Ongoing Clinical Trials for Immunodeficiency
Currently, there is 1 ongoing clinical trial investigating new treatments for immunodeficiency disorders. This trial focuses on gene therapy for Wiskott-Aldrich Syndrome, a rare genetic condition that affects the immune system and blood clotting. The study is evaluating the safety and effectiveness of a gene therapy approach that modifies patients’ own cells to correct the underlying genetic defect.
Clinical trial locations
Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome
This clinical trial is investigating a novel gene therapy treatment called OTL-103 for patients with Wiskott-Aldrich Syndrome, a rare genetic disorder that weakens the immune system and causes problems with blood clotting. The condition leads to frequent infections, easy bruising, bleeding, and other serious health complications.
Who can participate:
- Male patients with a confirmed diagnosis of Wiskott-Aldrich Syndrome through genetic testing
- The diagnosis must be supported by at least one of the following: a severe mutation in the WASP gene, absence of WASP protein production in the body, or a severe clinical score of 3 or higher based on the Zhu clinical scoring system
- Patients who do not have an available family member with matching tissue type for a potential transplant
Who cannot participate:
- Patients who do not have Wiskott-Aldrich Syndrome
- Female patients, as only males are eligible
- Patients outside the specified age range, though the exact age limits are not detailed in the available information
What the trial involves:
The study uses a gene therapy approach where patients’ own stem cells are collected from their blood and modified in a laboratory. The modification involves using a special viral vector to introduce a healthy version of the WAS gene into these cells. Once modified, the cells are given back to the patient through an intravenous infusion.
Before receiving the gene therapy, participants undergo preparation with several medications. Rituximab is given through an intravenous infusion to help manage the immune system, and plerixafor is administered as an injection under the skin to help mobilize stem cells into the blood for collection. After stem cells are collected and modified, patients receive conditioning treatment with busulfan and fludarabine phosphate through intravenous infusions. These medications prepare the body to receive the modified cells by reducing immune system activity. After the gene therapy is given, patients may receive lenograstim injections to help the body produce more blood cells during recovery.
Main goal of the study:
The primary purpose of this trial is to evaluate whether OTL-103 gene therapy can safely and effectively reduce the frequency of severe bleeding episodes and infections in people with Wiskott-Aldrich Syndrome. Participants will be monitored closely for several years, with key assessments conducted at 12, 24, and 36 months after receiving the treatment. Throughout the study, researchers will track participants’ overall health, monitor for any side effects, and measure improvements in immune system function.
Summary
Currently, only one clinical trial is actively recruiting patients with immunodeficiency related to Wiskott-Aldrich Syndrome. This trial is being conducted in Italy and represents a promising approach using advanced gene therapy technology. The study focuses on OTL-103, a treatment that modifies patients’ own cells to address the genetic cause of the disease rather than just managing symptoms. The trial involves a comprehensive treatment protocol that includes cell collection, genetic modification, and supportive medications to help the body accept and benefit from the therapy. This research may offer hope for patients who do not have a suitable donor for traditional transplant options.
