Sickle Cell DiseasePhase 2NCT07224360
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
A Phase 2 study testing Anumigilimab in people with Sickle Cell Disease, run by CSL Behring at 38 sites in 7 countries.
Trial summary
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Anumigilimab
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- 38 sitesin 7 countries
- Study dates
- 2 Feb 2026to 15 Aug 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults aged greater than or equal to (>=) 18 years on the day of signing the informed consent form.
- Confirmed diagnosis of SCD of any genotype.
- Experienced 1 to 12 VOCs requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug within the 12 months before Screening.
- HU Regimen:
- a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.
- or
- b. HU was discontinued or refused (eg, due to concern of side effects or lack of effect).
Who cannot join
- Absolute neutrophil count less than (<) 2.5 ×10^9 cells/Litre at Screening or Baseline (Week 1 Day 1).
- If on SCD preventive medication, dose is not stable in the 30 days before Screening.
Trial locations and contacts
38 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
38 sites in 7 countries
United States14 sites, 14 recruiting
- Recruiting
Arthur M. Blank Hospital- Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
- Recruiting
Center for Cancer and Blood Disorders
Bethesda, Maryland, 20817
- Recruiting
Boston Medical Center
Boston, Massachusetts, 02118
- Recruiting
Ohio State University
Columbus, Ohio, 43210
- Recruiting
Southern Specialty Research
Flowood, Mississippi, 39232
- Recruiting
The Foundation for Sickle Cell Disease
Hollywood, Florida, 33023-6703
- Recruiting
UT Physicians Comprehensive Adult Sickle Cell Center
Houston, Texas, 77030
- Recruiting
NYC Health + Hospitals/Queens
Jamaica, New York, 11432
- Recruiting
UAMS Medical Center
Little Rock, Arkansas, 72202
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
- Recruiting
Columbia University Medical Center
New York, New York, 10032
- Recruiting
University of California Irvine
Orange, California, 92868
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23298
- Recruiting
Jacobi Medical Center
The Bronx, New York, 10461
United Kingdom7 sites, 7 recruiting
- Recruiting
Royal Liverpool University Hospital
Liverpool, L7 8YE
- Recruiting
The Royal London Hospital
London, E1 2ES
- Recruiting
Central Middlesex Hospital
London, NW10 7NS
- Recruiting
Guy's Hospital
London, SE17EH
- Recruiting
St George's University Hopsitals NHS Foundation Trust
London, SW17 0QT
- Recruiting
Hammersmith Hospital Imperial College Healthcare NHS Trust
London, W120HS
- Recruiting
Manchester Royal Infirmary
Manchester, England, M13 9WL
Italy6 sites, 6 recruiting
- Recruiting
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Cona, Ferrara, 44124
- Recruiting
IRCCS Istituto Giannina Gaslini
Genoa, 16132
- Recruiting
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900
- Recruiting
AORN Antonio Cardarelli
Naples, 80131
- Recruiting
IRCCS Bambino Gesù Children's Hospital
Rome, 00165
- Recruiting
AOUI Verona and University of Verona
Verona, 37134
France4 sites, 4 recruiting
- Recruiting
AP-HM Hôpital de La Timone
Marseille, Bouches-du-Rhône, 13005
- Recruiting
CHU de Nantes, Hotel-Dieu
Nantes, 44093
- Recruiting
CHU de Rouen-Hôpital Charles-Nicolle
Rouen, 76000
- Recruiting
Institut Universitaire du Cancer de Toulouse
Toulou, 31100
Turkey (Türkiye)4 sites, 4 recruiting
- Recruiting
Acibadem Adana Hastanesi
Adana, 1130
- Recruiting
Hacettepe University Medical Faculty
Ankara, 6230
- Recruiting
VM Medical Park Mersin Hospital
Mezitli, Mersin, 33000
- Recruiting
Mersin University Medical Faculty
Yenişehir, Mersin, 33110
Belgium2 sites, 2 recruiting
- Recruiting
CHU Saint-Pierre
Brussels, 1000
- Recruiting
UZ Antwerpen
Edegem, Antwerp, 2650
Netherlands1 site, 1 recruiting
- Recruiting
Amsterdam UMC - VUmc
Amsterdam, North Holland, 1105AZ
About this study
Official title: Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD.
The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.
