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Gene Modified T Cells for Bone Marrow Failure Syndrome

Phase 1
Waitlist Available
Research Sponsored by Bellicum Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Fanconi anemia patients ONLY: 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.
Diagnosis of a nonmalignant disorder considered treatable by HCT
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 24
Awards & highlights

Study Summary

This trial is testing a new way to give gene-modified T cells to people who have had a stem cell transplant from a donor with a different blood type. Researchers want to find out if it is safe and helps prevent a complication called GVHD.

Who is the study for?
This trial is for individuals under 55 years old with non-malignant disorders treatable by stem cell transplant but lacking a perfect donor match. They must have at least a partial genetic match with the donor, agree to contraception if capable of reproduction, and not be pregnant or breastfeeding. Excluded are those with serious organ dysfunction, active infections, HIV, certain Fanconi anemia complications, or bovine product allergies.Check my eligibility
What is being tested?
The study tests BPX-501 gene modified T cells after a half-matched stem cell transplant to help the new cells settle in and Rimiducid on day 7 post-transplant to prevent graft-versus-host disease (GVHD), where transplanted immune cells attack the patient's body.See study design
What are the potential side effects?
Potential side effects may include reactions related to infusion of modified T-cells or Rimiducid such as fever, chills, fatigue; risk of GVHD despite prevention efforts; and possible impact on blood counts leading to increased infection risk.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a non-cancerous condition that can be treated with a stem cell transplant.
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My donor and I match at least one allele in all required HLA types.
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I am eligible for a transplant from a donor.
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I am between 4 months and 55 years old.
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I don't have a perfect match donor or my disease is worsening too quickly.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Adverse Events
Engraftment
Secondary outcome measures
Graft rejection
GvHD
High grade toxicity
+3 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: BPX-501 and RimiducidExperimental Treatment1 Intervention
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7

Find a Location

Who is running the clinical trial?

Bellicum PharmaceuticalsLead Sponsor
26 Previous Clinical Trials
1,309 Total Patients Enrolled
Bellicum Pharmaceuticals Senior DirectorStudy DirectorClinical Development
1 Previous Clinical Trials
4 Total Patients Enrolled

Media Library

BPX-501 and Rimiducid Clinical Trial Eligibility Overview. Trial Name: NCT02231710 — Phase 1
Hemoglobin Disorders Research Study Groups: BPX-501 and Rimiducid
Hemoglobin Disorders Clinical Trial 2023: BPX-501 and Rimiducid Highlights & Side Effects. Trial Name: NCT02231710 — Phase 1
BPX-501 and Rimiducid 2023 Treatment Timeline for Medical Study. Trial Name: NCT02231710 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the application process still open for prospective participants in this trial?

"Unfortunately, recruitment for this clinical trial has concluded. It was first posted on February 1st 2015 and last edited on July 10th 2022. For those interested in alternative trials, 1147 studies are currently seeking participants with hemoglobinopathies and 16 studies have opened up for BPX-501 or Rimiducid research."

Answered by AI

Does this research involve adults aged 65 and over?

"According to the criteria listed, this trial is open to individuals between 4 months and 55 years of age. There are 356 trials for those younger than 18 and 757 for seniors over 65."

Answered by AI

What overarching goals are investigators pursuing with this research?

"Bellicum Pharmaceuticals, the clinical trial sponsor for this study, states that Adverse Events will be monitored over a 28-day period as the primary outcome. Additionally, Graft rejection incidence and severity of acute/chronic GvHD will also be evaluated in order to gauge Immune Reconstitution."

Answered by AI

Have there been any other investigations involving BPX-501 and Rimiducid?

"Currently, 16 clinical trials are underway for BPX-501 and Rimiducid with none in their terminal phase. These trails are located primarily in Nashville Tennessee but span a total of 94 sites globally."

Answered by AI

What is the cap on enrollment for this research project?

"Unfortunately, the recruitment period for this trial has ended; the first post was on February 1st 2015 and it was last edited on July 10th 2022. But those with hemoglobinopathies have 1147 viable trials to choose from if they are looking for a clinical study, while 16 studies related to BPX-501 and Rimiducid are actively recruiting participants."

Answered by AI

Have the drugs BPX-501 and Rimiducid been authorized for use by the U.S. Food and Drug Administration?

"The safety of BPX-501 and Rimiducid is assessed at a score of 1, as this clinical trial is in Phase 1. This implies that there is restricted data available to support the efficacy and security of these two medications."

Answered by AI

Who qualifies to take part in this clinical research?

"This research project is recruiting one person aged between 4 months old and 55 years that has a hemoglobinopathy. As well as the aforementioned condition, they must meet these criteria: age over four months and under fifty-five, diagnosis of nonmalignant disorder treatable by haematopoietic cell transplantation (HCT), eligible for allogeneic HCT with either an exact 5/10 match or no suitable donor at hand; Fanconi anaemia sufferers requiring red blood cells or platelet transfusions due to bone marrow failure also qualify."

Answered by AI
~0 spots leftby Mar 2025