CHARLOTTE, NC

Sickle Cell Disease Clinical Trials

4 sickle cell studies are enrolling in Charlotte NC. Notably, none of them uses a dummy treatment. Also, one question decides which of the two treatment studies is open to you. Compensation is provided for eligible participants.

Enrolling in Charlotte

With a Placebo

+/- $100

Per Visit

New here? Read how this page works

First, these four are not a menu you pick from. One question routes you before anything else. So here is how to work out which door is yours.

Ask whether anyone has been HLA typed. Because that one blood test decides which of the two treatment studies is open to you.
Check the ages carefully. Notably, the transplant study takes 2 to 25 and the gene editing study takes 12 to 35, so age alone can route you.
Know your exact type. In fact, one study takes only two of the genotypes, while another takes every one.
Finally, tap the button. Then we check you against all of them at once and tell you when a new one opens.

Of course, checking never signs you up for anything. You are only seeing where you might fit.

The 4 Sickle Cell Studies Enrolling in Charlotte

Notably, all four run inside Charlotte. However, two of them are mutually exclusive by design, so read the first group carefully.

One question decides which of these two you can join

Do you have a brother or sister who is a matched donor? Notably, one study rules you out if you do, and the other requires one. So HLA typing is the first step.

Gene Editing, Using Your Own Cells

Vertex Pharmaceuticals / Cell therapy

Your own blood forming stem cells are collected, edited in a lab, then given back. The edit switches back on the haemoglobin you made as a baby, and that kind does not sickle.

Late stage study of an already approved therapy No matched sibling

Payment is not published. Notably, ask the site about travel and lodging support.

Check eligibility →

More details

Why researchers are excited: because the cells are your own there is no rejection and no graft versus host disease. So this is the route for people with no matched brother or sister.

Who this study is looking for

Age 12 to 35
Also, severe sickle cell disease, with at least two bad pain crises a year for two years
Besides that, your doctor judges you fit enough for a transplant using your own cells
Finally, you have never had a stem cell transplant before

Probably not a fit if: you have a willing, healthy, fully matched brother or sister. That surprises families, and the reason is that a sibling transplant is the safer known route.

Placebo group: None at all. Every single person receives the real therapy, and everybody knows it. Also, your usual care carries on and in fact steps up beforehand.
How long: About a year of active process, then 12 months of measured follow up.
Clinic visits: Many months of near continuous hospital contact rather than occasional visits. The record names day 42, day 100 and month 12, with roughly 4 to 6 weeks as an inpatient.
Phone calls: Not published on the public record.
Most invasive part: Very. Months of transfusions first, then hours hooked to a machine to collect stem cells, then chemotherapy that wipes out your marrow, then a long hospital stay. Also, infertility is a known consequence, so fertility preservation is discussed first.
Setting: Outpatient.
Where it runs: Levine Children’s Hospital, Charlotte NC 28203.
Read this before anything else: gene therapy is not an injection. It is closer to a transplant using your own cells, and it takes most of a year.
Full study record: View on ClinicalTrials.gov →

The Old Cure, Made Much Gentler

Investigator led, with Levine Children’s / Transplant

A transplant from a matched brother or sister has cured sickle cell since the 1980s. This study asks whether the same cure works with a far gentler preparation.

Mid stage, 100 people Matched sibling needed

Payment is not published. Notably, the donor sibling has a procedure too.

Check eligibility →

More details

Why researchers are excited: the old preparation caused infertility, organ damage and a real risk of death. So making it safer means it could be offered earlier, before damage sets in.

Who this study is looking for

Age 2 to 25, with sickle cell disease
Also, you have a brother or sister who is an identical match, which is the whole premise
Besides that, your disease has already caused serious problems such as strokes, chest crises or repeated pain
Finally, your heart, lungs, liver and kidneys work well enough

Probably not a fit if: you have built up antibodies against your donor’s red cells from past transfusions. Notably, that catches heavily transfused people, which is one of the things that qualifies you.

