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Hematologic MalignanciesPhase 1NCT02487459

Safety Study of Gene Modified Donor T-Cells in Adults With Advanced Hematologic Malignancies

A Phase 1 study testing BPX-501 and AP1903 in people with Hematologic Malignancies, run by Bellicum Pharmaceuticals at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This is an open-label, non-randomized study to evaluate the safety of two planned infusions of BPX-501 T cells after partially mismatched, related (haploidentical) HSCT in adults with hematologic malignancies. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Hematologic Malignancies
What is being tested?
BPX-501 and AP1903
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
Jul 2016to Jun 2017
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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed informed consent
  • Patients with one of the life-threatening hematological malignancies:
  • Acute lymphocytic leukemia (ALL) in CR1 with high-risk features including adverse cytogenetics such as t(9;22), t(1;19), t(4;11), or MLL gene rearrangements; greater than 1 cycle to achiever remission or with persistent MRD; ALL in second or greater remission with or without MRD. Acute myeloid leukemia (AML) in CR1 with high-risk features defined as: Greater than 1 cycle of induction therapy required to achieve remission; Preceding myelodysplastic syndrome (MDS) or myeloproliferative disease; Presence of FLT3 mutations or internal tandem duplications; FAB M6 or M7 classification; Adverse cytogenetics, -5, del 5q, -7, del7q, abnormalities involving 3q, 9q, 11q, 20q, 21q, 17, +8 [> 3 abnormalities];
  • AML in second or greater remission, primary induction failure and patients with relapsed disease;
  • Advanced chronic myeloid leukemia (CML) who have progressed to blast phase or accelerated phase and are in need of a transplant and do not have an HLA matched donor;
  • MDS with IPSS intermediate-2 or higher or therapy-related MDS.Hodgkin lymphoma or Non-Hodgkin lymphoma (NHL): relapsed disease where remission duration is less than 1 year, relapse after previous autologous transplant, or failure to achieve CR with chemotherapy.
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  • Age ≥ 18 years and ≤ 65 years
  • Deemed eligible for allogeneic stem cell transplantation
  • Lack of suitable conventional donor (i.e. 8/8 related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
  • HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, and DRBl loci
  • A minimum genotypic identical haplotype match of 4/8 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, and HLA- DRB1
  • Subjects with adequate organs function as measured by:
  • Cardiac: Left ventricular ejection fraction at rest must be >45%
  • Pulmonary: FEV 1, FVC, DLCO (diffusion capacity) > 50% predicted (corrected for hemoglobin); or O2 saturation > 92% on room air
  • Hepatic: Direct bilirubin ≤ 3 x upper limit of normal (ULN), or AST/ALT ≤ 5 x ULN
  • Renal: Serum creatinine within normal range for age or creatinine clearance, or with a recommended GFR ≥ 50 mL/min/1.73m2
  • Performance status: Karnofsky ≥ 80%

Who cannot join

  • HLA 8/8 allele matched (HLA-A,-B,-Cw,-DRBl) related or unrelated donor able to donate;
  • Autologous hematopoietic stem cell transplant ≤ 3 months prior to enrollment;
  • Prior allogeneic transplantation;
  • Active CNS involvement by malignant cells (less than 2 months from the conditioning);
  • Current uncontrolled bacterial, viral or fungal infection (currently taking medication with evidence of progression of clinical symptoms or radiologic findings); the PI is the final arbiter of this criterion;
  • Positive HIV serology or viral RNA (≥ Grade III per CTCAE criteria);
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  • Pregnancy (positive serum or urine βHCG test) or breast-feeding;
  • Fertile men or women unwilling to use effective forms of birth control or abstinence for a year after transplantation;
  • Bovine product allergy.
  • Severe obesity (patient's weight is >/= 1.5x the donor weight).

Trial locations and contacts

1 site in 1 country.

United States1 site

  • MD Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: A Phase I/II Safety Study of Planned BPX-501 T Cell Infusion After Partially Mismatched, Related, HSCT in Adults With Advanced Hematologic Malignances at High Risk for Relapse

This is an open-label, non-randomized study to evaluate the safety of two planned infusions of BPX-501 T cells after partially mismatched, related (haploidentical) HSCT in adults with hematologic malignancies.

Read the detailed description

The objective is to evaluate the safety of two planned infusions of BPX-501 after partially mismatched, related HSCT with post-transplant cyclophosphamide and to evaluate the safety and efficacy of the treatment of dimerizer drug, rimiducid (AP1903), to subjects who received BPX-501 and have uncontrolled GvHD. Assuming no toxicity, enrollment will proceed sequentially for the initial 9 patients (following the 3+3 design), who will be followed for 100 days, prior to enrolling the subsequent 31 patients. Toxicity may increase the number of initial group of patients). As multiple dose levels may be administered among the first 9 (or more) patients, toxicity will be assessed on the cohort with the maximum tolerated dose (MTD).

The Medical Monitoring committee will review the data with the investigators and determine whether to proceed and or implement any changes to the protocol

BPX-501 contains genetically modified donor T cells that have an inducible safety switch iCasp9 suicide gene. In the event of acute GvHD, administration of rimiducid dimerizes and activates caspase 9; this activates downstream caspases, obligating cellular apoptosis within 24 hours.

Being tested
BPX-501, AP1903
Keywords
acute lymphoblastic leukemia, acute myeloid leukemia, chronic myeloid leukemia, myelodysplastic syndrome, non-hodgkin lymphoma, hodgkin lymphoma
Medical subject headings
Hematologic Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Hodgkin Disease

What participants receive

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

  • BPX-501Biological

    T cells transduced with CaspaCIDe suicide gene

  • AP1903Drug

    Also called Rimiducid

    dimerizer drug administered to treat GVHD

Study groups in NCT02487459
Study groupRoleWhat they receive
BPX-501 and AP1903ExperimentalThree cohorts, 3 patients each, will receive two infusions (at the same dose) of BPX-501. If needed to treat aGVHD, a single dose of AP1903 will be administered IV.
Receives: Biological: BPX-501; Drug: AP1903

What is being measured

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

  1. Adverse events

    Number of adverse events after BPX-501 as a measure of safety

    Measured: 2 years

Secondary outcomes (1)

  1. Adverse events

    Number of adverse events after AP1903 as a measure of safety

    Measured: 48 hours

Timeline

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

  1. First posted1 Jul 2015 (estimated)
  2. Study startJul 2016
  3. Primary completionJun 2017
  4. Study completionJun 2017

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
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02487459
Sponsor's protocol ID
BP-005
Status last verified
Oct 2020
First submitted
29 Jun 2015
Last updated
5 Oct 2020
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 study testing BPX-501 and AP1903 in people with Hematologic Malignancies, run by Bellicum Pharmaceuticals at 1 site 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 18 inclusion and 10 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: withdrawn, as of the sponsor's last update on 5 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 1 site 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 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?

The study was withdrawn before enrolling participants, so there are no results.

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 events (time frame: 2 years). 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 NCT02487459.

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 5 Oct 2020.

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