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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

NCT05987449•Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

2 - 59 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•Diagnosis of severe (Factor VIII \[FVIII\] coagulant activity \<1 IU/dL) or moderate (FVIII coagulant activity ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
•Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures
•Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status
•Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as \<0.6 Bethesda unit (BU)/mL (\<1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and in vivo recovery \>66%
•Documentation of number and type of bleeding episodes in the last 24 weeks prior to enrollment
•Adequate hematologic function, defined as platelet count ≥100,000 cells/μL and hemoglobin ≥11 g/dL at the time of screening
•Adequate hepatic function defined as total bilirubin ≤1.5× age-adapted upper limit of normal (ULN) (excluding Gilbert syndrome) and both aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3× age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis. For patients with Gilbert syndrome, bilirubin should be \<4 mg/dL or 68.4 umol/L at the time of screening.
•For Part 1 only: Adequate renal function, defined as serum creatinine ≤2.5× age-adapted ULN and calculated creatinine clearance ≥30 mL/min by Cockroft-Gault formula
•For Part 2 only: Adequate renal function, defined as serum creatinine ≤1.5× age-adapted ULN. When the serum creatinine is ≥1.5× ULN, creatinine clearance by Bedside Schwartz formula must be \>70 mL/min/1.73m\^2.
•Willingness and ability to comply with schedules visits, treatment plans, laboratory tests, and other study procedures

Exclusion Criteria

•Inherited or acquired bleeding disorders other than congenital hemophilia A
•Ongoing or planned ITI therapy
•Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
•At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigator's judgment
•For Part 1 only: Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus
•For Part 1 only: Strong family history of ischemic heart disease or cerebrovascular disease (i.e., first degree relatives such as parents, full siblings, or children): male relatives diagnosed under the age of 55 years and females under the age of 65 years
•For Part 1 only: Previous or concomitant malignancies or leukemia
•Other conditions (e.g., autoimmune conditions such as Systemic Lupus erythematosus and other systemic inflammatory disorders) that may currently increase the risk of bleeding or thrombosis
•History of clinically significant allergies
•Receipt of any of the following:
+9 more criteria

Locations (13)

UC Davis Cancer Center

Sacramento, California, United States

Georgetown Uni Medical Center

Washington D.C., District of Columbia, United States

Indiana Hemophilia & Thrombosis center

Indianapolis, Indiana, United States

University of Iowa Hospitals and Clnics Dept of Pediatrics

Iowa City, Iowa, United States

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

Hamilton Health Sciences Corporation

Hamilton, Ontario, Canada

Istituto Clinico Humanitas

Rozzano (MI), Lombardy, Italy

Auckland Cancer Trial Centre

Auckland, New Zealand

Uniwersyteckie Centrum Kliniczne

Gda?sk, Poland

Instytut Hematologii i Transfuzjologii

Warsaw, Poland

Key Dates

Start Date

September 21, 2023

Primary Completion Date

June 16, 2030

Completion Date

June 16, 2030

Last Updated

March 5, 2026

Enrollment

ESTIMATED participants

Conditions

Hemophilia A

Interventions

NXT007

DRUG

Contacts

Reference Study ID Number: WP44714 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S. Only)global-roche-genentech-trials@gene.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 5, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Inclusion Criteria

Exclusion Criteria

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