Placebo group: None at all and no randomising. Everybody enrolled receives the transplant.
How long: The main check is at 100 days, and engraftment is judged at one year. Plan for a full year of intensive involvement.
Clinic visits: Not published as a total. The named points are day 30, day 100 and day 365, with frequent outpatient visits between, often several times a week early on.
Phone calls: Not published on the public record.
Most invasive part: Very. A low dose of total body radiation, an antibody drip, then tablets for at least a year. Also, a long hospital stay, a central line, and transfusions during recovery.
Setting: Outpatient.
Where it runs: Levine Children’s Hospital, Charlotte NC 28203.
Ask this first: has anybody in the family been HLA typed? That single blood test decides which of the two Charlotte studies is open to you.
Full study record: View on ClinicalTrials.gov →

Nothing is done to you in these

Notably, neither of these gives any treatment or changes your care. However, one of them takes almost anybody with this diagnosis.

Transplant or Medicines? Nobody Ever Compared Them

University of Rochester with PCORI / Observational

Nothing is given and nothing is changed. Researchers follow children going down each path and compare what actually happens over three years.

Observational, 480 children Nothing is done to you

Payment is not published. However, the study adds nothing to your child’s treatment.

Read the details →

More details

Why researchers are excited: this is the question every family asks, and astonishingly nobody has ever properly compared the two at scale in children.

Who this study is looking for

Age 3 to 20.9
Also, sickle cell anaemia or the S beta zero type, because other types are not included
Besides that, your child is heading for a matched sibling transplant, which is the arm open in Charlotte
Finally, the family agrees to three years of follow up

Probably not a fit if: your child has the SC type, which is common and sometimes serious but is not included here. Also, being in another trial that might interfere can rule you out.

Placebo group: None at all. There is no randomising and no study drug, so your child’s treatment is untouched.
How long: Three years of follow up per child.
Clinic visits: Clearly published. The transplant group is assessed before, then at day 100, 6 months, and years 1, 2 and 3. The medicines group is assessed at the start then yearly.
Phone calls: Not published on the public record.
Most invasive part: Low. Questionnaires and thinking and memory tests, plus collecting information already gathered. Notably, the study adds no transfusions, scans, chemotherapy or extra blood draws of its own.
Setting: Outpatient.
Where it runs: Atrium Health, Charlotte NC 28203.
Worth knowing: Charlotte is open only for the transplant side of this study. So a child staying on medicines would need a different site.
Full study record: View on ClinicalTrials.gov →

A Thirty Year Record of a Whole Life

St. Jude Children’s Research Hospital / Observational

Nothing is tested. Researchers record the results of tests people are having anyway, at ordinary clinic visits, across decades.

Observational, 10,000 people, running to 2044 Almost anybody qualifies

Payment is not published. However, the study creates no extra visits.

Read the details →

More details

Why researchers are excited: organ damage builds slowly and shows up in young adulthood, exactly when people move to adult care and often fall out of the system. So nobody has a good long run picture.

Who this study is looking for

Sickle cell disease of any type at all, which is the broadest rule on this page
Also, at the Charlotte site, age 0 to 18
Besides that, you are willing to have your routine results recorded
Finally, there is essentially no other requirement

Probably not a fit if: there is only one exclusion, and it is broad. The investigators can decide participation is unwise for you.

Placebo group: None at all, and no study drug of any kind. Your care carries on exactly as it is.
How long: Effectively for life. Data is gathered every 2 years up to age 30, then every 6 years after that.
Clinic visits: No extra visits are created. You attend the clinic appointments you would attend anyway, and data is captured at those.
Phone calls: Not published on the public record.
Most invasive part: Low. Blood draws that are already being done, plus swabs, stored samples every 6 years, and thinking and memory testing.
Setting: Outpatient.
Where it runs: Novant Health Hemby Children’s Hospital, Charlotte NC 28204.
Worth knowing: if you do not qualify for anything else here, this is very likely the one you can join. Also, it runs at a different hospital system from the other three.
Full study record: View on ClinicalTrials.gov →

Not sure which one fits you?

First, answer a few quick health questions. Then we check you against all 4 studies. Also, no account and no paperwork.

