Clinical trial • Phase II • Haematology
ANUMIGILIMAB for Sickle cell disease
Phase II trial of ANUMIGILIMAB for Sickle cell disease.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Sickle cell disease
- Trial Stage
- Phase II
- Drug Modality
- Monoclonal antibody
Key dates
- Initial CTIS Submission Date
- 19-12-2025
- First CTIS Authorization Date
- 28-04-2026
Trial design
Randomised, placebo (saline 0.9%), matching volume; dose and schedule not specified in the record.-controlled, adaptive Phase II trial in Netherlands, France, Belgium and others.
- Randomised
- Yes
- Comparator
- Placebo (Saline 0.9%), matching volume; dose and schedule not specified in the record.
- Adaptive
- True, Dose escalation period present; detailed dose escalation rules, interim analysis plans or stopping rules not provided in the CTIS record.
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 51
- Trial Duration For Participant
- 455
Eligibility
Recruits 51 Vulnerable population selected (isVulnerablePopulationSelected=true) in the record; no further details on consent/assent handling for vulnerable groups are provided in the CTIS record..
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected=true) in the record; no further details on consent/assent handling for vulnerable groups are provided in the CTIS record.
Inclusion criteria
- {"criterion_text":"- Adults aged greater than or equal to (>=) 18 years on the day of signing the informed consent form."}
- {"criterion_text":"- Confirmed diagnosis of SCD of any genotype."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- 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
- {"criterion_text":"- Absolute neutrophil count less than (<) 2.5 ×10^9 cells/Litre at Screening or Baseline (Week 1 Day 1)."}
- {"criterion_text":"- If on SCD preventive medication, dose is not stable in the 30 days before Screening."}
- {"criterion_text":"- History of myeloproliferative disorder or malignancy (other than skin cancer) within 5 years before Screening."}
- {"criterion_text":"- History of stem cell transplant or gene therapy."}
- {"criterion_text":"- Hemoglobin < 6.5 g/dL at Screening or Baseline (Week 1 Day 1)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number and percentage of subjects with Treatment Emergent Adverse Events (TEAEs), overall and by severity, seriousness and relationship to Investigational Product (IP)","definition_or_measurement_approach":"Count and percentage of subjects experiencing TEAEs, reported overall and categorized by severity, seriousness and relationship to the IP."}
- {"endpoint_text":"- Number and percentage of subjects with Adverse Events of Special Interest (AESIs)","definition_or_measurement_approach":"Count and percentage of subjects with AESIs."}
- {"endpoint_text":"- Clinically relevant changes from baseline in laboratory assessments and vital signs","definition_or_measurement_approach":"Assessment of changes from baseline in laboratory parameters and vital signs considered clinically relevant."}
Secondary endpoints
- {"endpoint_text":"- From the first dose of anumigilimab through Week 65: • Serum anumigilimab","definition_or_measurement_approach":"Measurement of serum concentrations of anumigilimab (PK) from first dose through Week 65."}
- {"endpoint_text":"- During the 52-week Maintenance Period: • Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)","definition_or_measurement_approach":"Calculation of annualized rate of VOCs requiring medical facility visit during the 52-week maintenance period."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Center For Information And Study On Clinical Research Participation Inc.
- Digital Remote Recruitment
- True, digital methods include patient recruitment websites, patient portal, patient recruitment website country variants, patient videos and online consent navigator tools (documents indicate digital/online recruitment materials for Belgium, France, Netherlands and Italy).
- Planned Sample Size
- 51
- Recruitment Window Months
- 24
- Consent Approach
- Adults (>=18 years) provide informed consent. Subject information and informed consent forms (Main ICF, Optional Future Research ICF, Pregnancy ICF) and consent-support materials (ICF infographic, Consent-Navigator) are provided. ICF and recruitment materials are available in multiple languages indicated by document titles (Dutch, French, English, Italian). No assent process described.
Methods
- Patient recruitment websites (country-specific patient recruitment website documents present for France, Belgium, Netherlands).
- Patient portal (Belgium patient-portal documents present).
- Printed materials: brochures and posters (country-specific brochures and posters for NLD, FRA, ITA, BEL).
- Multimedia: patient video/storyboard/script (documents present for NLD, FRA, ITA, BEL).
- Consent support tools: Consent-Navigator and ICF infographics (documents present for multiple countries).
- Pre-screening/scout telephone consent forms (Belgium and Italy scout clinical pre-ICF telephone data consent documents present).
