Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruitment pending
Participants planned
63
Countries
4
Sites
15
Sickle Cell Disease
The primary objective of this study is to assess the safety of anumigilimab in adults with SCD
Key facts
- Sponsor
- CSL Behring LLC
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Decision date (initial)
- 2026-04-28
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- CSL Behring LLC
External identifiers
- EU CT number
- 2025-521154-42-00
- ClinicalTrials.gov
- NCT07224360
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
The primary objective of this study is to assess the safety of anumigilimab in adults with SCD
Secondary objectives 2
- To assess the PK of anumigilimab
- To assess the effect of anumigilimab on the frequency of VOCs
Conditions and MedDRA coding
Sickle Cell Disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | SOC | 10010331 | Congenital familial and genetic disorders | 21 |
Study design 2 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Dose escalation period This is a double blind, placebo controlled study.
|
Randomised Controlled | Double | [{"id":188073,"code":1,"name":"Subject"},{"id":188074,"code":4,"name":"Analyst"},{"id":188071,"code":3,"name":"Monitor"},{"id":188075,"code":2,"name":"Investigator"},{"id":188072,"code":5,"name":"Carer"}] | |
| 2 | Maintenance period This is a double blind, placebo controlled study.
|
Randomised Controlled | Double | [{"id":188077,"code":3,"name":"Monitor"},{"id":188081,"code":2,"name":"Investigator"},{"id":188080,"code":5,"name":"Carer"},{"id":188078,"code":1,"name":"Subject"},{"id":188079,"code":4,"name":"Analyst"}] | Anumigilimab: Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD) Placebo: Participants will receive matching volume of placebo |
Regulatory references
- Scientific advice from competent authorities
- Food And Drug Administration, European Medicines Agency
- Plan to share IPD
- Yes
- IPD plan description
- 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 [email protected].
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- 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).
Exclusion criteria 5
- 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.
- History of myeloproliferative disorder or malignancy (other than skin cancer) within 5 years before Screening.
- History of stem cell transplant or gene therapy.
- Hemoglobin < 6.5 g/dL at Screening or Baseline (Week 1 Day 1).
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Number and percentage of subjects with Treatment Emergent Adverse Events (TEAEs), overall and by severity, seriousness and relationship to Investigational Product (IP)
- Number and percentage of subjects with Adverse Events of Special Interest (AESIs)
- Clinically relevant changes from baseline in laboratory assessments and vital signs
Secondary endpoints 2
- From the first dose of anumigilimab through Week 65: • Serum anumigilimab
- During the 52-week Maintenance Period: • Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11100686 · Product
- Active substance
- Anumigilimab
- Other product name
- Recombinant Anti-G-CSF Receptor Monoclonal Antibody
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 1480 mg milligram(s)
- Max treatment duration
- 64 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- CSL INNOVATION GMBH
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
CSL Behring LLC
- Sponsor organisation
- CSL Behring LLC
- Address
- 1020 1st Avenue
- City
- King Of Prussia
- Postcode
- 19406-1310
- Country
- United States
Scientific contact point
- Organisation
- CSL Behring LLC
- Contact name
- Study Director
Public contact point
- Organisation
- CSL Behring LLC
- Contact name
- Trial Registration Coordinator
Third parties 10
| Organisation | City, country | Duties |
|---|---|---|
| Suvoda LLC ORG-100043523
|
Conshohocken, United States | Interactive response technologies (IRT) |
| Center For Information And Study On Clinical Research Participation Inc. ORG-100044581
|
Boston, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Other |
| Q2q Communications Limited ORG-100041455
|
Richmond, United Kingdom | Other |
| Fisher Clinical Services GmbH ORG-100012942
|
Allschwil, Switzerland | Other |
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | Other |
| PPD Development LP ORG-100011560
|
Wilmington, United States | On site monitoring, Other, Code 5, Data management |
| Azenta Singapore Pte Ltd ORG-100049467
|
Singapore, Singapore | Other |
| CSL Innovation Pty Limited ORG-100051289
|
Melbourne, Australia | Other |
Locations
4 EU/EEA countries · 15 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Authorised, recruitment pending | 1 | 2 |
| France | Authorised, recruitment pending | 3 | 4 |
| Italy | Authorised, recruitment pending | 7 | 8 |
| Netherlands | Authorised, recruitment pending | 1 | 1 |
| Rest of world
Saudi Arabia, United States, Turkey, United Kingdom, Canada
|
— | 51 | — |
Investigational sites
Universitair Ziekenhuis Antwerpen
Hematology, Drie Eikenstraat 655, 2650, Edegem
CHU Saint Pierre
Hematology, Hoogstraat 322, 1000, Brussels
Centre Hospitalier Regional De Marseille
Medecine Interne, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Universitaire De Nantes
Médecine Interne, 1 Place Alexis Ricordeau, 44000, Nantes
Centre Hospitalier Universitaire Rouen
Medicine intensive et reanimation, 1 Rue De Germont, Bp 96031, Rouen Cedex
Oncopole Claudius Regaud
Médecine Interne, 1 Avenue Irene Joliot Curie, 31100, Toulouse
IRCCS Istituto Giannina Gaslini
UOC Ematologia, Via Gerolamo Gaslini 5, 16147, Genoa
Centro Ricerche Cliniche Di Verona S.r.l.