See if I may fit one of these studies →

✓ Takes just a few minutes✓ No cost, no obligation✓ You are never enrolled by checking

Why Sickle Cell Research Matters Here in Charlotte

A cure exists, and the hard part is getting one

Two gene therapies are approved. A transplant from a matched brother or sister has worked since the 1980s. So for the first time this is not a disease without a cure. However, only about one in six or seven people has a suitable sibling, and gene therapy takes most of a year and heavy chemotherapy. Meanwhile, the daily reality for most people is still pain crises and hospital visits.

What is actually being studied here

Gastonia, Rock Hill, Monroe, Concord, Kannapolis. Notably, sickle cell disease turns up across all of them, and care has long been underfunded next to how serious it is. However, Charlotte is genuinely strong here, with four studies running across two hospital systems. In fact, two of them cover the two halves of the same population rather than competing.

So why do families here fall behind?

Nobody gets HLA typed. That one blood test decides which studies are open. However, most families have never been offered it.
Gene therapy is misunderstood. People picture an injection. So they say yes before understanding it means chemotherapy and weeks in hospital.
Pain gets treated with suspicion. People in crisis are often doubted in emergency rooms. Meanwhile, that damages trust in the whole system.
The move to adult care loses people. Organ damage shows up in young adulthood, exactly when people fall out of paediatric care.
Over 35 has nothing here. The gene editing study stops at 35. So we say plainly that older adults have no open treatment study in Charlotte.

Research is a care option, not a last resort

There is a saying in medicine that research is a care option rather than a last resort. Generally, that is the honest way to think about a study. You are not giving up your own doctor. Instead, you are adding a second set of eyes. Notably, none of these four uses a dummy treatment, and two of them change nothing about your care at all.

What being on the list actually gets you

This is a directory, not a clinic. So being on the list is simple and it costs nothing.

What you actually get

A call from us the day a new study opens near you
The clinic name and number, so you call them yourself
A check against every other study on our list, not just this one
No cost, and no insurance needed
Roughly $100 a visit for your time, once you are in a study
You are never enrolled by checking

So add it up. Four studies inside Charlotte, none of them with a dummy treatment. Meanwhile, two real cures now exist, and the barrier is eligibility rather than availability. So the first useful step is not a decision. Instead, it is one blood test that tells you which door is yours.

What Is Actually Changing in Sickle Cell Disease

Two cures now exist. Getting one is the hard part.

What Goes Wrong

A change in one gene makes haemoglobin, the part of red cells that carries oxygen, stick together into stiff rods. So the cells bend into a curved shape and jam in small blood vessels. That causes sudden severe pain, damage to organs over time, and a shortage of red cells. Also, it is far more common in people of African ancestry, and care has long been underfunded.

Gene Therapy Arrived in 2023

In December 2023 the first two gene therapies were approved. One uses gene editing to switch back on the haemoglobin you made as a baby, which does not sickle. Nicole Verdun of the FDA called it exciting to advance the field for people whose lives had been severely disrupted. Then in July 2026 one of them was expanded down to age 2.

It Is Not an Injection

This is the part people are not told plainly. Gene therapy here is closer to a transplant using your own cells, and it takes most of a year. Months of transfusions, hours hooked to a machine collecting stem cells, months of lab work, then chemotherapy that wipes out your marrow and four to seven weeks in hospital.

Eligibility Is the Real Barrier

As Regina Hartfield of the Sickle Cell Disease Association of America put it, this is a historic milestone, but everyone may not be eligible for gene therapy. That single sentence is why this page is built the way it is. So the question is not whether a cure exists. Instead, it is whether a door is open to you.

The Older Cure Still Works

A transplant from a matched brother or sister has cured this disease since the 1980s. However, only about one in six or seven people has a suitable sibling. So the field is split down that line, and two Charlotte studies sit on opposite sides of it. In fact, one of them rules you out if you have a matched sibling, and the other requires one.

A Drug Was Also Taken Away

In September 2024 voxelotor was withdrawn worldwide over safety concerns. It had been a widely used daily tablet, so people who remember stopping it abruptly are remembering correctly. Meanwhile, the everyday treatments that remain are hydroxyurea, glutamine, an infusion that stops cells sticking, pain care, transfusions, and yearly brain scans in children to catch stroke risk early.

