Elivaldogene Autotemcel for Adrenoleukodystrophy
(Stargazer Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on understanding the long-term safety and effectiveness of a treatment called eli-cel (Skysona) for individuals with cerebral adrenoleukodystrophy (CALD). Researchers aim to assess how well participants manage without major disabilities over time and monitor for any new health issues. To join, participants must already be receiving eli-cel treatment in the U.S. and be willing to provide regular health updates. The trial will closely examine a specific group with more severe early-stage CALD to evaluate the treatment's impact. As a Phase 4 trial, this research emphasizes that the treatment is already FDA-approved and effective, aiming to understand its benefits for more patients.
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 your doctor.
What is the safety track record for this treatment?
Research has shown that the treatment elivaldogene autotemcel, or Skysona, has been tested in patients with early active cerebral adrenoleukodystrophy (CALD). In these studies, patients generally tolerated the treatment well. Most patients remained alive and free from major disabilities for up to seven years after receiving it, suggesting long-term safety.
While researchers continue to study any long-term effects, some patients have experienced side effects, which is common with many treatments. Importantly, the FDA has approved Skysona for CALD, indicating it has met certain safety standards. However, patients should discuss potential risks with their healthcare provider before joining any clinical trial.12345Why are researchers excited about this trial?
Elivaldogene autotemcel is unique because it uses a gene therapy approach to treat cerebral adrenoleukodystrophy (CALD), a rare and severe brain disorder. Unlike traditional treatments, which might include steroids or bone marrow transplants, this therapy works by delivering a functional copy of the ABCD1 gene directly into the patient's cells. Researchers are excited because this could potentially halt the disease's progression at its source, offering hope for improved outcomes and long-term benefits for those affected by CALD.
What is the effectiveness track record for eli-cel in treating CALD?
Research has shown that eli-cel, also known as Skysona, effectively treats cerebral adrenoleukodystrophy (CALD). In earlier studies, patients who received eli-cel showed significant improvement in survival without major disabilities for up to 24 months. The treatment uses a special virus to modify certain blood cells, which then help produce the missing protein needed by CALD patients. Results suggest that the benefits of eli-cel outweigh the risks, with some patients followed for over five years. These findings support eli-cel's effectiveness in improving the condition of CALD patients. Participants in this trial will receive eli-cel and be followed for up to 15 years to collect real-world longitudinal data and evaluate outcomes.14678
Who Is on the Research Team?
Kiran Bhirangi, MD
Principal Investigator
bluebird bio, Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Post-infusion Follow-up
Participants are monitored for safety outcomes, including newly diagnosed malignancies, and MFD-free survival over time
Long-term Monitoring
Participants continue to be monitored for adverse events and effectiveness outcomes in a real-world setting
What Are the Treatments Tested in This Trial?
Interventions
- Elivaldogene Autotemcel
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants with CALD treated with eli-cel in the post marketing setting will be followed in this registry study for up to 15 years after eli-cel infusion to collect real-world longitudinal data, and evaluate the outcomes.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genetix Biotherapeutics Inc.
Lead Sponsor
bluebird bio
Lead Sponsor
Center for International Blood and Marrow Transplant Research
Collaborator
Citations
NCT06224413 | A Study of Participants With Cerebral ...
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in ...
Elivaldogene autotemcel approved for treatment of cerebral ...
Outcomes and efficacy. Cartier et al demonstrated that autologous transplant of lentiviral vector engineered CD34+ cells led to functional production of ALD ...
Evaluation of Mobilization, Apheresis, and Conditioning ...
These data demonstrate the utility of these approaches in achieving sufficient CD34+ cell yields and engraftment for CALD gene therapy.
Interim Results from Phase 2/3 (ALD-102) and ...
As of February 2020, 45 patients with CALD received eli-cel. The treatment showed a favorable benefit/risk profile with up to 71 months follow-up in ALD-102/ ...
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
The primary efficacy end point was survival without any of six major functional disabilities at month 24. The secondary end points included ...
A Study of Participants With Cerebral Adrenoleukodystrophy ...
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel ...
Results from clinical studies
SKYSONA was tested in 2 clinical studies to determine efficacy and safety in patients with early active cerebral ALD. Please see Important Safety ...
8.
investor.bluebirdbio.com
investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-presents-long-term-data-elivaldogene-autotemcel-elibluebird bio Presents Long-Term Data for elivaldogene ...
Patients in long-term follow-up study (LTF-304) continue to remain alive and MFD-free through up to nearly seven years of follow-up, suggesting eli-cel ...
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