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Gene therapy aims to stop severe infections in rare immune disorder

NCT ID NCT03837483

Disease control Sponsor: Fondazione Telethon Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, then given back as a one-time infusion. The study will check if this reduces severe infections and bleeding episodes compared to before treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
OTL-103 (gene therapy using the patient's own blood stem cells modified to carry a working copy of the WAS gene)
What this could lead to
If successful, this could offer a one-time treatment that reduces severe infections and bleeding episodes in people with Wiskott-Aldrich syndrome, potentially improving long-term health.
What could go wrong
This is a small early-phase trial with only 10 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or the modified cells not lasting long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

10 people

The number who actually took part.

Started

Jan 2019

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age: up to 65 years * Diagnosis of WAS defined by genetic mutation and at least one of the following criteria: * Severe Wiskott-Aldrich Syndrome (WAS) gene mutation, defined by literature data (genotype/phenotype studies).; * Absent WASP expression, assessed by flow cytometry; * Severe clinical score (Zhu clinical score ≥ 3); * No human leukocyte antigen (HLA)-identical related donor available for hematopoietic stem cells transplant (HSCT). Exclusion Criteria: * End-organ dysfunction, severe active infection not responsive to treatment or other severe disease or clinical condition which, in the judgment of the investigator, would make the patient inappropriate for entry into this study. * Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome. * Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia , or other serious haematological disorders * Documented human immunodeficiency virus (HIV) infection * Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin * Symptomatic herpes zoster, not responsive to specific treatment * Evidence of acute tuberculosis * Acute or chronic stable Hepatitis B * Presence of positive Hepatitis C RNA test result at screening * Patients not eligible for mobilization protocols in order to obtain CD34+ cells * Previous Gene Therapy

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Conditions

The condition(s) this trial relates to.

Wiskott-Aldrich syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Wiskott-Aldrich syndrome

Contacts and locations

Locations

  • Children's Healthcare of Atlanta, Inc

    Atlanta, Georgia, 30329, United States

  • Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

    Milan, 20132, Italy

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