Actively Recruiting
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Led by Hoffmann-La Roche · Updated on 2026-05-05
60
Participants Needed
14
Research Sites
351 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: * Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. * Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors. The overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
CONDITIONS
Official Title
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 2 to 59 years
- Diagnosis of severe (FVIII coagulant activity <1 IU/dL) or moderate (FVIII coagulant activity 61 IU/dL and 65 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
- Participants with FVIII inhibitors using or willing to use recombinant activated factor VII (rFVIIa) as primary bypassing agent
- Availability of historic local FVIII inhibitor test results during screening
- Participants who completed immune tolerance induction (ITI) at least 5 years prior with no inhibitor recurrence
- Documentation of bleeding episodes in the last 24 weeks before enrollment
- Adequate hematologic function: platelet count 61 100,000 cells/bcL and hemoglobin 61 11 g/dL at screening
- Adequate hepatic function: total bilirubin 61 1.5 �d7 age-adapted upper limit of normal (ULN) (excluding Gilbert syndrome), AST and ALT 61 3 �d7 age-adapted ULN, no clinical or radiographic cirrhosis; for Gilbert syndrome bilirubin <4 mg/dL or 68.4 umol/L
- Adequate renal function: serum creatinine 61 2.5 �d7 age-adapted ULN and creatinine clearance 61 30 mL/min (Part 1); serum creatinine 61 1.5 �d7 age-adapted ULN or creatinine clearance >70 mL/min/1.73m2 (Part 2)
- Willingness and ability to comply with study visits, treatment, and procedures
You will not qualify if you...
- Inherited or acquired bleeding disorders other than congenital hemophilia A
- Ongoing or planned immune tolerance induction (ITI) therapy
- Previous or current thromboembolic disease treatment, except resolved catheter-associated thrombosis
- High risk for thrombotic microangiopathy (TMA) per investigator judgment
- Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus (Part 1 only)
- Strong family history of ischemic heart disease or cerebrovascular disease in male relatives diagnosed under 55 years or female relatives under 65 years (Part 1 only)
- Previous or current malignancies or leukemia (Part 1 only)
- Conditions increasing bleeding or thrombosis risk, including autoimmune disorders
- History of significant allergies
- Recent or current use of investigational drugs or gene therapy as specified
- Low protein C activity, protein S free antigen, or anti-thrombin III activity levels at screening
- Known HIV infection with CD4 count <200 cells/bcL
- History of severe allergic reactions to monoclonal antibodies or fusion proteins
- Known hypersensitivity to Chinese hamster ovary cell products or excipients
- Clinically significant abnormal ECG findings including specific conduction blocks, atrial fibrillation, or prior myocardial infarction
- QTcF interval >450 ms confirmed by two ECGs
- History or risk factors for ventricular dysrhythmias
- Current treatment with medications known to prolong QT interval
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
UC Davis Cancer Center
Sacramento, California, United States, 95817
Actively Recruiting
2
Georgetown Uni Medical Center
Washington D.C., District of Columbia, United States, 20007
Withdrawn
3
Indiana Hemophilia & Thrombosis center
Indianapolis, Indiana, United States, 46260
Actively Recruiting
4
University of Iowa Hospitals and Clnics Dept of Pediatrics
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
British Columbia Children's Hospital
Vancouver, British Columbia, Canada, V6H 3N1
Actively Recruiting
6
Hamilton Health Sciences Corporation
Hamilton, Ontario, Canada, L8N 3Z5
Actively Recruiting
7
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy, 20122
Actively Recruiting
8
Istituto Clinico Humanitas
Rozzano (MI), Lombardy, Italy, 20089
Actively Recruiting
9
Auckland Cancer Trial Centre
Auckland, New Zealand, 1023
Actively Recruiting
10
Uniwersyteckie Centrum Kliniczne
Gda?sk, Poland, 80-214
Actively Recruiting
11
Instytut Hematologii i Transfuzjologii
Warsaw, Poland, 02-776
Actively Recruiting
12
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain, 08950
Actively Recruiting
13
Hospital Universitario la Paz
Madrid, Spain, 28046
Actively Recruiting
14
Hospital Regional Universitario Carlos Haya
Málaga, Spain, 29010
Active, Not Recruiting
Research Team
R
Reference Study ID Number: WP44714 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
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