Research Sites Near Charlotte

Generally, Levine Children’s, Atrium Health and Novant Health Hemby carry these between them. But sites add new studies all the time, so it is worth knowing what sits near you.

Atrium Health

Multiple Charlotte locations / 161+ active studies

Crohn’s, Heart Failure, COPD, Dementia, Type 2 Diabetes, Hypertension

Charlotte’s biggest health system. It is tied to Wake Forest University School of Medicine. Also, it runs far more trials than anyone else nearby.

Visit Website →

American Health Research Network

Ballantyne, NC 28277 / 25 to 30+ active studies

Asthma, COPD, Chronic Cough, Type 2 Diabetes

Led by Dr. Selwyn Spangenthal. It is the biggest private lung research group in Charlotte. Specifically, it has 4 offices: Charlotte, Rock Hill, Lake Norman, and Charleston.

Visit Website →

Monroe Biomedical Research

Monroe, NC / 17+ active studies

Asthma, COPD, Hypertension, Obesity, Ulcerative Colitis

Notably, one of the 5 busiest research sites in the country. Its 6,300 sq ft space can host overnight stays. It is also an SCRS Global Impact Partner.

Visit Website →

Clinical Research of Gastonia

Gastonia, NC / 11+ active studies

Asthma, COPD, Chronic Cough, Idiopathic Hypersomnia

Led by Dr. Anup Banerjee. Its 3,500 sq ft space sits next to CaroMont Regional Medical Center. Also, it has been running for over 10 years.

Visit Website →

OnSite Clinical Solutions

Ballantyne, NC 28277 / 10+ active studies

Ulcerative Colitis, Crohn’s, COPD, Heart Failure, Atopic Dermatitis

It works with Charlotte doctors in heart, gut, skin, nerve, and joint care. Besides, it runs studies from Phase I through Phase IV.

Visit Website →

Flourish Research

Matthews, NC 28105 / 4+ active studies

Alzheimer’s Disease, Early Alzheimer’s, Alzheimer’s-Related Psychosis

Led by Dr. M. Reza Bolouri. He has 20+ years in Alzheimer’s research. The site is an Alzheimer’s Center of Excellence. In fact, its network has finished 5,500+ trials.

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New Hope Clinical Research

SouthPark, NC 28211 / 4+ active studies

Major Depression, Schizophrenia, Bipolar Disorder

Led by Dr. Kurian Abraham. It runs Phase I through IV studies and has 30 beds for overnight stays. Its last FDA audit came back clean. Also, it has finished 200+ brain studies.

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Queen City Clinical Research

SouthPark, NC 28211 / 2+ active studies

Chronic Pain, Chronic Migraine, Osteoarthritis

Led by Dr. Leonardo Kapural, a former Cleveland Clinic professor. Notably, he has written 200+ journal articles. On top of that, many call this the biggest pain research site in the country.

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Novant Health

Multiple Charlotte locations / Several active studies

Cardiovascular Disease, Obesity, Hypertension

One of the biggest health systems in the Carolinas. Its Heart and Vascular Institute runs heart studies and sees a lot of patients.

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DLVSC

4 Charlotte-area offices / 1+ active studies

Eczema, Psoriasis, Rosacea

Led by Dr. Gilly Munavalli. This award-winning skin practice owns 50+ FDA-approved lasers and devices. Also, it has its own research team.

Visit Website →

DelRicht Research

South Charlotte, NC 28210 / 1+ active studies

Bipolar Disorder, Bipolar Depression

Part of a national network of mental health research sites. Currently, it runs the azetukalner bipolar depression trial in Charlotte. Previously, it ran other mental health studies.

Visit Website →

TMS of the Carolinas

South Charlotte, NC 28210 / 1+ active studies

Major Depression, Treatment-Resistant Depression

A specialty site for brain stimulation care. It runs the fast TMS study that packs 6 weeks into 2.