Policlinico GB Rossi di Borgo Roma, Piazzale Ludovico Antonio Scuro 10, 37134, Verona
Ospedale Pediatrico Bambino Gesu
Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietic, Piazza Di Sant'onofrio 4, 00165, Rome
University Hospital Of Ferrara
UOC Talassemie ed Emoglobinopatie, Via Aldo Moro 8, 44124, Ferrara
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
U.O.S.D. Malattie Rare del Globulo Rosso - Dipartimento Onco-ematologico e Pneumo-toracico, Via Antonio Cardarelli 9, 80131, Naples
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
U.O.C. Ematologia per le Malattie Rare del Sangue e degli, Via Trabucco 180, 90146, Palermo
Azienda Ospedaliero Universitaria Di Modena
S.C. Medicina Interna, Largo Del Pozzo 71, 41124, Modena
Fondazione IRCCS San Gerardo Dei Tintori
SSD Malattie Rare, Via Giovanbattista Pergolesi 33, 20900, Monza
Amsterdam UMC Stichting
Hematology, De Boelelaan 1117, 1081 HV, Amsterdam
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 74 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_CSLB_CSL324_2002_Protocol_2025-521154-42-00_Public | 1.0 |
| Protocol (for publication) | D2_CSLB_CSL324_2002_Global Protocol Clarification Letter to Amendment 1_Public | n/a |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_DE_BE_Public | 1.0 |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_FR_BE_Public | 1.0 |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_FRE_FR_Public | 1.0 |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_IT_ITA_Public | 1.0 |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_NL_BE_Public | 1.0 |
| Protocol (for publication) | D4_CSL Behring_CSL324_2002_All questionnaires_NL_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K1_CSL324_2002_Recruitment-and-Informed-Consent-Procedure_ITA_ENG_Public | 1.0 |
| Recruitment arrangements (for publication) | K1_CSL324_2002_Recruitment-Arrangements_BEL | 1.0 |
| Recruitment arrangements (for publication) | K1_CSL324_2002_Recruitment-arrangements_NLD_ENG_Public | n/a |
| Recruitment arrangements (for publication) | K1_CSL324-2002_Recruitment-Arrangements_FRA_fra_Public | n/a |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Brochure_ITA_ITA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Brochure_NLD_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Brochure-Master_BEL_ENG_Public | 2.1 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Brochure-Master_BEL_FRA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Brochure-Master_BEL_NLD_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Consent-Navigator_FRA_fra_eng_Public | 1.2 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Consent-Navigator_ITA_ITA_Public | 1.1 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Consent-Navigator_NLD_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_ICF-Infographic_ITA_ITA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_ICF-Infographic_NLD_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_ICF-Infographic-Master_BEL_ENG_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_ICF-Infographic-Master_BEL_FRA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_ICF-Infographic-Master_BEL_NLD_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Patient-Video-Storyboard-and-Script_BEL_FRA_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Patient-Video-Storyboard-and-Script_BEL_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Patient-Video-Storyboard-and-Script_ITA_ITA_Public | 1.2 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Patient-Video-Storyboard-Script_FRA_fra_Public | 1.1 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Patient-Video-Storyboard-Script_NLD_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Poster_ITA_ITA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Poster_NLD_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Poster-Master_BEL_ENG_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Poster-Master_BEL_FRA_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_Poster-Master_BEL_NLD_Public | 2.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_StudyVideo-Patients-Storyboard_BEL_ENG_Public | 1 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Consent-Navigator_BEL_ENG_FRA_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Consent-Navigator_BEL_ENG_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Patient-Portal_BEL_ENG_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Patient-Portal_BEL_FRA_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Patient-Portal_BEL_NLD_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Patient-Recruitment-Website_BEL_fra_Public | 1.2 |
| Recruitment arrangements (for publication) | K2_CSL324_2002_TRANSCENDING-Patient-Recruitment-Website_BEL_nld_Public | 1.2 |
| Recruitment arrangements (for publication) | K2_CSL324-2002_ICF_Infographic_FRA_fra_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324-2002_Patient-Recruitment-Website_FRA_fra_Public | 1.2 |
| Recruitment arrangements (for publication) | K2_CSL324-2002_Poster_FRA_fra_Public | 1.0 |
| Recruitment arrangements (for publication) | K2_CSL324-2002_Recruitement_Brochure_FRA_fra_Public | 2.1 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Adult-ICF_ITA_ITA_Public | 1 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_ICF_Use-of-personal-data_ITA_ITA_Public | 1 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Main-ICF_BEL_ENG_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Main-ICF_BEL_FRA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Main-ICF_BEL_NLD_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_PP-ICF_BEL_ENG_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_PP-ICF_BEL_FRA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_PP-ICF_BEL_NDL_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Pregnant-Partner-ICF_ITA_ITA_Public | 1 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Scout-Clinical-Pre-ICF-Telephone-Data-Consent_BEL_ENG_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Scout-Clinical-Pre-ICF-Telephone-Data-Consent_BEL_FRA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Scout-Clinical-Pre-ICF-Telephone-Data-Consent_BEL_NLD_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Scout-Clinical-Pre-ICF-Telephone-Data-Consent_ITA_ITA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_SIS-and-ICF-Adults_NLD_NLD_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_SIS-and-ICF-Pregnancy_NLD_NLD_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324_2002_Sponsor-Statement-Main-ICF_BEL_ENG_Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_CSL324-2002_Main_ICF_FRA_fra_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_CSL324-2002_Optional_Future_Research_ICF_FRA_fra_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_CSL324-2002_Pregnancy_ICF_FRA_FR_Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_CSL324-2002_Patient_Card_FRA_fra_Public | 1.0.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_ENG_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_FRA_ Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_FRA_BEL_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_GER_BEL_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_ITA_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_NLD_BEL_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_CSLB_CSL324_2002_Protocol synopsis_2025-521154-42-00_NLD_Public | 1.0 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-12-19 | Belgium | Acceptable 2026-04-24
|
2026-04-28 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-05-27 | Belgium | Acceptable 2026-04-24
|
2026-05-27 |