Blood

Sickle Cell Disease

Also known as SCD, HbSS, sickle cell anemia, hemoglobin SC disease

ORPHA:275752 ↗Gene HBBPrevalence 1-5 per 10,000 (Orphanet)Onset All agesGenetic (autosomal recessive)

Search all Sickle Cell Disease trialsAlert me when a trial opens

studies recruiting now

checking live

studies registered in total

checking live

countries with a recruiting site

checking live

recruiting study posted to date

checking live

Recruiting trials

Fetching live from ClinicalTrials.gov. This condition is not yet in our weekly snapshot; live results only.

Keep watching

Get an email when a new Sickle Cell Disease study opens.

One email a day at most. Unsubscribe with one click.

Used only for these alerts. Privacy.

Support

Patient organisations

Sickle Cell Disease Association of AmericaPatient association
Visit website ↗

Registry: Registry and Surveillance System for Hemoglobinopathies (RuSH) · Join ↗. Registries connect patients to researchers and often hear about trials first.

NORD ↗GARD ↗Orphanet ORPHA:275752 ↗

About Sickle Cell Disease

Sickle cell disease is caused by a mutation in the HBB gene that causes hemoglobin to polymerize under low-oxygen conditions, deforming red blood cells into a sickle shape. These cells block blood flow, causing painful vaso-occlusive crises, organ damage, stroke, and acute chest syndrome. Two gene therapies (exagamglogene autotemcel / Casgevy, a CRISPR-based therapy, and lovotibeglogene autotemcel / Lyfgenia) received FDA approval in 2023.

Common clinical features

Chronic hemolytic anemiaPainHemolytic anemiaRecurrent infectionsAvascular necrosisChest painLeukocytosisOsteomyelitis

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

7 approved treatments and 121 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Crizanlizumab (Adakveo)Approved: Lovotibeglogene Autotemcel (Lyfgenia)Approved: Exagamglogene Autotemcel (Casgevy)Approved: Hydroxyurea (Droxia)Approved: Voxelotor (Oxbryta)Approved: Glutamine (Endari)Approved: Deferiprone (Deferiprone lipomed)

Phase 3Rilzabrutinib

Phase 3Deferasirox (Deferasirox)

Phase 3Etavopivat

Phase 3Melphalan (Alkeran)

Phase 3Hydromorphone

Phase 3Senicapoc

Phase 3Dexamethasone (Aeroseb-dex)

Phase 3Alemtuzumab (Campath mabcampath)

+ 113 more in development

Before you apply

Things trial teams commonly ask about for Sickle Cell Disease. Not eligibility rules; those are set by each study.

Hemoglobin genotype (HbSS, HbSC, HbS-beta-thal) determines which trials you qualify for
Vaso-occlusive crisis frequency and prior hospitalizations are key baseline eligibility criteria
Gene therapy trials require stopping hydroxyurea and have demanding mobilization/apheresis protocols

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).