A Phase 1 interventional study of BPX-501 and Rimiducid in Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis and Inherited Bone Marrow Failure Syndrome, sponsored by Bellicum Pharmaceuticals. Terminated at 1 site in United States. Open to participants aged 4 Months to 55 Years. Per ClinicalTrials.gov, last updated 2024-03-25.
Sponsored by Bellicum Pharmaceuticals · Phase 1, Interventional, and Treatment
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.
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.
211 studies on the registry are indexed under Primary Immunodeficiency Diseases; 51 are open to participants now.
This study's enrollment of 1 is below the median of 36 across 125 interventional studies indexed under Primary Immunodeficiency Diseases.
Browse Primary Immunodeficiency Diseases studies →Bellicum Pharmaceuticals is the lead sponsor of 15 studies on the registry; none are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 5 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, DRBl, and DQB1 loci.
i. 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
Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single 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
Drug pages: Rivogenlecleucel · AP 1903 reagent
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7
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.
Time frame: 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
Time frame: Day 28
GvHD
To determine the incidence and severity of acute and chronic GVHD
Time frame: Month 24
Immune Reconstitution
Measure immune reconstitution
Time frame: Month 24
Infection Rates
Determine the risk for severe infections
Time frame: Day 200
Graft Rejection
Incidence of graft rejection
Time frame: Month 24
Rimiducid Activity
Time to resolution of acute and chronic GvHD following administration of Rimiducid
Time frame: Month 24
High Grade Toxicity
Rate of high grade toxicity
Time frame: Month 24
| Milestone | BPX-501 and Rimiducid |
|---|---|
| Started | 1 |
| Completed | 0 |
| Not completed | 1 |
| Withdrew: Physician decision | 1 |
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.
No measurements were reported for this outcome.
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
No measurements were reported for this outcome.
To determine the incidence and severity of acute and chronic GVHD
Results for this outcome have not been posted.
Measure immune reconstitution
Results for this outcome have not been posted.
Determine the risk for severe infections
Results for this outcome have not been posted.
Incidence of graft rejection
Results for this outcome have not been posted.
Time to resolution of acute and chronic GvHD following administration of Rimiducid
Results for this outcome have not been posted.
Rate of high grade toxicity
Results for this outcome have not been posted.
Collected over was intended for 24 months. Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BPX-501 and Rimiducid | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | BPX-501 and Rimiducid |
|---|---|
| haemolysisBlood and lymphatic system disorders | 1/1 |
| Event | BPX-501 and Rimiducid |
|---|---|
| Platelet Count DecreasedInvestigations | 1/1 |
| Haematocrit DecreasedInvestigations | 1/1 |
| Activated Partial Thromboplastin Time abnormalityInvestigations | 1/1 |
| HypersensitivityImmune system disorders | 1/1 |
| Febrile NeutropeniaBlood and lymphatic system disorders | 1/1 |
| Disseminated Intravascular CoagulationBlood and lymphatic system disorders | 1/1 |
| Age, Continuous(years) | BPX-501 and Rimiducid |
|---|---|
| Mean | 12 (12 to 12) |
| Sex: Female, Male(Participants) | BPX-501 and Rimiducid |
|---|---|
| Female | 0 |
| Male | 1 |
| Race and Ethnicity Not Collected(Participants) | BPX-501 and Rimiducid |
|---|
| Region of Enrollment(participants) | BPX-501 and Rimiducid |
|---|---|
| United States | 1 |
| sickle cell disease(Participants) | BPX-501 and Rimiducid |
|---|---|
| Count of participants | 1 |
Plan to share: No
Ran from Feb 2015 to primary completion in Sep 2015, 7 months.
Results were first posted in Mar 2024, 8 years 6 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Rivogenlecleucel · AP 1903 reagent
Primary Immunodeficiency Diseases →
Bellicum Pharmaceuticals