Gene Therapy for Wiskott-Aldrich Syndrome

Recruiting at 1 trial location

Overseen By Orchard Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Fondazione Telethon

Disqualifiers: Cancer, HIV, Hepatitis B, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

10 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What data supports the effectiveness of the treatment OTL-103 for Wiskott-Aldrich Syndrome?

Research shows that gene therapy for Wiskott-Aldrich Syndrome, like OTL-103, has led to improvements in symptoms such as eczema, infections, and bleeding issues. Patients in clinical trials experienced better immune function and platelet counts, indicating the treatment's effectiveness. ^{1 2 3 4 5}

Is gene therapy for Wiskott-Aldrich Syndrome safe?

Gene therapy for Wiskott-Aldrich Syndrome has shown an acceptable safety profile in clinical trials, with patients experiencing clinical improvements and no serious safety concerns directly linked to the therapy. In animal studies, the therapy did not affect lifespan, and any tumors observed were not associated with the treatment. ^{1 2 3 4 5}

How is the treatment OTL-103 for Wiskott-Aldrich Syndrome different from other treatments?

OTL-103 is a gene therapy that uses a lentiviral vector to modify the patient's own stem cells, offering a potentially curative option for those without a well-matched donor for stem cell transplantation. This approach avoids complications like graft-versus-host disease and has shown improvements in immune function and symptoms like eczema and bleeding. ^{1 3 4 5 6}

What is the purpose of this trial?

This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.

Research Team

Orchard Clinical Trials

Principal Investigator

Orchard Therapeutics (Europe) Limited

Eligibility Criteria

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.

Inclusion Criteria

I have been diagnosed with WAS through genetic testing.

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

I am 65 years old or younger.

Exclusion Criteria

I have stable Hepatitis B.

Documented human immunodeficiency virus (HIV) infection

I have shingles that didn't improve with treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Gene Therapy Treatment

Participants receive OTL-103 gene therapy, involving autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the WAS gene

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including successful engraftment and hematological reconstitution

6 months

Treatment Details

Interventions

• OTL-103

Trial Overview 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.

Participant Groups

Group I: Gene Therapy Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Fondazione Telethon

Lead Sponsor

Trials

20

Recruited

1,500+

Orchard Therapeutics

Lead Sponsor

Trials

23

Recruited

106,000+

Ospedale San Raffaele

Collaborator

Trials

86

Recruited

388,000+

References

1. United States pubmed.ncbi.nlm.nih.gov

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Stem-cell gene therapy for the Wiskott-Aldrich syndrome. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein. [2016]

6. England pubmed.ncbi.nlm.nih.gov

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. [2020]