Primary Immune Deficiency DisordersPhase 1NCT02231710
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
A Phase 1 study testing BPX-501 and Rimiducid in people with Primary Immune Deficiency Disorders and Hemophagocytic Lymphohistiocytosis, run by Bellicum Pharmaceuticals at 1 site in United States.
Trial summary
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD. Read more about this study
- Who is it for?
- Ages 4 months to 55 years
- Conditions the study is for
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome
- What is being tested?
- BPX-501 and Rimiducid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2015to Jan 2018
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 4 months to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient must meet eligibility criteria for allogeneic transplantation
- Lack of suitable conventional donor (10/10 allele matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- Males or females
- Age < 55 years old and > 4 months
- Diagnosis of a nonmalignant disorder considered treatable by HCT.
- HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, DRBl, and DQB1 loci.
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- A minimum match of 5/10 is required. ii. The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following
- If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment.
- Informed consent signed by patient (if ≥18 years old) or parent/guardian (if <18 years old).
- Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:
- Any patient with Fanconi anemia and bone marrow failure involving 2 of the following 3 lineages: granulocyte count <0.5 x 109/L, platelet count <20 x 109/L, or hemoglobin <8 g/dL.
- Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure
- Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage.
Who cannot join
- Serious organ dysfunction
- Pregnant or breast-feeding
- Evidence of HIV infection
- Bovine product allergy
- Patients with an active infectious disease
- Patients with Fanconi anemia with AML/MDS.
Trial locations and contacts
1 site in 1 country.
United States1 site
Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, 98109
About this study
Official title: A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
Read the detailed description
This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by a Rimiducid infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of Rimiducid which can facilitate engraftment and prevent the occurrence of GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Being tested
- BPX-501 and Rimiducid
- Keywords
- Severe Combined Immune Deficiency, Congenital T-cell Defect, Congenital T-cell Deficiency, Chronic Granulomatous Disease, Shwachman Diamond Syndrome, Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Sickle Cell Disease, Thalassemia, Mucopolysaccharidosis, Sphingolipidoses
- Medical subject headings
- Primary Immunodeficiency Diseases, Lymphohistiocytosis, Hemophagocytic, Congenital Bone Marrow Failure Syndromes, Hemoglobinopathies, Metabolic Diseases, Severe Combined Immunodeficiency, Granulomatous Disease, Chronic, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BPX-501 and RimiducidBiological
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7
| Study group | Role | What they receive |
|---|---|---|
| BPX-501 and Rimiducid | Experimental | Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 Receives: Biological: BPX-501 and Rimiducid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7.
this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.
Measured: 24 months
Engraftment
Determine the engraftment rate (defined as >50% donor CD3 chimerism) on day 28 after HCT with HLA-haploidentical CD34+ selected PBSC grafts per dose cohort of BPX 501 T cells followed by Rimiducid infusion on Day 7.
NOTE: only one patient was enrolled who received the dose of 5x 10^6cell/kg dose of BPX-501
Measured: Day 28
Secondary outcomes (6)
GvHD
To determine the incidence and severity of acute and chronic GVHD
Measured: Month 24
Immune Reconstitution
Measure immune reconstitution
Measured: Month 24
Infection Rates
Determine the risk for severe infections
Measured: Day 200
Graft Rejection
Incidence of graft rejection
Measured: Month 24
Rimiducid Activity
Time to resolution of acute and chronic GvHD following administration of Rimiducid
Measured: Month 24
High Grade Toxicity
Rate of high grade toxicity
Measured: Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2014 (estimated)
- Study startFeb 2015
- Primary completionSep 2015
- Study completionJan 2018
Why it stopped: due to lack of enrollment and changes to the sponsor development portfolio
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary Immune Deficiency DisordersStudies 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bellicum Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bellicum Pharmaceuticals Senior Director, Study director, Clinical Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02231710
- Sponsor's protocol ID
- BP-003
- Status last verified
- Sep 2023
- First submitted
- 28 Aug 2014
- Last updated
- 25 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Diseases
- ClinicalTrials.gov ID
- NCT02231710
- Acronym / sponsor ID
- BP-003
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Keywords
- Severe Combined Immune Deficiency, Congenital T-cell Defect, Congenital T-cell Deficiency, Chronic Granulomatous Disease, Shwachman Diamond Syndrome, Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: due to lack of enrollment and changes to the sponsor development portfolio
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Bellicum PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2015 (actual)
- Primary completion
- Sep 2015 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 4 Sep 2014 (estimated)
- Results
- Posted 25 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
Detailed description
This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by a Rimiducid infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of Rimiducid which can facilitate engraftment and prevent the occurrence of GVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BPX-501 and Rimiducid | Experimental | Biological: BPX-501 and RimiducidSingle administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 |
Who may qualify
- Ages
- 4 months to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Patient must meet eligibility criteria for allogeneic transplantation
- Lack of suitable conventional donor (10/10 allele matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- Males or females
- Age < 55 years old and > 4 months
- Diagnosis of a nonmalignant disorder considered treatable by HCT.
Key exclusion criteria (6 listed)
- Serious organ dysfunction
- Pregnant or breast-feeding
- Evidence of HIV infection
- Bovine product allergy
- Patients with an active infectious disease
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 |
|---|---|---|
| BPX-501 and RimiducidSingle administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7 | Biological | BPX-501 and Rimiducid |
Primary outcome
Adverse Events
Time frame: 24 months
To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7. this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.
Engraftment
Time frame: Day 28
Determine the engraftment rate (defined as >50% donor CD3 chimerism) on day 28 after HCT with HLA-haploidentical CD34+ selected PBSC grafts per dose cohort of BPX 501 T cells followed by Rimiducid infusion on Day 7. NOTE: only one patient was enrolled who received the dose of 5x 10^6cell/kg dose of BPX-501
6 secondary outcomes
GvHD
Time frame: Month 24
Immune Reconstitution
Time frame: Month 24
Infection Rates
Time frame: Day 200
Graft Rejection
Time frame: Month 24
Rimiducid Activity
Time frame: Month 24
High Grade Toxicity
Time frame: Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Bellicum Pharmaceuticals Senior Director study director
Clinical Development
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BPX-501 and Rimiducid in people with Primary Immune Deficiency Disorders and Hemophagocytic Lymphohistiocytosis, 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 13 inclusion and 6 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: stopped early, as of the sponsor's last update on 25 Mar 2024. 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?
Yes. Results are posted in the registry (first posted 25 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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: 24 months). 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?
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 25 Mar 2024.