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Gene Therapy

Gene Therapy for Wiskott-Aldrich Syndrome

Phase 3

Waitlist Available

Research Sponsored by Fondazione Telethon

Eligibility Criteria Checklist

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

Must have

Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:

No human leukocyte antigen (HLA)-identical related donor available for hematopoietic stem cells transplant (HSCT)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

Study Summary

This trial is testing a new gene therapy for Wiskott-Aldrich Syndrome, a rare blood disorder. The therapy uses patients' own stem cells, which are modified with a new gene, and then re-injected.

Who is the study for?

This trial is for individuals up to 65 years old with Wiskott-Aldrich Syndrome (WAS), confirmed by genetic mutation and severe symptoms or lack of WAS protein expression. Candidates must not have an identical donor for stem cell transplant, nor any end-organ dysfunction, active infections unresponsive to treatment, serious blood disorders, HIV, prior transplants with remaining donor cells, tuberculosis, hepatitis B or C infection, inability to mobilize stem cells for collection, cancer history except local skin cancer without hereditary syndrome or previous gene therapy.Check my eligibility

What is being tested?

The study tests a cryopreserved version of OTL-103 Gene Therapy in patients with WAS. OTL-103 uses the patient's own blood-forming stem cells modified by a lentiviral vector to correct the faulty gene responsible for WAS. It's an open-label study where all participants receive this single intervention without comparison groups.See study design

What are the potential side effects?

While specific side effects are not listed here, gene therapies like OTL-103 may cause immune reactions as the body adjusts to newly introduced genes; potential risks include inflammation at injection sites and general flu-like symptoms.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with WAS through genetic testing.

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I don't have a matching family donor for a stem cell transplant.

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I am 65 years old or younger.

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My tests show I lack WASP protein.

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I have a severe form of Wiskott-Aldrich Syndrome according to genetic tests.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 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

Annualized rate of moderate and severe bleeding episodes up to 1 year after gene therapy compared with 1 year before gene therapy

Annualized rate of severe infections from 6 to 18 months after gene therapy compared with 1 year before gene therapy

Secondary outcome measures

Evaluation of the overall survival

Number of participants with successful engraftment of OTL-103

Cells

+3 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Gene TherapyExperimental Treatment1 Intervention

OTL-103, Autologous CD34+ hematopoietic stem cells transduced ex vivo with a lentiviral vector encoding the human WAS gene

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

OTL-103

2010

Completed Phase 2

~10

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Fondazione TelethonLead Sponsor

17 Previous Clinical Trials

939 Total Patients Enrolled

1 Trials studying Wiskott-Aldrich Syndrome

8 Patients Enrolled for Wiskott-Aldrich Syndrome

Ospedale San RaffaeleOTHER

74 Previous Clinical Trials

367,968 Total Patients Enrolled

1 Trials studying Wiskott-Aldrich Syndrome

8 Patients Enrolled for Wiskott-Aldrich Syndrome

Orchard TherapeuticsLead Sponsor

22 Previous Clinical Trials

181,436 Total Patients Enrolled

2 Trials studying Wiskott-Aldrich Syndrome

6,008 Patients Enrolled for Wiskott-Aldrich Syndrome

Media Library

OTL-103 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03837483 — Phase 3

Wiskott-Aldrich Syndrome Research Study Groups: Gene Therapy

OTL-103 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03837483 — Phase 3

Wiskott-Aldrich Syndrome Clinical Trial 2023: OTL-103 Highlights & Side Effects. Trial Name: NCT03837483 — Phase 3

Frequently Asked Questions

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

Are patients being recruited for participation in this research project at present?

"No, this particular clinical trial is not presently looking for patients to participate. The most recent update on the study was September 27th, 2022 and it was originally posted on January 21st, 2019. Although this specific study isn't recruiting right now, there are 1,446 other studies that are seeking participants."

Answered by AI

When might OTL-103 receive FDA approval?

"OTL-103 has been given a score of 3 for safety by our team at Power. This is because OTL-103 is in Phase 3 clinical trials, which means that there is some data to support its efficacy and multiple rounds of data supporting its safety."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

2019. "A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03837483.