Image

Conditioning SCID Infants Diagnosed Early

Early care for babies with SCID in clinical trial.

Recruiting
2 years and younger
All
Phase 2

Study Overview: This study is focusing on babies with Severe Combined Immunodeficiency (SCID), a condition where babies have very weak immune systems. Researchers are testing if low doses of chemotherapy drugs can help these babies build a stronger immune system with fewer side effects after they receive a bone marrow transplant. The study uses two different doses of a chemotherapy drug called busulfan. Babies will get their transplants early in life, using donor cells that are not a perfect match but are safe to use. After the transplant, babies will be monitored for 3 years to see how well their immune systems recover.

Key Points:

  • Babies will be followed for 3 years after the transplant to monitor immune recovery.
  • The study is only for babies using unrelated or partially matched donor cells.
  • Vaccinations will be given and tested 9-18 months post-transplant to check immune response.

Please consult with healthcare providers to understand if this study is suitable for your child and discuss any potential risks or benefits.

Study details
    SCID

NCT03619551

Michael Pulsipher, MD

10 October 2024

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

  Other languages supported:

FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

Do I get compensated for taking part in clinical trials?

Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.