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Gene Transfer

Gene Therapy for Wiskott-Aldrich Syndrome

Q: Is this clinical trial open to individuals aged sixty and beyond?

Any prospective patients must be between 3 months and 35 years old. There are 375 clinical trials available to minors, while those aged 65 or over have access to 983 different studies.

Q: What is the recommended demographic for participants in this experiment?

To meet the entry criteria of this clinical trial, participants must have Wiskott-Aldrich Syndrome and fall within 3 months to 35 years old. The total number of required patients is 5.

Q: Are there opportunities to participate in this trial at the present moment?

Unfortunately, this trial is not actively recruiting anymore. It was originally listed on July 1st 2011 and most recently updated on the 19th of July 2022. However, there are a plethora of other medical studies that have opened up their enrolment process to patients at present time.

Phase 1 & 2

Waitlist Available

Led By Jennifer Whangbo, M.D.

Research Sponsored by David Williams

Eligibility Criteria Checklist

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

Must have

For subjects 5 years of age or older: Lack of HLA-genotypically identical bone marrow donor

For subjects < 5 years of age: Lack of HLA-genotypically identical bone marrow donor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study Summary

This trial is for boys with Wiskott-Aldrich Syndrome, an inherited disorder that causes defects in the blood and bone marrow. The goal is to see if gene transfer, a new treatment, is safe and effective.

Who is the study for?

This trial is for boys with Wiskott-Aldrich Syndrome, who have a confirmed diagnosis and no suitable bone marrow donor after searching for 3 months. They must be aged between 3 months to 35 years, willing to follow up for 5 years, and have good heart function and overall health. Those previously transplanted need specific criteria met.Check my eligibility

What is being tested?

The study tests a gene transfer treatment using the patient's own bone marrow cells that are modified by retrovirus-mediated gene transfer to correct the genetic defect causing WAS. This could potentially lead to a healthy immune system without needing an external donor.See study design

What are the potential side effects?

Potential side effects may include reactions similar to those seen in standard bone marrow transplants such as infection risk due to low immunity during the procedure, chemotherapy-related issues like nausea or hair loss, and complications from the gene transfer itself.

Eligibility Criteria

Inclusion Criteria

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

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I am over 5 years old and do not have a matching bone marrow donor.

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I am under 5 and do not have a matching bone marrow donor.

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I can do most of my daily activities by myself.

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My lung function tests are above 50% of what's expected for my age.

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I am between 3 months and 35 years old.

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My kidney function, measured by creatinine clearance or GFR, is adequate.

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My condition was confirmed by DNA test and I either lack WAS protein or have a clinical score of 3-5.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 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

Engraftment of genetically corrected T cells

Safety of infusion of transduced cells

Trial Design

1Treatment groups

Experimental Treatment

Group I: Gene transferExperimental Treatment1 Intervention

Open label single arm study

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

David WilliamsLead Sponsor

4 Previous Clinical Trials

53 Total Patients Enrolled

Jennifer Whangbo, M.D.Principal InvestigatorBoston Children's Hospital

Frequently Asked Questions

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

Is this clinical trial open to individuals aged sixty and beyond?

"Any prospective patients must be between 3 months and 35 years old. There are 375 clinical trials available to minors, while those aged 65 or over have access to 983 different studies."

Answered by AI

What is the recommended demographic for participants in this experiment?

"To meet the entry criteria of this clinical trial, participants must have Wiskott-Aldrich Syndrome and fall within 3 months to 35 years old. The total number of required patients is 5."

Answered by AI

Are there opportunities to participate in this trial at the present moment?

"Unfortunately, this trial is not actively recruiting anymore. It was originally listed on July 1st 2011 and most recently updated on the 19th of July 2022. However, there are a plethora of other medical studies that have opened up their enrolment process to patients at present time."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

David Williams 2011. "Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01410825.