Stem Cell Transplant + Drug Therapy for Chronic Granulomatous Disease

This trial aims to improve the success of stem cell transplants for individuals with chronic granulomatous disease (CGD), a condition that weakens the immune system and increases the likelihood of frequent infections. Researchers seek to determine if a new combination of drug treatments, including Alemtuzumab (an immunosuppressive drug), Busulfan (a chemotherapy drug), Cyclophosphamide (a chemotherapy drug), and Emapalumab-Izsg (an immunotherapy drug), can enhance the effectiveness of stem cell transplants. Participants will receive a series of medications and radiation before receiving donor stem cells through a catheter. This trial may suit those with CGD who experience complications like ongoing inflammation or infections despite preventive treatment. Participants must stay near the hospital for follow-up care after the transplant. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

The trial protocol 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 to get specific guidance based on your situation.

Research shows that the treatments in this clinical trial have varying safety levels. Here's a simple breakdown:

Alemtuzumab has undergone extensive study. It can increase the risk of infections and other complications, including a higher chance of death from infections. However, it has been used successfully for other conditions, so its risks are known and manageable.

Busulfan is often used in stem cell transplants and is carefully monitored to achieve the best results. It is administered in precise doses to minimize side effects, but it can temporarily impair the body's ability to produce enough blood cells.

Emapalumab generally has a good safety record, with most people tolerating it well. However, serious side effects like infections can still occur. It is approved for another condition, so doctors understand its effects on the body.

Tocilizumab is another drug used in this trial. While it can lead to serious infections, its safety has been well-documented in various conditions. It is commonly used in medical settings, which helps doctors manage any side effects.

Total Body Irradiation is part of the treatment plan for this trial. Although it can cause side effects, it is a standard component of many stem cell transplant procedures and effectively prepares the body to accept new cells.

Researchers are excited about this treatment for Chronic Granulomatous Disease (CGD) because it combines stem cell transplants with innovative drug therapies that enhance the immune system in unique ways. Unlike traditional treatments that focus mainly on antibiotics and antifungals to manage infections, this approach uses Alemtuzumab, Busulfan, and Cyclophosphamide to prepare the body for a stem cell transplant, potentially offering a long-term solution. Additionally, Emapalumab-Izsg targets specific immune pathways to reduce inflammation, which is not a focus of current standard therapies. This combination not only aims to replace defective immune cells but also modulates the immune response, offering a comprehensive approach to tackling CGD.

Research has shown that stem cell transplants can effectively cure chronic granulomatous disease (CGD), but they don't always succeed on their own. In this trial, participants in different arms will receive various treatment combinations to improve success rates.

In Arm 1, participants will receive alemtuzumab and busulfan, which have shown promise in reducing complications like acute GVHD (graft-versus-host disease) and improving transplant outcomes.

In Arm 2, participants will receive emapalumab, which has been effective in controlling severe immune responses in other conditions, potentially aiding successful transplants.