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

NCT ID: NCT02860559

Last Updated: 2020-10-08

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-08-31
• Study Completion Date: 2024-03-31

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.

Conditions

Severe Combined Immunodeficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

TBX-1400 treatment

Single intravenous infusion of TBX-1400

Group Type: EXPERIMENTAL

TBX-1400

Intervention Type: BIOLOGICAL

Hematopoietic stem cells transplantation

Interventions

TBX-1400

Hematopoietic stem cells transplantation

Intervention Type: BIOLOGICAL

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 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.

• Minimum Eligible Age: 1 Month
• Maximum Eligible Age: 4 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Taiga Biotechnologies, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hadassah Medical Center (Ein Kerem site)

Jerusalem, , Israel

Schneider Children's Medical Center

Petah Tikva, , Israel

Countries

Israel

Central Contacts

Yosef Refaeli, Dr.

Role: CONTACT

refaeli@taigabiotech.com

+1-720-859-3547

Brian Turner, Dr.

Role: CONTACT

turner@taigabiotech.com

+1-720-859-3547

Facility Contacts

Polina Stepensky, Dr.

Role: primary

polina@hadassah.org.il

+972-2-6779095

Hila Yosef

Role: backup

hilay@hadassah.org.il

+972-2-6777511

Jerry Stein, Dr.

Role: primary

jstein@clalit.org.il

+972-3-9253657

Shoshana Hanimov

Role: backup

shoshanaha1@clalit.org.il

+972-3-9253507

Other Identifiers

TBX-1400-001

Identifier Type: -

Identifier Source: org_study_id