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Gene Therapy

Single Arm Study for Chronic Granulomatous Disease

Phase 1 & 2

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

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up initiated from the time of the infusion of base-edited cells through 2 years post-infusion

Awards & highlights

Study Summary

This trial aims to see if editing a person's own stem cells can improve the function of white blood cells in individuals with Chronic Granulomatous Disease (CGD). The study will focus on males

Who is the study for?

This trial is for adult males with X-linked Chronic Granulomatous Disease (CGD), a rare immune disorder. Participants must have the specific CYBB gene mutation targeted by this study and be at least 18 years old.Check my eligibility

What is being tested?

The trial tests if base-edited stem cells can improve white blood cell function in CGD patients, reducing infections. It involves collecting participants' stem cells, editing them to correct the genetic defect, then returning these cells after chemotherapy.See study design

What are the potential side effects?

Potential side effects include those related to chemotherapy with busulfan (nausea, fatigue, hair loss), reactions from growth factors like Plerixafor and Filgrastim (bone pain, headache), and risks associated with stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ initiated from the time of the infusion of base-edited cells through 2 years post-infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~initiated from the time of the infusion of base-edited cells through 2 years post-infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and initiated from the time of the infusion of base-edited cells through 2 years post-infusion for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

To evaluate the efficacy of base-edited autologous CD34+ cells

To evaluate the safety of base-edited autologous CD34+ cells

Secondary outcome measures

Evaluate clinical efficacy

Evaluate efficacy in restoring NADPH oxidase function.

Evaluate the efficiency in restoring gp91phox expression.

+3 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single Arm StudyExperimental Treatment5 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Plerixafor

2011

Completed Phase 3

~720

Busulfan

2008

Completed Phase 3

~1120

Palifermin

2006

Completed Phase 3

~1200

Filgrastim

2000

Completed Phase 3

~3670

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

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

3,274 Previous Clinical Trials

5,484,350 Total Patients Enrolled

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

4 Previous Clinical Trials

111 Total Patients Enrolled

Frequently Asked Questions

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

What are the anticipated results that this research aims to achieve?

"The primary aim of this research, to be assessed approximately 12 months post-administration of base-altered cells, is to scrutinize the safety of autologous CD34+cells subject to genetic editing. The secondary objectives encompass appraising clinical efficacy by gauging infection rates and comorbidity progression in CGD patients, assessing the effectiveness of base-editing through quantifying gp91-expressing cell ratios, and determining the engraftment potential of genetically modified hematopoietic stem progenitors via measuring edited myeloid cell proportions."

Answered by AI

Is the enrollment for this study currently available to prospective participants?

"The current status on clinicaltrials.gov indicates that recruitment for this particular study is closed. This trial was initially listed on 3/27/2024 and last revised on 3/21/2024. Despite the closure of this trial, there are currently 20 other trials actively seeking participants."

Answered by AI

Are individuals younger than 50 eligible for participation in this medical research study?

"Participants in this research must fall within the age range of 18 to 75 years. There are a total of 15 ongoing trials catering to individuals under 18 years old and an additional 14 for those above the age of 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

2024. "Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06325709.

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