- Conditions
- Sickle Cell Disease
- Being tested
- Anumigilimab, Placebo
- Keywords
- Anemia, Genetic Disorder, Vaso-occlusive Crises
- Medical subject headings
- Anemia, Sickle Cell, Anemia, Genetic Diseases, Inborn, Vaso-Occlusive Crises
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AnumigilimabBiological
Participants will receive anumigilimab, escalated to a maximum dose guided by absolute neutrophil count (ANC) and safety concerns.
PlaceboDrug
Volume matched saline will be administered SC.
| Study group | Role | What they receive |
|---|---|---|
| Anumigilimab | Experimental | Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD) Receives: Biological: Anumigilimab |
| Placebo | Placebo comparator | Participants will receive matching volume of placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
Measured: From baseline up to Week 75
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP
Measured: From baseline up to Week 75
Number of participants with Adverse Events of Special Interest (AESI)
The following AEs are to be reported as AESI: neutropenia (Common Terminology Criteria for Adverse Events [CTCAE] Grade 3 or 4), severe infection and neutropenic fever.
Measured: From baseline up to Week 75
Percentage of participants with AESI
The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Measured: From baseline up to Week 75
Number of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Measured: From baseline up to Week 75
Percentage of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Measured: From baseline up to Week 75
Secondary outcomes (2)
Serum concentration of Anumigilimab
Measured: From the first dose of anumigilimab to Week 65
Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)
VOC (medical facility) is defined as VOC that results in a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug.
Measured: During the maintenance period from Week 13 to Week 65
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2025
- Study start2 Feb 2026
- Primary completion15 Aug 2028 (estimated)
- Study completion15 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a double blind, placebo controlled study.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 63 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07224360
- Sponsor's protocol ID
- CSL324_2002
- Other IDs
- 2025-521154-42-00, 2025
- Status last verified
- Oct 2026
- First submitted
- 31 Oct 2025
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT07224360
- Acronym / sponsor ID
- CSL324_2002
- Conditions
- Sickle Cell Disease
- Keywords
- Anemia, Genetic Disorder, Vaso-occlusive Crises
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 63 participants (estimated)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 2 Feb 2026 (actual)
- Primary completion
- 15 Aug 2028 (estimated)
- Study completion
- 15 Aug 2028 (estimated)
- First posted
- 4 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD.
The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a double blind, placebo controlled study.
| Arm | Type | Receives |
|---|---|---|
| Anumigilimab | Experimental | Biological: AnumigilimabSafety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD) |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive matching volume of placebo. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Adults aged greater than or equal to (>=) 18 years on the day of signing the informed consent form.
- Confirmed diagnosis of SCD of any genotype.
- Experienced 1 to 12 VOCs requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug within the 12 months before Screening.
- HU Regimen:
- a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.
Key exclusion criteria (2 listed)
- Absolute neutrophil count less than (<) 2.5 ×10^9 cells/Litre at Screening or Baseline (Week 1 Day 1).
- If on SCD preventive medication, dose is not stable in the 30 days before Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AnumigilimabParticipants will receive anumigilimab, escalated to a maximum dose guided by absolute neutrophil count (ANC) and safety concerns. | Biological | Anumigilimab |
| PlaceboVolume matched saline will be administered SC. | Drug | Placebo |
Primary outcome
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
Time frame: From baseline up to Week 75
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP
Time frame: From baseline up to Week 75
Number of participants with Adverse Events of Special Interest (AESI)
Time frame: From baseline up to Week 75
The following AEs are to be reported as AESI: neutropenia (Common Terminology Criteria for Adverse Events [CTCAE] Grade 3 or 4), severe infection and neutropenic fever.
Percentage of participants with AESI
Time frame: From baseline up to Week 75
The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Number of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Time frame: From baseline up to Week 75
Percentage of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Time frame: From baseline up to Week 75
2 secondary outcomes
Serum concentration of Anumigilimab
Time frame: From the first dose of anumigilimab to Week 65
Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)
Time frame: During the maintenance period from Week 13 to Week 65
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2026.
- Individual participant dataSharing planned: Yes. CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
CSL Behring
38 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Anumigilimab in people with Sickle Cell Disease, run by CSL Behring at 38 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, United Kingdom, Italy, France, Turkey (Türkiye) and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Registration Coordinator (+1 610-878-4697, clinicaltrials@cslbehring.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP) (time frame: From baseline up to Week 75). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.