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Tests treatment safety and results for Hemophilia A
Official title A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
ClinicalTrials.gov ID: NCT05987449
What this study is testing
What is NXT007?
NXT007 is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for hemophilia a.
Also referred to as Zemocimig, RO7589655.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 59, men only
You may be able to join if
- Diagnosis of severe (Factor VIII [FVIII] coagulant activity \<1 IU/dL) or moderate (FVIII coagulant activity ≥1 IU/dL and ≤5 IU/dL) congenital...
- Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing...
- 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...
- Documentation of number and type of bleeding episodes in the last 24 weeks prior to enrollment
You likely can't join if
- 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...
- 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...
See the full eligibility 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...
- 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×...
- 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
- 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...
- 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: i) An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration or normalization of targeted parameters (e.g., anti-thrombin)...
- Protein C activity, protein S free antigen, or anti-thrombin III activity levels below the lower limit of the reference range at screening
- Known HIV infection with CD4 counts \<200 cells/μL
- History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins
- Known hypersensitivity to Chinese hamster ovary cell products or to excipient content
- History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third -degree atrioventricular heart block), including atrial fibrillation or evidence...
- QT interval corrected through use of Fridericia's formula (QTcF) \>450 ms demonstrated by at least two ECGs \>30 minutes apart
- History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart...
- Current treatment with medications that are well known to prolong the QT interval
The study team makes the final eligibility decision.
Where it's taking place
- Sacramento, California, United States
- Washington D.C., District of Columbia, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Vancouver, British Columbia, Canada
- Hamilton, Ontario, Canada
- Milan, Lombardy, Italy
- Rozzano (MI), Lombardy, Italy
- Auckland, New Zealand
- Gda?sk, Poland
- Warsaw, Poland
- Esplugues de Llobregat, Barcelona, Spain
- Madrid, Spain
- Málaga, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 59 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sacramento, California, United States; Washington D.C., District of Columbia, United States; Indianapolis, Indiana, United States; Iowa City, Iowa, United States; Vancouver, British Columbia, Canada; Hamilton, Ontario, Canada and 8 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.