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CAR T-cell Therapy

Gene Modified T-cells for Blood Disorders

Phase 1 & 2

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

Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative or 10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor

Age > 1 month and < 26 years

Must not have

Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 mL / min)

Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up month 24

Awards & highlights

No Placebo-Only Group

Summary

This trial will look at whether T cells from a family member can help a patient's immune system recover after a stem cell transplant, with the added safety measure of a self-destruct switch.

Who is the study for?

This trial is for children and young adults (1 month to <26 years old) with certain life-threatening blood disorders or cancers, who are eligible for a stem cell transplant but don't have a fully matched donor available. Participants need to be in relatively good health aside from their primary condition, with major organs functioning well and a reasonable life expectancy.

What is being tested?

The study tests genetically modified T-cells called BPX-501 following a special type of stem cell transplant from partially matched family donors. The goal is to see if these engineered T-cells can help rebuild the immune system more quickly while having the ability to be turned off if they cause harmful reactions.

What are the potential side effects?

Potential side effects may include reactions related to the infusion of BPX-501 cells, such as fever or chills. There's also a risk that these cells could attack healthy tissues causing graft versus host disease, although they've been designed with a safety switch to destroy them if this occurs.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I don't have a matching donor or my disease is progressing too quickly to wait for one.

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I am between 1 month and 26 years old.

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I have a serious blood cancer and might need a stem cell transplant.

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I am eligible for a stem cell transplant from a donor.

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My genetic match for the treatment is at least half identical.

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My donor and I match in at least one allele for specific genetic markers.

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I am able to perform most of my daily activities.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My liver and kidney functions are within normal limits.

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I do not have severe heart problems like chronic arrhythmias, heart failure, or poor heart pump function.

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I have severe graft-versus-host disease from a past transplant.

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I do not have any active infections, including HIV.

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I am currently on immunosuppressive drugs for GVHD from a past transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 24

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Event

TRM/NRM

Secondary study objectives

Disease-free survival

Engraftment

GvHD

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: BPX-501 T cells and RimiducidExperimental Treatment2 Interventions

TCR alpha beta depleted graft infusion with addback of BPX-501 T cells. Rimiducid: Dimerizer drug administered to subjects who present with Grade I-IV acute GVHD with inadequate response to steroids within 48 hours of treatment or mild to severe chronic GVHD with inadequate response to steroids within 7 days of treatment.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rimiducid

2019

Completed Phase 1

~20

0 / 12Eligibility criteria met