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Trackers› Clinical trials› Sickle Cell Disease
Clinical trial register · United States · industry-sponsored

Sickle Cell Disease Trials in the USA, 2026

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9123 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 05 Nov 2026, 03:16 UTC
9123
Studies in scope
industry-sponsored, all four open statuses
4965
Recruiting now
open to new participants
2185
Lead sponsors
across recruiting studies
Top states
Florida 15 Georgia 15 Alabama 13 California 13 Illinois 11
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Recruitment status
Phase
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Sickle Cell Disease trials in the USA

27 of 9123 studies
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Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
Agios Pharmaceuticals, Inc.
Phase 3
159
Indiana, Michigan, Mississippi, New York, Texas
2026-07-21
Recruiting
CSL Behring
Phase 2
63
Arkansas, California, Florida, Georgia, Maryland, Massachusetts, Minnesota, Mississippi
2026-02-02
Recruiting
Sanofi
Phase 3
100
Georgia, Nebraska, New Jersey, Texas
2026-01-20
Recruiting
Disc Medicine, Inc
Phase 1
24
Alabama, California, Georgia, Illinois, Indiana, New York, North Carolina, Ohio
2026-01-06
Recruiting
A Long-term Follow-up Study in Patients Who Received BEAM-101
NCT07373639Hemoglobinopathy, Sickle Cell Disease
Beam Therapeutics Inc.
Observational
50
Alabama, Arizona, Florida, Georgia, Massachusetts, Michigan, Minnesota, Missouri
2025-12-15
Recruiting
Sanofi
Phase 3
192
Alabama, Arizona, California, District of Columbia, Florida, Illinois, Indiana, Louisiana
2025-08-12
Not yet open
Asklepion Pharmaceuticals, LLC
Phase 3
120
District of Columbia, Florida, Georgia, Maryland, Michigan, North Carolina
2025-07-25
Active, closed
Agios Pharmaceuticals, Inc.
Phase 2
59
Connecticut, District of Columbia, Georgia, Michigan, New York, Pennsylvania, South Carolina, Texas
2025-05-01
Recruiting
Novo Nordisk A/S
Phase 3
510
Alabama, Arizona, Arkansas, California, Colorado, Delaware, District of Columbia, Florida
2025-02-17
Recruiting
Novo Nordisk A/S
Phase 3
480
Alabama, Arizona, California, Connecticut, District of Columbia, Florida, Georgia, Illinois
2025-01-10
Recruiting
Novartis Pharmaceuticals
Phase 3
354
Alabama, California, District of Columbia, Florida, Georgia, Illinois, Kentucky, Maryland
2024-10-24
Recruiting
Novartis Pharmaceuticals
Phase 1/2
161
Alabama, Florida, Massachusetts, New York, North Carolina, Pennsylvania, Rhode Island, Texas
2024-08-15
By invitation
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
NCT06363760Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Editas Medicine, Inc.
Observational
54
California, Colorado, Connecticut, Florida, Georgia, Minnesota, New York, North Carolina
2024-06-17
Active, closed
Novartis Pharmaceuticals
Phase 1
4
Illinois, New York, Tennessee
2024-04-16
Recruiting
Pfizer
Phase 2/3
389
Alabama, Connecticut, Florida, Georgia, Illinois, Louisiana, Michigan, Mississippi
2022-09-22
Active, closed
Beam Therapeutics Inc.
Phase 1/2
15
Alabama, Arizona, Florida, Georgia, Massachusetts, Michigan, Minnesota, Missouri
2022-08-30
Active, closed
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05329649Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
Vertex Pharmaceuticals Incorporated
Phase 3
13
North Carolina, Pennsylvania, Tennessee
2022-05-02
Active, closed
Agios Pharmaceuticals, Inc.
Phase 2/3
286
California, Connecticut, District of Columbia, Florida, Illinois, Indiana, Louisiana, Maryland
2022-02-11
Recruiting
Kamau Therapeutics
Phase 1/2
15
California, Missouri, New York, Ohio
2021-11-15
Recruiting
Novartis Pharmaceuticals
Phase 4
130
Alabama, District of Columbia, Georgia, North Carolina, Pennsylvania, Texas
2021-06-10
Active, closed
Editas Medicine, Inc.
Phase 1/2
45
California, Colorado, Connecticut, Florida, Georgia, Illinois, Mississippi, New Jersey
2021-05-04
Active, closed
Forma Therapeutics, Inc.
Phase 3
450
Alabama, Arizona, Arkansas, California, Connecticut, District of Columbia, Florida, Georgia
2021-01-29
By invitation
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
Vertex Pharmaceuticals Incorporated
Phase 3
160
California, Illinois, New York, North Carolina, Pennsylvania, Tennessee, Texas
2021-01-20
By invitation
Genetix Biotherapeutics Inc.
Observational
85
Alabama, California, Georgia, Illinois, Maryland, Minnesota, New Jersey, New York
2020-10-21
Active, closed
Genetix Biotherapeutics Inc.
Phase 3
35
Alabama, District of Columbia, Massachusetts, Minnesota, New Jersey, New York, North Carolina, Texas
2020-02-14
Recruiting
EpiDestiny, Inc.
Phase 1
20
Illinois
2020-01-24
Active, closed
Imara, Inc.
Phase 2
30
Connecticut, Florida, Texas
2019-05-22
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Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 5 November 2026. https://priyalifescience.com/clinical-trials-usa/condition/sickle-cell-disease

Query this register directly
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About this register

This register is built from ClinicalTrials.gov, the trial registry run by the U.S. National Library of Medicine. It includes every industry-sponsored study on the registry that lists at least one investigative site in the US and is recruiting, not yet recruiting, active but no longer recruiting, or enrolling by invitation: 9,123 studies in total at the last sync on 05 Nov 2026, 03:16 UTC. Academic and other non-industry sponsors are out of scope by design. The full list can be downloaded as CSV or queried as JSON through the register API.

This page shows the 27 of those studies listing the condition Sickle Cell Disease.

How many Sickle Cell Disease trials are recruiting in the US?

14 of the 27 studies on this page are recruiting new participants as of the 05 Nov 2026, 03:16 UTC sync. A further 1 have not yet opened recruitment, 9 are active but closed to new participants, and 3 are enrolling by invitation only. Eligibility for any study is decided by its study team.

Summary by phase

Studies by phase (Sickle Cell Disease), 27 studies
PhaseStudiesRecruitingShare
Phase 311640.7%
Phase 1/24214.8%
Observational3111.1%
Phase 13211.1%
Phase 23111.1%
Phase 2/3217.4%
Phase 4113.7%

Most active sponsors

Lead sponsors with the most studies (Sickle Cell Disease)
Lead sponsorStudiesRecruiting
Novartis Pharmaceuticals43
Agios Pharmaceuticals, Inc.31
Beam Therapeutics Inc.21
Editas Medicine, Inc.20
Genetix Biotherapeutics Inc.20
Novo Nordisk A/S22
Sanofi22
Vertex Pharmaceuticals Incorporated20
Asklepion Pharmaceuticals, LLC10
CSL Behring11

Top states

Top states (Sickle Cell Disease), by number of studies with a site there
StateStudies
Florida15
Georgia15
Alabama13
California13
Illinois11
District of Columbia10
New York10
Connecticut8
North Carolina8
Texas8
Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.