Multivirus-specific Cytotoxic T Lymphocytes (mCTL)

Completed · Phase 1

Conditions studied: Refractory Viral Infections

In brief

PIDD represent an expanding group of genetic disorders that compromise immunity against bacteria, viruses, and fungi. The most severe forms of PIDD cause profound susceptibility to opportunistic infections due to impaired or absent T-cell immunity. These diseases can be rapidly fatal unless treated via hematopoietic stem cell transplantation (HSCT). Chronic viral illnesses are a common presenting feature of many of these disorders, and studies have shown that survival of HSCT is profoundly impacted by the patient's pre-transplant disease status. Primary infections with viruses such as cytomegalovirus (CMV) and Epstein-Barr virus (EBV) are common, and respiratory viruses such as adenovirus also frequently cause infection. In patients with severe combined immunodeficiency (SCID), a prior study identified these viruses as the most common causes of mortality in the immediate period following HSCT. Though some forms of PIDD are amenable to HSCT without requiring conditioning chemotherapy, many forms require a variable degree of pre-conditioning to ensure that stable engraftment of the donor cells is achieved. The administration of cytotoxic chemotherapy used in the conditioning regimens can however increase the risk for regimen related toxicity and for some patients (especially those with active viral infections) this risk is particularly high, leading to high treatment related mortality rates. For these reasons, many such patients are not even considered candidates for HSCT or are delayed getting to HSCT and ultimately succumb to infection before they can receive the transplant. The primary objective of this study is to determine the safety of administering third-party multivirus-specific cytotoxic T lymphocytes (mCTL) from adult CMV seropositive donors to treat refractory viral infections in patients with primary immunodeficiency disorders (PIDD) prior to hematopoietic stem cell transplantation (HSCT).

Key facts

Study ID

NCT02510404

Run by

Catherine Bollard

People needed

1

Starts

2014-04-01

Expected to finish

2019-06-08

Last updated by the study team

2020-01-18

Who can join

Age: 0 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of primary immunodeficiency with established plan to undergo myeloablative or non-myeloablative allogeneic hematopoietic stem cell transplant for treatment thereof or diagnosis of a form of primary immunodeficiency for which hematopoietic stem cell transplantation is not indicated.
• Active infection with EBV, CMV, and/or Adenovirus, unable to be successfully controlled with standard therapy.
• Steroids less than 0.5 mg/kg/day prednisone
• Karnofsky/Lansky score of ≥ 50
• ANC greater than 500/µL.
• Bilirubin <2x, AST <3x, Serum creatinine <2x upper limit of normal, Hgb >8.0
• Pulse oximetry of > 90% on room air
• Negative pregnancy test (if female of childbearing potential)
• Patient or parent/guardian capable of providing informed consent.

You may not qualify if…

• Patients with other uncontrolled infections (see 2.3.2 for definitions)
• Patients who received ATG, Campath, or other T cell immunosuppressive monoclonal antibodies in the last 28 days
• Received donor lymphocyte infusion in last 28 days
• Diagnosis of Omenn's syndrome or MHC class I deficiency
• Active and uncontrolled malignancy
• Pregnant or lactating
• Unable to wean steroids to ≤0.5 mg/kg/day prednisone.
• Patients with Grade 3 hyperbilirubinemia

Where it is running

• Childrens National Medical Center — Washington D.C., District of Columbia, United States

Full record on ClinicalTrials.gov

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