Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene therapy breakthrough? new treatment aims to fix sickle cell at the source

NCT ID NCT04819841

Disease control Sponsor: Kamau Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The therapy modifies a patient's own blood stem cells to produce normal hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the treatment after a conditioning regimen. The main goals are to check safety and see if the therapy can reduce or eliminate sickle cell complications.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nula-cel (a gene therapy that modifies a patient's own blood stem cells to produce normal hemoglobin)
What this could lead to
If it works, this could provide a one-time treatment that controls severe sickle cell disease by enabling the body to make healthy hemoglobin instead of sickle hemoglobin.
What could go wrong
This is a very early, first-in-human trial with only 15 participants. The conditioning regimen has serious risks, and the gene therapy may not work as expected or could cause side effects. Long-term safety and effectiveness are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2021

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 50 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * ≥12 to ≤ 40 years * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures: * recurrent severe VOC (≥ 4 episodes in the preceding 2 years) * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year) * Lansky/Karnofsky performance status of ≥ 80 Exclusion Criteria: * Available 10/10 HLA-matched sibling donor * Prior HSCT or gene therapy * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder * Clinically significant and active bacterial, viral, fungal or parasitic infection * Pregnancy or breastfeeding in a postpartum female * Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Sickle cell disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    6 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Lucile Packard Children's Hospital

    RECRUITING

    Palo Alto, California, 94304, United States

  • Memorial Sloan Kettering

    RECRUITING

    New York, New York, 10065, United States

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.