Severe Combined ImmunodeficiencyPhase 1NCT02860559
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
A Phase 1 study testing TBX-1400 in people with Severe Combined Immunodeficiency, run by Taiga Biotechnologies, Inc. at 2 sites in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 4 years
- Conditions the study is for
- Severe Combined Immunodeficiency
- What is being tested?
- TBX-1400
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesIsrael
- Study dates
- Aug 2021to Mar 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 4 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.)
Who cannot join
- 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,
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- 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.
Trial locations and contacts
2 sites in 1 country.
Israel2 sites
Hadassah Medical Center (Ein Kerem site)
Jerusalem
Polina Stepensky, Dr., +972-2-6779095, polina@hadassah.org.il
Hila Yosef, +972-2-6777511, hilay@hadassah.org.il
Schneider Children's Medical Center
Petah Tikva, 4920235
Jerry Stein, Dr., +972-3-9253657, jstein@clalit.org.il
Shoshana Hanimov, +972-3-9253507, shoshanaha1@clalit.org.il
About this study
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
- Being tested
- TBX-1400
- Keywords
- Hematopoietic Stem Cell Transplantation, Severe Combined Immunodeficiency
- Medical subject headings
- Severe Combined Immunodeficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TBX-1400Biological
Hematopoietic stem cells transplantation
| Study group | Role | What they receive |
|---|---|---|
| TBX-1400 treatment | Experimental | Single intravenous infusion of TBX-1400 Receives: Biological: TBX-1400 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events following transplant with TBX-1400
Adverse events from subject or parent reporting or other assessments
Measured: Two years
Secondary outcomes (11)
Transplant Engraftment
Assessment of transplant engraftment will include analysis of T-cells , B-cells and Natural Killer cells
Measured: Up to Day 180
Chimerism
Assessment of chimerism will include analysis of T-cells , B-cells and Natural Killer cells
Measured: Up to Day 180
Absolute numbers of T-cells
Measured: Days 30 to 360
T-cell receptor excision circles (TREC)
Measured: Days 30 to 360.
Kappa-deleting recombination excision circles (KREC)
Measured: Days 30 to 360.
Immunoglobulin (Ig) levels
Measured: Days 30 to 360.
Immunoglobulin G (IgG) titers to pneumococcal antigens in 13-valent vaccine
Measured: Sixty days after final immunization
T-cell responses to anti-CD3 and phytohemagglutinin (PHA)
Measured: Days 30, 60, 90, 120, and 180.
Number of infections following transplant
Measured: Two years
Number of days granulocyte colony stimulating factor (G-CSF) was administered
Measured: Up to 1 year
Number of days to specific cell counts and last packed red blood cell (PRBC) transfusion
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2016 (estimated)
- Study startAug 2021 (estimated)
- Primary completionFeb 2024 (estimated)
- Study completionMar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Combined ImmunodeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taiga Biotechnologies, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02860559
- Sponsor's protocol ID
- TBX-1400-001
- Status last verified
- Oct 2020
- First submitted
- 26 Jul 2016
- Last updated
- 8 Oct 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
- ClinicalTrials.gov ID
- NCT02860559
- Acronym / sponsor ID
- TBX-1400-001
- Conditions
- Severe Combined Immunodeficiency
- Keywords
- Hematopoietic Stem Cell Transplantation, Severe Combined Immunodeficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Taiga Biotechnologies, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2021 (estimated)
- Primary completion
- Feb 2024 (estimated)
- Study completion
- Mar 2024 (estimated)
- First posted
- 9 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TBX-1400 treatment | Experimental | Biological: TBX-1400Single intravenous infusion of TBX-1400 |
Who may qualify
- Ages
- 1 month to 4 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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:
Key exclusion criteria (13 listed)
- 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,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TBX-1400Hematopoietic stem cells transplantation | Biological | TBX-1400 treatment |
Primary outcome
Adverse Events following transplant with TBX-1400
Time frame: Two years
Adverse events from subject or parent reporting or other assessments
11 secondary outcomes
Transplant Engraftment
Time frame: Up to Day 180
Chimerism
Time frame: Up to Day 180
Absolute numbers of T-cells
Time frame: Days 30 to 360
T-cell receptor excision circles (TREC)
Time frame: Days 30 to 360.
Kappa-deleting recombination excision circles (KREC)
Time frame: Days 30 to 360.
Immunoglobulin (Ig) levels
Time frame: Days 30 to 360.
Immunoglobulin G (IgG) titers to pneumococcal antigens in 13-valent vaccine
Time frame: Sixty days after final immunization
T-cell responses to anti-CD3 and phytohemagglutinin (PHA)
Time frame: Days 30, 60, 90, 120, and 180.
Number of infections following transplant
Time frame: Two years
Number of days granulocyte colony stimulating factor (G-CSF) was administered
Time frame: Up to 1 year
Number of days to specific cell counts and last packed red blood cell (PRBC) transfusion
Time frame: Up to 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2020.
- Individual participant dataSharing planned: No
Related trials for Severe Combined Immunodeficiency
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Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
X-linked Severe Combined Immunodeficiency (XSCID)
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SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
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Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TBX-1400 in people with Severe Combined Immunodeficiency, run by Taiga Biotechnologies, Inc. at 2 sites in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yosef Refaeli, Dr. (+1-720-859-3547, refaeli@taigabiotech.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events following transplant with TBX-1400 (time frame: Two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2020.