 days after the first dose, in subjects that have a partial response after one dose or who receive other therapy that may affect the persistence or function of the infused CTL

Multivirus-specific T cells 1*10^7 mCTLs/m2 for ProphylaxisMultivirus-specific T cells 1*10^7 mCTLs/m2 for Treatment

The feasibility and safety of 3 different dose levels will be evaluated and will determine the maximum tolerated dose (MTD) level. Dose Level Three: 2x10\^7 mCTLs/m2 There may be an option of administering 2 additional doses (at the same level the patient was receiving), 28 days after the first dose, in subjects that have a partial response after one dose or who receive other therapy that may affect the persistence or function of the infused CTL

Multivirus-specific T Cells 2*10^7 mCTLs/m2 for ProphylaxisMultivirus-specific T Cells 2*10^7 mCTLs/m2 for Treatment

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients will be eligible following any type of allogeneic transplant to receive CTLs as prevention or for early reactivations as defined below.
  • Prior myeloablative or non-myeloablative allogeneic hematopoietic stem cell transplant using either bone marrow or peripheral blood stem cells within 12 months.
  • Prevention for patients at risk of CMV, adenovirus, EBV, BK virus, or HHV6 infection
  • Treatment of reactivation or infection which is defined for each virus as below
  • CMV- CMV antigenemia is monitored at least weekly post transplant. Reactivation is defined at CMV antigenemia with \<10 leukocytes positive or elevated PCR. If any patient develops CMV antigenemia with \>10 leukocytes positive or clinical evidence of CMV infection (defined as the demonstration of CMV by biopsy specimen from visceral sites (by culture or histology) either pre or after CTL infusions, standard treatment with Ganciclovir, and/or Foscarnet and Immunoglobulins will be initiated. Patients may receive CTLs for antigenemia or elevated PCR without visceral infection.
  • Adenovirus- Adenovirus infection will be defined as the presence of adenoviral positivity as detected by PCR or culture from ONE site such as stool or blood or urine or nasopharynx.
  • Adenovirus disease will be defined as the presence of adenoviral positivity as detected by culture from more than two sites such as stool or blood or urine or nasopharynx.
  • In patients who meet the criteria for disease Cidofovir may be added unless the subject could not tolerate this agent due to nephrotoxicity. Patients may receive CTLs for elevated PCR in blood or stool.
  • EBV- EBV-LPD is defined according to recent guidelines as proven EBV-LPD defined by biopsy or probable EBV-LPD defined as an elevated EBV DNA level associated with clinical symptoms (adenopathy or fever or masses on imaging) but without biopsy confirmation. Patients with EBV DNA reactivation only may receive CTLs on study. Patients with proven or probable EBV-LPD should also receive Rituxan
  • BK virus- Patients post transplant may develop asymptomatic BKV viruria or viremia. BK reactivation will be defined as detection of elevated BK levels by PCR in blood or urine while disease will be defined as detection in multiple sites or in one site with symptoms. Cidofovir has been administered intravenously in a low dose (i.e. up to 1 mg/kg 3 times weekly, without probenecid) or a high dose (ie, 5 mg/kg per week with probenecid) to HSCT patients with BK infections but no randomized trials are available proving its clinical efficacy. In patients who meet the criteria for disease Cidofovir may be added unless the subject could not tolerate this agent due to nephrotoxicity.
  • HHV6- HHV6 reactivation will be defined as detection of elevated HHV6 levels by PCR in blood while disease will be defined as detection in multiple sites or in one site with symptoms. Ganciclovir, Cidofovir, and foscarnet have variable in vitro activity against HHV-6, and may have a role in treating HHV-6-associated disease - hence one or more of these agents will be added in patients with disease.
  • Treatment may be given to eligible patients with a single or multiple infections. Patients with multiple infections with one or more reactivation and one or more controlled infection are eligible to enroll.
  • Clinical status at enrollment to allow tapering of steroids to less than 0.5 mg/kg/day prednisone.
  • Karnofsky/Lansky score of ≥ 50
  • ANC greater than 500/µL.
  • +8 more criteria

You may not qualify if:

  • Patients receiving ATG, or Campath or other immunosuppressive T cell monoclonal antibodies within 28 days of screening for enrollment.
  • Patients with other uncontrolled infections. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. For fungal infections patients must be receiving definitive systemic anti-fungal therapy and have no signs of progressing infection for 1 week prior to enrollment.
  • Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
  • Patients who have received donor lymphocyte infusion (DLI) within 28 days.
  • Patients with active acute GVHD grades II-IV.
  • Active and uncontrolled relapse of malignancy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Papadopoulou A, Gerdemann U, Katari UL, Tzannou I, Liu H, Martinez C, Leung K, Carrum G, Gee AP, Vera JF, Krance RA, Brenner MK, Rooney CM, Heslop HE, Leen AM. Activity of broad-spectrum T cells as treatment for AdV, EBV, CMV, BKV, and HHV6 infections after HSCT. Sci Transl Med. 2014 Jun 25;6(242):242ra83. doi: 10.1126/scitranslmed.3008825.

MeSH Terms

Conditions

Virus DiseasesAdenoviridae InfectionsEpstein-Barr Virus Infections

Condition Hierarchy (Ancestors)

InfectionsDNA Virus InfectionsHerpesviridae InfectionsTumor Virus Infections

Results Point of Contact

Title
Dr. Helen Heslop
Organization
Center for Cell and Gene Therapy

Study Officials

  • Helen Heslop, MD

    Baylor College of Medicine

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

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

Study Record Dates

First Submitted

March 27, 2012

First Posted

April 4, 2012

Study Start

September 1, 2012

Primary Completion

August 1, 2016

Study Completion

September 1, 2017

Last Updated

May 30, 2019

Results First Posted

May 30, 2019

Record last verified: 2019-04

Locations