Actively Recruiting
A Phase I/II 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 2026-06-04
60
Participants Needed
14
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating NXT007 in a Phase I/II global trial for males aged 2 to 59 years with severe or moderate hemophilia A, including those with or without factor VIII inhibitors. The study aims to assess the safety, tolerability, how the body processes the drug, immune response, and effectiveness of NXT007. Participants will receive NXT007 through subcutaneous injections starting with two loading doses every two weeks, followed by maintenance doses every four weeks. The trial has two parts: Part 1 involves adult and adolescent males receiving escalating doses, while Part 2 includes pediatric males receiving multiple doses. This design allows researchers to study different age groups and dose levels. During the study, participants will undergo regular assessments including monitoring adverse events, blood tests for hematology and chemistry, vital signs, and ECGs for heart function. Researchers will measure drug levels in the blood, immune responses, and bleeding rates over up to 7.5 years. Participants will follow scheduled visits, treatment plans, and laboratory tests to provide comprehensive data on NXT007's impact and safety.
CONDITIONS
Brief 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...
- Diagnosis of severe (Factor VIII coagulant activity <1 IU/dL) or moderate (FVIII coagulant activity 651 IU/dL and 655 IU/dL) congenital hemophilia A with or without FVIII inhibitors
- Use of recombinant activated factor VII (rFVIIa) or willingness to switch to rFVIIa for treatment of breakthrough bleeds, trauma, or procedures for participants with FVIII inhibitors
- Availability of historic local FVIII inhibitor test results during screening
- Successful completion of immune tolerance induction at least 5 years before screening with no inhibitor recurrence
- Documentation of number and type of bleeding episodes in last 24 weeks prior to enrollment
- Adequate hematologic function (platelet count 65100,000 cells/bcL and hemoglobin 6511 g/dL) at screening
- Adequate hepatic function with specified bilirubin and liver enzyme limits and no cirrhosis
- Adequate renal function defined differently for Part 1 and Part 2 participants
- Willingness and ability to comply with scheduled visits, treatments, laboratory tests, and study procedures
You will not qualify if you...
- Inherited or acquired bleeding disorders other than congenital hemophilia A
- Ongoing or planned immune tolerance induction therapy
- Previous or current treatment for thromboembolic disease, except resolved catheter-associated thrombosis
- High risk for thrombotic microangiopathy based on history or investigator judgment
- For Part 1 only: personal or strong family history of ischemic heart disease, cerebrovascular disease, diabetes, or previous/concomitant malignancies or leukemia
- Conditions increasing risk of bleeding or thrombosis such as autoimmune disorders
- History of significant allergies
- Receipt of investigational drugs within specified timeframes or prior gene therapy
- Low protein C, protein S, or anti-thrombin III activity levels
- Known HIV infection with low CD4 counts
- History of severe allergic reactions to monoclonal antibody therapy or related products
- Known hypersensitivity to Chinese hamster ovary cell products or excipients
- Clinically significant abnormal ECG findings or prolonged QT interval
- History or risk factors for ventricular dysrhythmias
- Current use of medications that prolong QT interval
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 7.5 years or until study completion or discontinuation
Participants receive NXT007 with 2 loading doses once every two weeks followed by maintenance doses once every 4 weeks. Treatment includes safety, pharmacokinetics, pharmacodynamics, and efficacy assessments.
Visits occur frequently during the first 23 weeks, every 28 days from Week 25 to Week 49, and every 12 weeks thereafter
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
Active, Not 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/
F
Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry
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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