Cultured Thymus Tissue for DiGeorge Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial observes how RETHYMIC (cultured thymus tissue) aids patients with congenital athymia, a condition where the thymus, crucial for the immune system, is absent. Researchers aim to understand how this treatment supports immune system recovery over time and its effects on long-term survival and side effects. The trial includes those diagnosed with congenital athymia who have recently received or are about to receive RETHYMIC. Participants will provide insights into the treatment's impact on their health and daily life. As a Phase 4 trial, this research highlights that RETHYMIC is already FDA-approved and proven effective, aiming to understand its benefits for more patients.
Do I need to stop my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What is the safety track record for this treatment?
Research has shown that RETHYMIC, which uses specially grown thymus tissue, can help rebuild the immune system and extend life in patients born without a thymus. Studies indicate that this treatment often leads to the immune system starting to function again, which is a positive sign.
Regarding safety, these studies have not consistently reported any serious side effects, suggesting that the treatment is generally well-tolerated. However, like any treatment, side effects can occur, which is why this registry closely monitors any specific side effects that might develop over time.
In summary, while current research suggests the treatment is safe, ongoing monitoring of patients is important to ensure the best outcomes.
Why are researchers excited about this trial?
Researchers are excited about the cultured thymus tissue treatment for congenital athymia because it offers a novel approach to tackling this condition. Unlike standard treatments that often focus on managing symptoms or providing supportive care, this therapy involves implanting cultured thymus tissue into patients. This method aims to address the root cause by potentially restoring immune function in individuals who lack a thymus. By targeting the body's ability to produce T-cells, the treatment could significantly enhance the quality of life and long-term health outcomes for those affected by congenital athymia.
What evidence suggests that RETHYMIC treatment might be effective for congenital athymia?
Research has shown that RETHYMIC, which uses specially grown thymus tissue, can help patients born without a thymus by rebuilding their immune systems. This enhancement enables their bodies to fight infections more effectively, which is crucial for these patients. Studies have found that this treatment can extend life, significantly improving survival rates. In this trial, all eligible patients will receive RETHYMIC, with a focus on rebuilding the immune system to provide a better chance at a healthier life.
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
Treatment
Patients receive RETHYMIC treatment for immune reconstitution
Follow-up
Participants are monitored for safety, immune reconstitution, and adverse events
Long-term follow-up
Monitoring of long-term survival and adverse events of special interest
What Are the Treatments Tested in This Trial?
Interventions
- Cultured Thymus Tissue
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
All eligible patients who survived 1 year post treatment
All eligible patients
Find a Clinic Near You
Who Is Running the Clinical Trial?
Enzyvant Therapeutics GmBH
Lead Sponsor
Sumitomo Pharma Switzerland GmbH
Lead Sponsor
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