NXT007 vs Emicizumab for Hemophilia A

(ZEBRHA 2 Trial)

Recruiting at 5 trial locations

Overseen By Reference Study ID Number: BO45887 https://forpatients.roche.com/

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Hoffmann-La Roche

Must be taking: Emicizumab

Must not be taking: Systemic immunomodulators

Disqualifiers: Ventricular dysrhythmias, Structural heart disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks for loading doses, followed by maintenance dosing

360 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

Inclusion Criteria

I have moderate or severe hemophilia A, with or without inhibitors.

I have mild hemophilia A with long-term FVIII inhibitors and low FVIII levels for over a year.

* Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either NXT007 or Emicizumab prophylaxis during the main study treatment period

4 weeks for loading doses, followed by maintenance dosing

Weekly visits for loading doses, then variable for maintenance

Open-label extension

Participants may continue with NXT007 dosing or switch from Emicizumab to NXT007

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Emicizumab
• NXT007

How Is the Trial Designed?

Group I: Open-Label Extension Period: NXT007 Prophylaxis Experimental Treatment 1 Intervention

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.

Group II: Main Study Treatment Period: NXT007 Prophylaxis Experimental Treatment 1 Intervention

Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.

Group III: Main Study Treatment Period: Emicizumab Prophylaxis Active Control 1 Intervention

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.

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Chugai Pharmaceutical

Industry Sponsor

Trials

105

Recruited

25,000+

Dr. Osamu Okuda

Chugai Pharmaceutical

Chief Executive Officer since 2020

MD from Kyoto University

Dr. Mariko Y. Momoi

Chugai Pharmaceutical

Chief Medical Officer

MD from Jichi Medical University