Charlotte, NCNCT05477563Now EnrollingIRB Ready

Sickle Cell Disease Clinical Trial in Charlotte, NC

Access cutting-edge sickle cell disease treatment through this clinical trial at a research site in Charlotte. Study-provided care at no cost to qualified participants.

Sponsored by Vertex Pharmaceuticals Incorporated

Quick Self-Assessment

See if you qualify for this Charlotte location

Preparing your pre-screening questions...

Expert Care in Charlotte

Access sickle cell disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related sickle cell disease treatment provided free

Apply for This Charlotte Location

Check if you qualify for this sickle cell disease clinical trial in Charlotte, NC

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Charlotte

    Convenient for NC residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Charlotte site if eligible
  4. 4Begin participation

About This Sickle Cell Disease Study in Charlotte

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Sponsor: Vertex Pharmaceuticals Incorporated

Who Can Participate

Inclusion Criteria

Participants with TDT and SCD:
Eligible for autologous stem cell transplant as per investigator's judgment.
Participants with TDT:
Diagnosis of TDT as defined by:
Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
Participants with SCD:
Diagnosis of severe SCD as defined by:
Documented SCD genotypes
History of at least two severe VOCs events per year for the previous two years prior to enrollment Key

Exclusion Criteria

Participants with TDT and SCD:
A willing and healthy

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Charlotte?

Yes, this clinical trial (NCT05477563) has an active research site in Charlotte, NC that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Sickle Cell Disease Treatment Options in Charlotte, NC

If you're searching for sickle cell disease treatment options in Charlotte, NC, this clinical trial (NCT05477563) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Charlotte research site is actively enrolling participants for this clinical trial. You'll receive care from experienced sickle cell disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all sickle cell disease clinical trials near you to find additional studies recruiting in your area.

More Ankylosing Spondylitis Trials in Charlotte, NC

See all ankylosing spondylitis clinical trials recruiting in Charlotte — not just this study.

Browse Ankylosing Spondylitis Trials in Charlotte →

Browse More Trials by Condition

Ankylosing Spondylitis TrialsInterstitial Cystitis TrialsSickle Cell Disease TrialsAll Conditions →

Ready to Join in Charlotte?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Charlotte, NC