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Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency

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ClinicalTrials.gov Identifier: NCT02860559
Recruitment Status : Not yet recruiting
First Posted : August 9, 2016
Last Update Posted : April 11, 2019
Sponsor:
Information provided by (Responsible Party):
Taiga Biotechnologies, Inc.

Brief Summary:

This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID).

The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability of the donor cells to make blood and immune cells after transplant. Exposure of the donor cells to this protein does not modify the genes in the cells in any way.

This study has two goals. The first goal is to find out if transplant with TBX-1400 is safe. The second goal is to find out what effects TBX-1400 stem cells have on time to engraftment in pediatric subjects with SCID. The study hypothesis is that TBX-1400 cells will shorten the time to immune reconstitution after transplant.


Condition or disease Intervention/treatment Phase
Severe Combined Immunodeficiency Biological: TBX-1400 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
Estimated Study Start Date : September 2019
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : March 2023


Arm Intervention/treatment
Experimental: TBX-1400 treatment
Single intravenous infusion of TBX-1400
Biological: TBX-1400
Hematopoietic stem cells transplantation




Primary Outcome Measures :
  1. Adverse Events following transplant with TBX-1400 [ Time Frame: Two years ]
    Adverse events from subject or parent reporting or other assessments


Secondary Outcome Measures :
  1. Transplant Engraftment [ Time Frame: Up to Day 180 ]
    Assessment of transplant engraftment will include analysis of T-cells , B-cells and Natural Killer cells

  2. Chimerism [ Time Frame: Up to Day 180 ]
    Assessment of chimerism will include analysis of T-cells , B-cells and Natural Killer cells

  3. Absolute numbers of T-cells [ Time Frame: Days 30 to 360 ]
  4. T-cell receptor excision circles (TREC) [ Time Frame: Days 30 to 360. ]
  5. Kappa-deleting recombination excision circles (KREC) [ Time Frame: Days 30 to 360. ]
  6. Immunoglobulin (Ig) levels [ Time Frame: Days 30 to 360. ]
  7. Immunoglobulin G (IgG) titers to pneumococcal antigens in 13-valent vaccine [ Time Frame: Sixty days after final immunization ]
  8. T-cell responses to anti-CD3 and phytohemagglutinin (PHA) [ Time Frame: Days 30, 60, 90, 120, and 180. ]
  9. Number of infections following transplant [ Time Frame: Two years ]
  10. Number of days granulocyte colony stimulating factor (G-CSF) was administered [ Time Frame: Up to 1 year ]
  11. Number of days to specific cell counts and last packed red blood cell (PRBC) transfusion [ Time Frame: Up to 2 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month to 4 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed informed consent of the subject's legally authorized representative (in most cases, this will be the parent or parents),
  • Age 1 month to 4 years,
  • SCID, leaky SCID with <100 TRECs, or Omenn syndrome requiring stem cell transplant with conditioning therapy (patients with decreased T-cell numbers by flow cytometry, decreased TREC, and decreased in vitro responses to T cell mitogens will be eligible regardless of B-cell and/or natural killer (NK) cell function),
  • Identified donor (9 or 10/10 Human Leukocyte Antigen (HLA)-matched unrelated or haplocompatible relative),
  • Eligible patients must have adequate physical function to tolerate the conditioning regimen and hematopoietic stem cell transplantation (HSCT), as measured by:

    • Renal function: serum creatinine ≤3x upper limit of normal for age,
    • Hepatic function: adequate synthetic function as indicated by a serum fibrinogen at or above the normal limit for the child's age,
    • Cardiac function: fractional shortening ≥30% as determined by echocardiography. (For subjects with a fractional shortening value of exactly 30%, if conditioning is delayed for any reason, a repeat echocardiogram is to be performed before the conditioning regimen is initiated to confirm the subject's continued eligibility for participation in the study.)

Exclusion Criteria:

  • Lack of investigational review board (IRB) approval of the study at the treating institution,
  • Lack of consent by the child's legal guardians (Israeli law requires consent by both parents),
  • Adenosine deaminase (ADA) deficiency,
  • The patient has a brother/sister who is a matching and available donor and who was approved to be a donor in accordance with the law and regulations,
  • End-stage organ failure that precludes ability to tolerate the transplant procedure or conditioning,
  • Serum creatinine >3 times upper limit of normal for age,
  • Inadequate cardiac function, i.e., fractional shortening ≥30% as determined by echocardiography (for subjects with a fractional shortening value of exactly 30%, if conditioning is delayed for any reason, a repeat echocardiogram must be performed to confirm the subject's eligibility for participation in the study),
  • Inadequate hepatic synthetic function indicated by serum fibrinogen below normal for the child's age or signs of hepatic failure,
  • Major congenital abnormalities that adversely affect survival,
  • Expected survival <4 weeks despite transplant.

The following are NOT exclusion criteria:

  • The administration of supplemental oxygen,
  • The presence of infection per se, because patients with SCID frequently have infections with routine pathogens as well as opportunistic infections. Antibiotic, antifungal and antiviral prophylaxis therapy will be used as clinically indicated. Because transplantation is required for control of infections, subjects may be enrolled in the study even though infection is present although acute infections should be controlled prior to initiating transplant conditioning. Adjudication of controlled infection will be performed by the physician(s) treating the patient together with the clinical Principal Investigator of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02860559


Contacts
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Contact: Yosef Refaeli, Dr. +1-720-859-3547 refaeli@taigabiotech.com
Contact: Brian Turner, Dr. +1-720-859-3547 turner@taigabiotech.com

Locations
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Israel
Hadassah Medical Center (Ein Kerem site) Not yet recruiting
Jerusalem, Israel
Contact: Polina Stepensky, Dr.    +972-2-6779095    polina@hadassah.org.il   
Contact: Hila Yosef    +972-2-6777511    hilay@hadassah.org.il   
Principal Investigator: Polina Stepensky, Dr.         
Schneider Children's Medical Center Not yet recruiting
Petach Tikva, Israel, 4920235
Contact: Jerry Stein, Dr.    +972-3-9253657    jstein@clalit.org.il   
Contact: Shoshana Hanimov    +972-3-9253507    shoshanaha1@clalit.org.il   
Principal Investigator: Jerry Stein, Dr.         
Sponsors and Collaborators
Taiga Biotechnologies, Inc.

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Responsible Party: Taiga Biotechnologies, Inc.
ClinicalTrials.gov Identifier: NCT02860559     History of Changes
Other Study ID Numbers: TBX-1400-001
First Posted: August 9, 2016    Key Record Dates
Last Update Posted: April 11, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Taiga Biotechnologies, Inc.:
Hematopoietic Stem Cell Transplantation
Severe Combined Immunodeficiency
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases