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

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Brief Title

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

Official Title

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

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.
    


Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Adverse Events following transplant with TBX-1400

Secondary Outcome

 Transplant Engraftment

Condition

Severe Combined Immunodeficiency

Intervention

TBX-1400

Study Arms / Comparison Groups

 TBX-1400 treatment
Description:  Single intravenous infusion of TBX-1400

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

8

Start Date

August 2020

Completion Date

March 2024

Primary Completion Date

February 2024

Eligibility 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.
      

Gender

All

Ages

1 Month - 4 Years

Accepts Healthy Volunteers

No

Contacts

, +1-720-859-3547, [email protected]

Location Countries

Israel

Location Countries

Israel

Administrative Informations


NCT ID

NCT02860559

Organization ID

TBX-1400-001


Responsible Party

Sponsor

Study Sponsor

Taiga Biotechnologies, Inc.


Study Sponsor

, , 


Verification Date

March 2020