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CAR T-cell Therapy

CD4^LVFOXP3 for IPEX Syndrome

Q: Are any minors able to take part in this medical experiment?

This trial is recruiting individuals aged between 4 months and 35 years.

Q: Who is eligible to take part in this research endeavor?

This clinical trial is recruiting up to 36 subjects aged between 4 months and 35 years suffering from ipex. To be eligible, patients must also have a Lansky/Karnofsky Performance Scale score of at least 50%, no concurrent infections, evidence of progressive IPEX symptoms requiring immunosuppressive treatment, acceptable organ and marrow functioning levels, body weight above 8kg or the ability to tolerate leukapheresis procedure and FOXP3 gene mutation.

Q: Are there any opportunities for enrollment in this experiment?

Affirmative. Clinicaltrials.gov reveals that this research endeavor, which was first published on March 22nd 2022, is actively recruiting participants. Approximately 36 candidates must be recruited from 1 different medical centre.

Q: How many participants are involved in the ongoing research?

Affirmative. According to clinicaltrials.gov, the recruitment process for this medical trial has been ongoing since March 22nd 2022 with recent updates on June 9th of the same year. 36 candidates must be identified across a single location.

Q: Does CD4^LVFOXP3 pose any health risks to individuals receiving treatment?

As this is a Phase 1 trial with limited evidence for safety and efficacy, our team at Power have rated the CD4LVFOXP3 as having low risk (1 on a scale of 3).

Phase 1

Recruiting

Led By Maria Grazia Roncarolo, MD

Research Sponsored by Bacchetta, Rosa, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

≥ 50% Performance rating on Lansky/Karnofsky Scale

Body weight greater than 8 kg, unless assessed as able to tolerate leukapheresis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 15 years

Awards & highlights

Study Summary

This trial will test a new treatment for IPEX, a rare autoimmune disease, and evaluate its safety and feasibility.

Who is the study for?

This trial is for individuals with IPEX Syndrome who have persistent symptoms despite medication, weigh over 8 kg, and can tolerate leukapheresis. Participants should have a FOXP3 gene mutation and be at least 50% functional on the Lansky/Karnofsky Scale. They cannot join if they have severe unrelated diseases, allergies to study treatments, certain cancer histories, are medically unstable or expected to live less than six months.Check my eligibility

What is being tested?

The trial is testing CD4^LVFOXP3 in people with IPEX Syndrome to see if it's safe and how it affects their disease. It's a Phase 1 trial focused on evaluating the manufacturing feasibility of this treatment and its administration safety in up to 36 participants.See study design

What are the potential side effects?

Since this is a first-in-human study of CD4^LVFOXP3 for IPEX Syndrome patients, specific side effects are not yet known but will be closely monitored due to the potential risks associated with new experimental therapies.

Eligibility Criteria

Inclusion Criteria

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

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I can do most activities but may need help.

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I weigh more than 8 kg or am considered able to undergo a specific blood filtering procedure.

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I have recurring IPEX symptoms and need medication, even after a stem cell transplant.

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I do not have any current infections.

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My condition involves a FOXP3 gene mutation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 15 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 15 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 15 years for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Find the safe maximum tolerated dose

Meet target cell number for dose manufacturing

Secondary outcome measures

Change in Autoantibody Profile

Change in Bilirubin levels

Change in Body Mass Index (BMI)

+28 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort B (<12 years)Experimental Treatment1 Intervention

Participants in Cohort B will always follow treatment of participants in Cohort A for the same dose level. Cohort B will start at Dose Level 2 and be administered 3 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%). If in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants.

Group II: Cohort A (≥12 years)Experimental Treatment1 Intervention

The first participant in Dose Level 1 will be administered 1.0 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in the first participant, the following participants in Dose Level 1 will be administered the same dose of 1.0 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 1, participants will be enrolled into Dose Level 2 and administered 3 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%). If in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants.

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Bacchetta, Rosa, MDLead Sponsor

California Institute for Regenerative Medicine (CIRM)OTHER

64 Previous Clinical Trials

3,127 Total Patients Enrolled

Food and Drug Administration (FDA)FED

171 Previous Clinical Trials

1,331,576 Total Patients Enrolled

Media Library

CD4^LVFOXP3 (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05241444 — Phase 1

IPEX Syndrome Research Study Groups: Cohort A (≥12 years), Cohort B (<12 years)

IPEX Syndrome Clinical Trial 2023: CD4^LVFOXP3 Highlights & Side Effects. Trial Name: NCT05241444 — Phase 1

CD4^LVFOXP3 (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05241444 — Phase 1

Frequently Asked Questions

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

Are any minors able to take part in this medical experiment?

"This trial is recruiting individuals aged between 4 months and 35 years."

Answered by AI

Who is eligible to take part in this research endeavor?

"This clinical trial is recruiting up to 36 subjects aged between 4 months and 35 years suffering from ipex. To be eligible, patients must also have a Lansky/Karnofsky Performance Scale score of at least 50%, no concurrent infections, evidence of progressive IPEX symptoms requiring immunosuppressive treatment, acceptable organ and marrow functioning levels, body weight above 8kg or the ability to tolerate leukapheresis procedure and FOXP3 gene mutation."

Answered by AI

Are there any opportunities for enrollment in this experiment?

"Affirmative. Clinicaltrials.gov reveals that this research endeavor, which was first published on March 22nd 2022, is actively recruiting participants. Approximately 36 candidates must be recruited from 1 different medical centre."

Answered by AI

How many participants are involved in the ongoing research?

"Affirmative. According to clinicaltrials.gov, the recruitment process for this medical trial has been ongoing since March 22nd 2022 with recent updates on June 9th of the same year. 36 candidates must be identified across a single location."

Answered by AI

Does CD4^LVFOXP3 pose any health risks to individuals receiving treatment?

"As this is a Phase 1 trial with limited evidence for safety and efficacy, our team at Power have rated the CD4LVFOXP3 as having low risk (1 on a scale of 3)."

Answered by AI

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