Geography
- Total Number Of Sites
- 15
- Total Number Of Participants
- 12
Netherlands
- Earliest CTIS Part Ii Submission Date
- 31-03-2026
- Latest Decision Or Authorization Date
- 29-04-2026
- Processing Time Days
- 29
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Amsterdam UMC Stichting
- Department Name
- Hematology
- Contact Person Name
- Bart Biemond
- Contact Person Email
- hematology@amsterdamumc.nl
France
- Earliest CTIS Part Ii Submission Date
- 18-03-2026
- Latest Decision Or Authorization Date
- 28-04-2026
- Processing Time Days
- 41
- Number Of Sites
- 4
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Médecine Interne
- Contact Person Name
- Alexandra ESPITIA
- Contact Person Email
- alexandra.espitiathibault@chu-nantes.fr
- Site Name
- Centre Hospitalier Universitaire Rouen
- Department Name
- Medicine intensive et reanimation
- Contact Person Name
- Maximilien GRALL
- Contact Person Email
- maximillien.grall@chu-rouen.fr
- Site Name
- Oncopole Claudius Regaud
- Department Name
- Médecine Interne
- Contact Person Name
- Pierre COUGOUL
- Contact Person Email
- cougoul.pierre@iuct-oncopole.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Medecine Interne
- Contact Person Name
- Estelle JEAN-MIGNARD
- Contact Person Email
- estelle.jean@ap-hm.fr
Belgium
- Earliest CTIS Part Ii Submission Date
- 24-03-2026
- Latest Decision Or Authorization Date
- 28-04-2026
- Processing Time Days
- 35
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- CHU Saint Pierre
- Department Name
- Hematology
- Contact Person Name
- Jade Ku
- Contact Person Email
- jade.ku@stpierre-bru.be
- Site Name
- Universitair Ziekenhuis Antwerpen
- Department Name
- Hematology
- Contact Person Name
- Ann Van de Velde
- Contact Person Email
- ann.vandevelde@uza.be
Italy
- Earliest CTIS Part Ii Submission Date
- 27-02-2026
- Latest Decision Or Authorization Date
- 29-04-2026
- Processing Time Days
- 61
- Number Of Sites
- 8
- Number Of Participants
- 7
Sites
- Site Name
- University Hospital Of Ferrara
- Department Name
- UOC Talassemie ed Emoglobinopatie
- Contact Person Name
- Filomena Longo
- Contact Person Email
- filomena.longo@ospfe.it
- Site Name
- Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
- Department Name
- U.O.C. Ematologia per le Malattie Rare del Sangue e degli
- Contact Person Name
- Rosario Di Maggio
- Contact Person Email
- rdm83@hotmail.it
- Site Name
- Azienda Ospedaliero Universitaria Di Modena
- Department Name
- S.C. Medicina Interna
- Contact Person Name
- Francesca Ferrara
- Contact Person Email
- ferrara.francesca@aou.mo.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- UOC Ematologia
- Contact Person Name
- Erika Massaccesi
- Contact Person Email
- erikamassaccesi@gaslini.org
- Site Name
- Centro Ricerche Cliniche Di Verona S.r.l.
- Department Name
- Policlinico GB Rossi di Borgo Roma
- Contact Person Name
- Lucia De Franceschi
- Contact Person Email
- lucia.defranceschi@univr.it
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori
- Department Name
- SSD Malattie Rare
- Contact Person Name
- Raffaella Mariani
- Contact Person Email
- raffaella.mariani@irccs-sangerardo.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietic
- Contact Person Name
- Franco Locatelli
- Contact Person Email
- franco.locatelli@opbg.net
- Site Name
- Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
- Department Name
- U.O.S.D. Malattie Rare del Globulo Rosso - Dipartimento Onco-ematologico e Pneumo-toracico
- Contact Person Name
- Paolo Ricchi
- Contact Person Email
- paolo.ricchi@aocardarelli.it
Sponsor
Primary sponsor
- Full Name
- CSL Behring LLC
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- PPD Development LP
- Responsibilities
- IRB/EC Management, Health Authority Management, Vendor Management, Home Health Services, Site Payments, Translations
- Name
- PPD Global Central Labs
- Responsibilities
- Hematology, biochemistry, coagulation, immunogenicity, biomarkers, sample storage
- Name
- Fisher Clinical Services GmbH
- Responsibilities
- Clinical Trial Supply, label translations and creation of IMP labels, packaging and labeling of IMP, storage, distribution and destruction of IMP
Third parties
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties code 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"sponsorDuties code 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Center For Information And Study On Clinical Research Participation Inc.","duties_or_roles":"Patient Advisory Board and Study Materials [e.g., brochure, videos, ICF infographic, poster, communication cards (including thank you and unblinding cards)]","organisation_type":"Patient organisation/association"}
- {"country":"Australia","full_name":"CSL Innovation Pty Limited","duties_or_roles":"PK/Biomarker/Immuno/Retention/Future Research samples testing; code 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Responsibilities include IRB/EC Management, Health Authority Management, Vendor Management, Home Health Services, Site Payments, Translations (sponsorDuties codes 1,15,5,6 referenced)","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"Clinical Trial Supply, label translations and creation of IMP labels, packaging and labeling of IMP, storage, distribution and destruction of IMP","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Electronic Clinical Outcome Assessment (eCOA)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q2q Communications Limited","duties_or_roles":"Investigator Meeting","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Singapore","full_name":"Azenta Singapore Pte Ltd","duties_or_roles":"Long term sample storage","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Hematology, biochemistry, coagulation, immunogenicity, biomarkers, sample storage; code 4","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Anumigilimab
- Active Substance
- ANUMIGILIMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- SUBCUTANEOUS
- Route
- SUBCUTANEOUS
- Authorisation Status
- EU MP number PRD11100686 (prodAuthStatus:1)
- Maximum Dose
- 50 mg (maxDailyDoseAmount)
- Investigational Product Name
- Saline 0.9%
- Modality
- Other
Related trials
Other published trials that may interest you.