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Acute Lymphoblastic LeukemiaPhase 1/2NCT03301168

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

A Phase 1/2 study testing BPX-501 T cells and Rimiducid in people with Acute Lymphoblastic Leukemia and Leukemia, Acute Myeloid (AML), Child, run by Bellicum Pharmaceuticals at 10 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. Read more about this study

Who is it for?
Ages 1 month to 26 years
What is being tested?
BPX-501 T cells and Rimiducid
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
120 participants
Where
10 sitesUnited States
Study dates
Apr 2014to May 2034
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 26 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age > 1 month and < 26 years
  • Life expectancy > 10 weeks
  • Subjects deemed eligible for allogeneic stem cell transplantation.
  • Subjects with life-threatening hematological malignancies (high-risk ALL in 1st CR, ALL in 2nd or subsequent CR, AML in 1st CR, AML in 2nd or subsequent CR, myelodysplastic syndromes, non-Hodgkin lymphomas in 2nd or subsequent CR, other hematologic malignancies eligible for stem cell transplantation per institutional standard);
  • Non-malignant disorders amenable to cure by an allograft:
  • primary immune deficiencies,
Show all 15Show fewer
  • severe aplastic anemia not responding to immune suppressive therapy,
  • osteopetrosis,
  • hemoglobinopathies, (thalassemias, and sickle cell anemia, and Diamond-Blackfan anemia among others)
  • congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML) Note: Subjects will be eligible if they meet either item 4 OR item 5.
  • Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative or 10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
  • A minimum genotypic identical match of 5/ 10 is required.
  • The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA- DRB1 and HLA-DQB1.
  • Lansky/Karnofsky score > 50
  • Signed written informed consent

Who cannot join

  • Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
  • Subject receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
  • Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 mL / min)
  • Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
  • Current active infectious disease (including positive HIV serology or viral RNA)
  • Serious concurrent uncontrolled medical disorder
  • Pregnant or breastfeeding subject
  • For subjects who have received more than 1 x 10E5 alpha/beta T cells/kg with the graft infusion the clinical trial site must contact the sponsor for approval to be eligible to receive BPX-501 infusion.

Trial locations and contacts

10 sites in 1 country.

10 sites in 1 country

United States10 sites

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Dana-Farber Boston Children's Cancer and Blood Disorders Center

    Boston, Massachusetts, 02215

  • University of Texas Southwestern-Children's Medical Center

    Dallas, Texas, 77390

  • Baylor College of Medicine/ Texas Children's Hospital

    Houston, Texas, 77030

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine

    Palo Alto, California, 94304

  • Oregon Health Sciences University - Doernbecher Children's Hospital

    Portland, Oregon, 97239

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109

  • Children's Hospital at Montefiore

    The Bronx, New York, 10467

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).

Read the detailed description

This is a Phase 1/2 study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to determine whether BPX-501 infusion can enhance immune reconstitution in those patients with hematologic disorders, with the potential for reducing the severity and duration severe acute graft versus host disease (GvHD).

The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with GVHD that does not adequately respond to standard of care therapy.

Being tested
BPX-501 T cells, Rimiducid
Keywords
ALL, AML, hematologic neoplasms, hematologic malignancies, primary immune deficiences, allogeneic stem cell transplant
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Leukemia, Myeloid, Acute, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immunodeficiency Diseases, Osteopetrosis, Cytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BPX-501 T cellsBiological

    Also called rivogenlecleucel

    T cells transduced with CaspaCIDe® safety switch

  • RimiducidDrug

    Also called AP1903

    administered to inactivate BPX-501 cells in the event of GVHD

Study groups in NCT03301168
Study groupRoleWhat they receive
BPX-501 T cells and RimiducidExperimentalTCR alpha beta depleted graft infusion with addback of BPX-501 T cells. Rimiducid: Dimerizer drug administered to subjects who present with Grade I-IV acute GVHD with inadequate response to steroids within 48 hours of treatment or mild to severe chronic GVHD with inadequate response to steroids within 7 days of treatment.
Receives: Biological: BPX-501 T cells; Drug: Rimiducid

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse Event

    Demonstrate safety of BPX-501 MTD

    Measured: Month 24

  2. TRM/NRM

    Assess the cumulative incidence of non-relapse/transplant related mortality

    Measured: Day 180, Month 12

Secondary outcomes (7)

  1. Disease-free survival

    Disease-free survival rates after transplantation

    Measured: Month 24

  2. Relapse

    Cumulative incidence of relapse

    Measured: Month 12

  3. Engraftment

    Cumulative incidence of neutrophil and platelet engraftment, primary \& secondary graft failure

    Measured: Month 24

  4. GvHD

    Cumulative incidence and severity of acute and chronic GvHD

    Measured: Month 24

  5. Rimiducid Efficacy

    Time to resolution of acute or chronic GvHD after administration of rimiducid

    Measured: Month 24

  6. Infection

    Rate of infectious complications

    Measured: Month 24

  7. Hospitalizations

    Duration of hospitalization and rehospitalization

    Measured: Month 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Oct 2017
  2. Study startApr 2014
  3. Primary completion11 May 2021
  4. Study completionMay 2034 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
120 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03301168
Sponsor's protocol ID
BP-U-004
Status last verified
May 2022
First submitted
29 Sep 2017
Last updated
12 Jul 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No
Expanded access
Available

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing BPX-501 T cells and Rimiducid in people with Acute Lymphoblastic Leukemia and Leukemia, Acute Myeloid (AML), Child, run by Bellicum Pharmaceuticals at 10 sites in United States.

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 15 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 12 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Event (time frame: Month 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03301168.

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 12 Jul 2022.

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