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mRNA Therapy

gp91 Grans for Chronic Granulomatous Disease

Phase 1

Recruiting

Led By Suk S De Ravin, M.D.

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

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

Must have

CGD confirmed by DHR and gp91phox-deficiency subtype confirmed by protein analysis and/or genetic sequencing

Males aged 18 to 75 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 months

Awards & highlights

Study Summary

This trial is testing a new procedure to see if it can help people with a certain gene mutation kill germs better.

Who is the study for?

This trial is for males aged 18-75 with Chronic Granulomatous Disease (CGD) due to a gp91phox gene mutation. Participants must be able to consent, have follow-up care arranged, and agree to use contraception if they can cause pregnancy.Check my eligibility

What is being tested?

The study tests a procedure where mRNA is added to blood cells in CGD patients. It aims to correct the gene defect temporarily so cells can better fight infections. Patients will undergo cell collection, receive mRNA-corrected cells via IV, and be monitored.See study design

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions related to IV infusion such as discomfort at the injection site or an immune response against the modified cells.

Eligibility Criteria

Inclusion Criteria

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

Select...

My CGD and its gp91phox-deficiency subtype are confirmed by specific tests.

Select...

I am a man between 18 and 75 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 months for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.

MTD determination based on the rate of AEs. MTD is defined as the highest dose level that does not cause the same grade 3 or 4 AEs in 3 or more patients

Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent

Secondary outcome measures

Determine percent of circulating dihydrorhodamine (DHR) positive granulocytes following study agent infusion.

Serial measurement of circulating DHR+ granulocytes from peripheral blood until day 3 following study agent infusion or disappearance of DHR+ granulocytes.

Trial Design

3Treatment groups

Experimental Treatment

Group I: IV infusion of gp91-Grans at dose K: 1e6 cells/kgExperimental Treatment1 Intervention

Adult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K, and safety of dose will be determined.

Group II: IV infusion of gp91-Grans at dose K+2: 1-5e8 cells/kgExperimental Treatment1 Intervention

Group III: IV infusion of gp91-Grans at dose K+1:1e7 cells/kgExperimental Treatment1 Intervention

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor

3,269 Previous Clinical Trials

5,481,444 Total Patients Enrolled

Suk S De Ravin, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)

4 Previous Clinical Trials

96 Total Patients Enrolled

Media Library

NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells (mRNA Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05189925 — Phase 1

Frequently Asked Questions

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

Has the FDA ratified gp91 Grans for public use?

"At this stage of the clinical trial process, there is limited data available to support gp91 Grans' safety and efficacy, leading our team at Power to rate it a 1 on a scale from 1-3."

Answered by AI

Are individuals of advanced age being allowed to partake in the experiment?

"This clinical trial requires that potential participants meet certain age requirements, with a lower limit of 18 years old and an upper cap of 75."

Answered by AI

How extensive is the participant pool of this clinical investigation?

"Affirmative, clinicaltrials.gov indicates that this research is actively looking for participants. The trial was published on July 22nd 2022 and has been amended as recently as November 24th 2022. 25 individuals are needed from a single location."

Answered by AI

Is it possible to enroll in this research at present?

"As per the clinicaltrials.gov database, this investigation is actively looking for participants. This study was first put up on July 22nd 2022 and recently amended on November 24th of that year."

Answered by AI

Is it possible to become a participant in this clinical experiment?

"A cohort of 25 individuals, aged between 18 and 75 with an active infection may be eligible for inclusion in this study. In addition to meeting the age requirement, participants must have a CGD diagnosis confirmed by DHR testing, gp91phox-deficiency subtype verified through protein analysis or genetic sequencing, access to medical care at home during follow up visits, willing to provide informed consent as well as use hormonal contraception/condoms."

Answered by AI

Recent research and studies

Journal of Allergy and Clinical ImmunologyJournal

Granulocyte Transfusions in Patients with Chronic Granulomatous Disease and Refractory Infections: The NIH Experience

Marciano, Beatriz E., Elisabeth S. Allen, Cathy Conry-Cantilena, Ervand Kristosturyan, Harvey G. Klein, Thomas A. Fleisher, Steven M. Holland, Harry L. Malech, and Sergio D. Rosenzweig. 2017. “Granulocyte Transfusions in Patients with Chronic Granulomatous Disease and Refractory Infections: The NIH Experience”. Journal of Allergy and Clinical Immunology. Elsevier BV. doi:10.1016/j.jaci.2017.02.026.

TransfusionJournal

Alloimmunization After Granulocyte Transfusions

Stroncek, D. F., K. Leonard, G. Eiber, H. L. Malech, J. I. Gallin, and S. F. Leitman. 1996. “Alloimmunization After Granulocyte Transfusions”. Transfusion. Wiley. doi:10.1046/j.1537-2995.1996.36111297091747.x.

TransfusionJournal

The Relationship Between Alloimmunization and Posttransfusion Granulocyte Survival: Experience in a Chronic Granulomatous Disease Cohort

Heim, K.F., T.A. Fleisher, D.F. Stroncek, S.M. Holland, J.I. Gallin, H.L. Malech, and S.F. Leitman. 2010. “The Relationship Between Alloimmunization and Posttransfusion Granulocyte Survival: Experience in a Chronic Granulomatous Disease Cohort”. Transfusion. Wiley. doi:10.1111/j.1537-2995.2010.02993.x.

clinicaltrials.govStudy