Acute Lymphoblastic LeukemiaPhase 1/2NCT03733249
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
A Phase 1/2 study testing Rimiducid and rivogenlecleucel in people with Acute Lymphoblastic Leukemia and Leukemia, Acute Myeloid (AML), Child, run by Bellicum Pharmaceuticals at 1 site in Italy.
Trial summary
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin
- What is being tested?
- Rimiducid and rivogenlecleucel
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 187 participants
- Where
- 1 siteItaly
- Study dates
- Jan 2017to 14 Apr 2023
- Phase
- Phase 1/2
- 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 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed written informed consent by the patient or the patient's guardian for children who are minors
- Enrolled on BP-004 protocol, received BPX-501 infusion, completed or discontinued from the study, and are beyond Day +180.
Who cannot join
- Lack of parents'/guardian's informed consent for children who are minors
- Loss of allograft prior to 6 months
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
Read the detailed description
Subjects enrolled on the BP-004 study who have completed or discontinued from the study, and are beyond Day 180 will be requested to enroll on this long-term follow up protocol. Long term follow up for gene therapy clinical and safety endpoints will continue up to 15 years.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immunodeficiency, Anemia, Aplastic
- Being tested
- Rimiducid, rivogenlecleucel
- Keywords
- Long term follow up, Gene-modified cells, ALL, AML, hematologic neoplasms, hematologic malignancies, anemia, congenital cytopenia, primary immune deficiencies, hemoglobinopathy
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Leukemia, Myeloid, Acute, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immunodeficiency Diseases, Anemia, Aplastic, Hemoglobinopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RimiducidDrug
Also called AP1903
Rimiducid is administered to treat chronic graft versus host disease
rivogenlecleucelBiological
Also called BPX-501
donor T-cells modified with iCasp safety switch
| Study group | Role | What they receive |
|---|---|---|
| Rimiducid and Rivogenlecleucel | Experimental | Rimiducid: to treat uncontrolled GVHD in patients who have received rivogenlecleucel Rimiducid will be given at 0.4 mg/kg weight (intravenous infusion)
No further rivogenlecleucel infusions are planned. Patients who received rivogenlecleucel in the BP-004 study will be evaluated for long-term safety and efficacy. Receives: Drug: Rimiducid; Biological: rivogenlecleucel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population
Measured: 1 and 2 years after rivogenlecleucel infusion
Incidence of Disease-free Survival
KM Parameter Estimates of disease-free survival (DFS) in the non-malignant subpopulation at 1 and 2 years in the Intent-to-Treat (ITT) Population
Measured: 1 and 2 years after rivogenlecleucel infusion
Relapse-free Survival
Kaplan-Meier Parameter Estimates of Relapse-free survival rate (number of patients survived without experiencing a recurrence) at the 1-year and 2-year timepoints in the Intent-to-Treat (ITT) Population in the malignant study arm (patients with a malignant reason for their transplant).
ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg
Measured: 1 and 2 years after rivogenlecleucel infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2018
- Study startJan 2017
- Primary completion30 Jun 2020
- Study completion14 Apr 2023
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 Acute Lymphoblastic LeukemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bellicum Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bellicum Pharmaceuticals, Study director, Bellicum Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03733249
- Sponsor's protocol ID
- BP-404
- Status last verified
- Sep 2023
- First submitted
- 2 Nov 2018
- Last updated
- 26 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders
- ClinicalTrials.gov ID
- NCT03733249
- Acronym / sponsor ID
- BP-404
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immunodeficiency, Anemia, Aplastic
- Keywords
- Long term follow up, Gene-modified cells, ALL, AML, hematologic neoplasms, hematologic malignancies, anemia, congenital cytopenia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 187 participants (actual)
- Lead sponsor
- Bellicum PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2017 (actual)
- Primary completion
- 30 Jun 2020 (actual)
- Study completion
- 14 Apr 2023 (actual)
- First posted
- 7 Nov 2018 (actual)
- Results
- Posted 26 Sep 2023
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 long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
Detailed description
Subjects enrolled on the BP-004 study who have completed or discontinued from the study, and are beyond Day 180 will be requested to enroll on this long-term follow up protocol. Long term follow up for gene therapy clinical and safety endpoints will continue up to 15 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rimiducid and Rivogenlecleucel | Experimental | Drug: Rimiducid, Biological: rivogenlecleucelRimiducid: to treat uncontrolled GVHD in patients who have received rivogenlecleucel Rimiducid will be given at 0.4 mg/kg weight (intravenous infusion) No further rivogenlecleucel infusions are planned. Patients who received rivogenlecleucel in the BP-004 study will be evaluated for long-term safety and efficacy. |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Signed written informed consent by the patient or the patient's guardian for children who are minors
- Enrolled on BP-004 protocol, received BPX-501 infusion, completed or discontinued from the study, and are beyond Day +180.
Key exclusion criteria (2 listed)
- Lack of parents'/guardian's informed consent for children who are minors
- Loss of allograft prior to 6 months
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 |
|---|---|---|
| RimiducidAlso: AP1903Rimiducid is administered to treat chronic graft versus host disease | Drug | Rimiducid and Rivogenlecleucel |
| rivogenlecleucelAlso: BPX-501donor T-cells modified with iCasp safety switch | Biological | Rimiducid and Rivogenlecleucel |
Primary outcome
Overall Survival
Time frame: 1 and 2 years after rivogenlecleucel infusion
Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population
Incidence of Disease-free Survival
Time frame: 1 and 2 years after rivogenlecleucel infusion
KM Parameter Estimates of disease-free survival (DFS) in the non-malignant subpopulation at 1 and 2 years in the Intent-to-Treat (ITT) Population
Relapse-free Survival
Time frame: 1 and 2 years after rivogenlecleucel infusion
Kaplan-Meier Parameter Estimates of Relapse-free survival rate (number of patients survived without experiencing a recurrence) at the 1-year and 2-year timepoints in the Intent-to-Treat (ITT) Population in the malignant study arm (patients with a malignant reason for their transplant). ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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 study director
Bellicum Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Rimiducid and rivogenlecleucel in people with Acute Lymphoblastic Leukemia and Leukemia, Acute Myeloid (AML), Child, run by Bellicum Pharmaceuticals at 1 site in Italy.
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 2 inclusion and 2 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 26 Sep 2023. 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 Italy. 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?
Yes. Results are posted in the registry (first posted 26 Sep 2023). 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?
Overall Survival (time frame: 1 and 2 years after rivogenlecleucel infusion). The study lists 3 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 26 Sep 2023.