A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A
Status: Recruiting
Location: See all (42) locations...
Intervention Type: Drug, Combination product
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors or with hemophilia A of any severity (severe, moderate, and mild) with FVIII inhibitors.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:
• Diagnosis of severe (FVIII:C \<1 International Unit per decilitre \[IU/dL\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
• Diagnosis of mild (FVIII:C between \>5 IU/dL and \<40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor ( ≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to \<5 IU/dL for ≥12 months
• Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment
• Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening
• For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization
Locations
United States
California
Center for Inherited Blood Disorders
RECRUITING
Orange
Colorado
University of Colorado Hemophilia and Thrombosis Center
RECRUITING
Aurora
Florida
St Joseph's Children's Hospital of Tampa
RECRUITING
Tampa
Iowa
University Of Iowa Hospitals And Clinics
RECRUITING
Iowa City
Indiana
Innovative Hematology, Inc.
RECRUITING
Indianapolis
Washington
Washington Center for Bleeding Disorders
RECRUITING
Seattle
Other Locations
Argentina
Arbesu Hematologia
RECRUITING
Godoy Cruz
Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli
RECRUITING
Rosario
Belgium
Cliniques Universitaires Saint-Luc
RECRUITING
Brussels
UZ Leuven
RECRUITING
Leuven
Brazil
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
RECRUITING
Ribeirão Preto
China
Chengdu Women's and Children's Central Hospital
RECRUITING
Chengdu
Nanfang Hospital, Southern Medical University
RECRUITING
Guangzhou
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
SUSPENDED
Tianjin
Denmark
Rigshospitalet
RECRUITING
København Ø
France
Hospices Civils de Lyon
RECRUITING
Bron
AP-HP - Hôpital Bicêtre
RECRUITING
Le Kremlin-bicêtre
CHU de Lille - Institut Cœur Poumon
RECRUITING
Lille
Germany
Universitätsklinikum Bonn
RECRUITING
Bonn
Hungary
Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus
RECRUITING
Debrecen
Israel
The Chaim Sheba Medical Center - PPDS
RECRUITING
Ramat Gan
Italy
Azienda Ospedaliera Universitaria Careggi
RECRUITING
Florence
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
RECRUITING
Milan
IRCCS Istituto Clinico Humanitas
RECRUITING
Rozzano
Japan
Nara Medical University Hospital
RECRUITING
Kashihara-shi
Gunma University Hospital
RECRUITING
Maebashi
Nagoya University Hospital
RECRUITING
Nagoya
Ogikubo Hospital
RECRUITING
Suginami-ku
Netherlands
Universitair Medisch Centrum Utrecht Cancer Center - PPDS
RECRUITING
Utrecht
New Zealand
Auckland City Hospital
RECRUITING
Auckland
Republic of Korea
Kyungpook National University Hospital
RECRUITING
Daegu
South Africa
Haemophilia Comprehensive Care Centre
RECRUITING
Parktown
Spain
Hospital de la Santa Creu i Sant Pau
RECRUITING
Barcelona
Hospital Universitario Vall d'Hebron - PPDS
RECRUITING
Barcelona
Hospital Universitario La Paz - PPDS
RECRUITING
Madrid
Hospital Regional Universitario de Malaga ? Hospital General
RECRUITING
Málaga
Hospital Universitario Virgen del Rocio - PPDS
RECRUITING
Seville
Hospital Universitari i Politecnic La Fe de Valencia
RECRUITING
Valencia
Taiwan
National Taiwan University Hospital
RECRUITING
Taipei
United Kingdom
University Hospital of Wales
RECRUITING
Cardiff
Great Ormond Street Hospital
RECRUITING
London
The Royal London Hospital
RECRUITING
London
Contact Information
Primary
Reference Study ID Number: BO45887 https://forpatients.roche.com/ No attachments to email below.
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Backup
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
Time Frame
Start Date:2026-04-27
Estimated Completion Date:2032-01-29
Participants
Target number of participants:360
Treatments
Experimental: Main Study Treatment Period: NXT007 Prophylaxis
Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.
Active_comparator: Main Study Treatment Period: Emicizumab Prophylaxis
Participants randomized to this arm will receive emicizumab prophylaxis for the main study treatment period at 3 mg/kg once weekly (QW) for 4 weeks as loading doses, followed by maintenance dosing of either 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W). Loading doses are not required for participants who were taking emicizumab prior to study start.
After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the Emicizumab arm will be able to switch to NXT007, in the open-label extension period.