Visit Website →

What to Expect in a Sickle Cell Study

New to studies? Here is how one works, step by step.

Prescreening

First comes a short phone call or online form. Also, it takes about 10 to 15 minutes. The staff check whether you might fit. Then they tell you what is open.

Screening Visit

Next the clinic runs the tests that decide it, which usually means blood work. Also, they go through the consent form line by line, and you can bring somebody with you.

Treatment Period

Then the study begins. So you take the study drug or the placebo on the agreed schedule. Meanwhile, your own doctor keeps managing everything else.

Follow-Up

Finally, there is a check in period at the end. Also, your own doctor gets the results, and you can stop at any point without affecting your care.

Sickle Cell Trial FAQs

Real answers about sickle cell disease studies near Charlotte NC.

Is there a sickle cell study near Charlotte?

Yes, four, and all of them run inside Charlotte itself. In fact, two are treatment studies and two record what happens without changing anything. Notably, the two treatment studies are opposites, because one needs a matched sibling and the other rules you out if you have one. So HLA typing is the first thing to sort out.

What is the sibling question about?

It is the single thing that routes you, and it catches families out. The gene editing study rules you out if you have a willing, healthy, fully matched brother or sister. The transplant study requires exactly that. So the first move is a simple blood test called HLA typing, because it decides everything downstream.

Is gene therapy just an injection?

No, and this is the most important thing to understand. It is closer to a transplant using your own cells, and it takes most of a year. Months of transfusions first, then hours hooked to a machine collecting stem cells, then months of lab work, then chemotherapy that wipes out your marrow and four to seven weeks in hospital.

Does gene therapy affect fertility?

Yes, that is a recognised consequence of the chemotherapy used to make room for the edited cells. It is why sperm banking or preserving eggs or ovarian tissue is discussed before anything starts. So raise it at the first appointment rather than waiting to be told. Meanwhile, the sibling transplant study deliberately uses a lighter preparation, partly for this reason.

What happened to the tablet I was taking?

If you mean voxelotor, it was withdrawn worldwide in September 2024 over safety concerns. So people who remember stopping it abruptly are remembering correctly. It also now appears in the rules that block you from joining newer studies. Meanwhile, hydroxyurea, glutamine and the anti sticking infusion all remain in use.

Is there a placebo in any of these?

No, in none of them. Both treatment studies give the real thing to every person enrolled, because neither randomises anybody. The other two give no treatment at all, since they only record what happens. So nobody on this page receives a dummy.

Can adults take part?

Some can, but the ages are tighter than people expect. The gene editing study takes 12 to 35, so it is not open ended. The transplant study takes 2 to 25, and both observational studies are for children and young people. So an adult over 35 has no open study here, and we would rather say that plainly.

Does it cost anything to be on the list?

No. There is no cost, and no insurance is needed. Tether is a nonprofit list, so we do not run studies and we are not paid to send you anywhere. Also, being on the list never enrolls anyone in anything. You would always call the clinic yourself.

What if I do not qualify for anything?

There is one study here with almost no rules at all. It takes sickle cell disease of any type, it creates no extra visits, and it simply records the results of tests you are already having. So if the others are closed to you, that is very likely the one you can join.

Where can I get sickle cell care near Charlotte NC?

Generally, Levine Children’s Hospital and Novant Health Hemby Children’s Hospital both run sickle cell care in Charlotte. Also, ask about the yearly brain scan for children, because it catches stroke risk early and is easy to miss. Your own doctor can refer you to a sickle cell specialist. Meanwhile, if you are moving from child to adult care, ask who is taking over before you turn 18.

Ready to Explore a Study?

Check your eligibility, or just reach out to our Charlotte team. No pressure. Also, no sales pitch.

See if I may fit one of these studies → Contact Us

← Back to All Clinical Trials

Sources

Study details and per-site recruiting status verified against ClinicalTrials.gov on August 15, 2026. A large blood disorder registry lists sickle cell among its conditions but has no open group for it, so it is deliberately not listed here. Study availability and criteria change; the research clinic confirms everything before you enroll.

See which sickle cell study you qualify for →