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CAR T-cell Therapy

Gene Modified T-cells for Blood Disorders

Q: How widespread is the implementation of this experiment?

11 medical establishments are currently taking part in this clinical trial, including <a href="https://www.withpower.com/clinical-trials/oregon">Oregon</a> Health Sciences University - Doernbecher Children's Hospital of Portland and the Fred Hutchinson <a href="https://www.withpower.com/clinical-trials/cancer">Cancer</a> Research Center of Seattle. Additionally,Children's Healthcare of Atlanta is also recruiting patients for the study.

Q: Can I qualify for inclusion in this experiment?

In order to be accepted into this medical trial, the patient must have anemia or aplastic and fall between one month and 26 years of age. Around 120 people are needed for participation in total.

Q: What other experiments have utilized BPX-501 T cells and what was the outcome?

The development of BPX-501 T cells began in 2011 at <a href="https://www.withpower.com/clinical-trials/texas">Texas</a> Children's Hospital. Subsequently, 4 clinical trials have already been completed and there are currently 16 active studies, with a high concentration located in Portland, <a href="https://www.withpower.com/clinical-trials/oregon">Oregon</a>.

Q: What is the primary aim of this trial?

The principal aim of the trial over a timespan of 180 days and 12 months is to gauge Adverse Event. Furthermore, Rimiducid Efficacy determined by 'Time to resolution', Relapse calculated as 'Cumulative incidence' and Hospitalizations measured through 'Duration' are <a href="https://www.withpower.com/clinical-trials/all">all</a> considered secondary objectives.

Q: Does the age criterion for this research include participants below thirty?

The eligibility requirements stipulate that participants should be between 1 Month and 26 years old. Simultaneously, 638 studies are available for children under 18 and 1762 trials exist specifically for seniors over 65.

Q: How many individuals have been accepted into this research endeavor?

At the present moment, this research trial is not enrolling participants. It was initially advertised on April 1st 2014 and its most recent update occurred July 10th 2022. However, those interested in similar studies can find 2159 trials for anemia patients and 16 for BPX-501 T cells currently recruiting volunteers.

Q: Are there currently any opportunities for volunteers to participate in this research?

According to clinicaltrials.gov, this medical trial is not currently open for enrollment; the investigation was initially posted on April 1st 2014 and last updated on July 10th 2022. Nonetheless, there are 2175 other experiments actively looking for participants at present moment.

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up month 24

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Select...

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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Adverse Event

TRM/NRM

Secondary outcome measures

Disease-free survival

Engraftment

GvHD

+4 more

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 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Bellicum PharmaceuticalsLead Sponsor

27 Previous Clinical Trials

1,232 Total Patients Enrolled

1 Trials studying Osteopetrosis

187 Patients Enrolled for Osteopetrosis

Media Library

BPX-501 T cells (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03301168 — Phase 1 & 2

Osteopetrosis Research Study Groups: BPX-501 T cells and Rimiducid

Osteopetrosis Clinical Trial 2023: BPX-501 T cells Highlights & Side Effects. Trial Name: NCT03301168 — Phase 1 & 2

BPX-501 T cells (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03301168 — Phase 1 & 2

Frequently Asked Questions

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

How widespread is the implementation of this experiment?

"11 medical establishments are currently taking part in this clinical trial, including Oregon Health Sciences University - Doernbecher Children's Hospital of Portland and the Fred Hutchinson Cancer Research Center of Seattle. Additionally,Children's Healthcare of Atlanta is also recruiting patients for the study."

Answered by AI

Can I qualify for inclusion in this experiment?

"In order to be accepted into this medical trial, the patient must have anemia or aplastic and fall between one month and 26 years of age. Around 120 people are needed for participation in total."

Answered by AI

What other experiments have utilized BPX-501 T cells and what was the outcome?

"The development of BPX-501 T cells began in 2011 at Texas Children's Hospital. Subsequently, 4 clinical trials have already been completed and there are currently 16 active studies, with a high concentration located in Portland, Oregon."

Answered by AI

What is the primary aim of this trial?

"The principal aim of the trial over a timespan of 180 days and 12 months is to gauge Adverse Event. Furthermore, Rimiducid Efficacy determined by 'Time to resolution', Relapse calculated as 'Cumulative incidence' and Hospitalizations measured through 'Duration' are all considered secondary objectives."

Answered by AI

Does the age criterion for this research include participants below thirty?

"The eligibility requirements stipulate that participants should be between 1 Month and 26 years old. Simultaneously, 638 studies are available for children under 18 and 1762 trials exist specifically for seniors over 65."

Answered by AI

How many individuals have been accepted into this research endeavor?

"At the present moment, this research trial is not enrolling participants. It was initially advertised on April 1st 2014 and its most recent update occurred July 10th 2022. However, those interested in similar studies can find 2159 trials for anemia patients and 16 for BPX-501 T cells currently recruiting volunteers."

Answered by AI

Are there currently any opportunities for volunteers to participate in this research?

"According to clinicaltrials.gov, this medical trial is not currently open for enrollment; the investigation was initially posted on April 1st 2014 and last updated on July 10th 2022. Nonetheless, there are 2175 other experiments actively looking for participants at present moment."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

2014. "Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03301168.

All > Aplastic Anemia > Myelodysplastic Syndrome