Image

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Testing gene correction in stem cells for severe sickle cell treatment.

Recruiting
12-50 years
All
Phase 1/2
  • Simplified description (AI rewritten)
  • Technical description (Original)

This research is for people with severe Sickle Cell Disease (SCD), a blood disorder where red blood cells get misshaped and cause pain. The study will test a treatment that changes genes in blood stem cells. These stem cells are called **CD34+ Hematopoietic Stem Cells**. They will be taken from the participant, fixed to make normal hemoglobin (**HbA**), and then returned through an **IV infusion** after strong medicine helps prepare the body.

**Key Points:**

  • Participants must be between 12 and 40 years old.
  • The study involves a treatment that uses your own stem cells.
  • Participants cannot have had previous stem cell transplants or gene therapy.

Eligible participants need to have had multiple SCD crises or complications, like **VOC** (pain crises) or **ACS** (lung problems). They should also have a good performance status, meaning they can do daily activities well. Some risks include not having a matching sibling donor or prior health issues like cancer or infections. It's important for those interested to discuss all risks and benefits with their doctor.

Print
Study details
    Sickle Cell Disease

NCT04819841

Kamau Therapeutics

1 August 2026

Contact a study center
Step 1 Get in touch with the nearest study center
We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Email

View email

Phone

Phone country flag

View phone
Investigator Avatar

Primary Contact

  Other languages supported:

First name*
Last name*
Email*
Phone number*
Other language

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.