Nuwiq for Hemophilia A

(NuPOWER Trial)

Recruiting at 18 trial locations

Overseen By Sigurd Knaub, PhD

Age: Any Age

Sex: Male

Trial Phase: Phase 4

Sponsor: Octapharma

Must be taking: Emicizumab

Must not be taking: Investigational drugs

Disqualifiers: Coagulation disorders, Liver disease, others

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

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

28 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Nuwiq, a treatment designed to prevent bleeding in people with severe hemophilia A undergoing major surgery. The goal is to evaluate Nuwiq's effectiveness when patients are already taking emicizumab, another medication for bleeding prevention. Nuwiq will be administered through an IV to maintain specific blood levels that aid healing. Individuals with severe hemophilia A who have used emicizumab for at least a month before surgery may be suitable for this trial. As a Phase 4 trial, Nuwiq is already FDA-approved and proven effective, and this research aims to understand its benefits for more patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be on regular prophylaxis with emicizumab for at least 1 month before surgery. If you are taking any investigational drugs, you must stop them at least 30 days before joining the trial.

What is the safety track record for Nuwiq?

Research has shown that Nuwiq is generally safe for both treated and untreated patients with Hemophilia A. Studies indicate a low risk of developing inhibitors, substances that can reduce treatment effectiveness. In several clinical studies, patients reported no major unexpected side effects. The FDA has approved Nuwiq for use in both children and adults with Hemophilia A, suggesting a reliable safety record. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Nuwiq is unique because it is a recombinant factor VIII (FVIII) treatment specifically designed for Hemophilia A. Unlike plasma-derived FVIII products, Nuwiq is created using recombinant DNA technology, which reduces the risk of blood-borne pathogen transmission. Researchers are excited about Nuwiq because it can be administered intravenously and tailored to the patient's needs, ensuring consistent FVIII levels for effective wound healing. This flexibility in dosing every 8-24 hours, potentially extending treatment for an additional week, makes it a promising option for maintaining steady FVIII plasma levels and improving patient outcomes.

What is the effectiveness track record for Nuwiq in treating Hemophilia A?

Research has shown that Nuwiq, the treatment under study in this trial, effectively treats Hemophilia A. Studies have demonstrated that Nuwiq successfully controls bleeding and achieves 'excellent' or 'good' results during surgeries. One study found it worked as well as another treatment, with a strong response in 77% of cases. Another review confirmed Nuwiq's safety and effectiveness for both adults and children with Hemophilia A. Its approval for use underscores its reliability in managing bleeding episodes. ^{1 6 7 8 9}

Who Is on the Research Team?

Shveta Gupta, MD

Principal Investigator

Arnold Palmer Hospital for Children

Are You a Good Fit for This Trial?

This trial is for male patients aged 12 or older with severe Hemophilia A (less than 1% normal clotting factor VIII activity) who have been treated with any Factor VIII products for at least 150 days and are on regular emicizumab prophylaxis for a minimum of three months before planned major surgery. Consent must be given as per local rules.

Inclusion Criteria

I am a male and at least 12 years old.

I have been treated with Factor VIII products for over 150 days.

Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Perioperative Treatment

Administration of Nuwiq (recombinant FVIII) in combination with emicizumab prophylaxis during major surgery

During surgery

Continuous monitoring during surgery

Postoperative Treatment

Continued administration of Nuwiq to maintain FVIII plasma levels for at least 7 days post-surgery

7 days

Follow-up

Participants are monitored for safety, effectiveness, and adverse events after treatment

30 ± 3 days

What Are the Treatments Tested in This Trial?

Interventions

• Nuwiq

Trial Overview The study tests Nuwiq, a recombinant factor VIII product, to prevent bleeding in patients with severe hemophilia A during major surgeries while they're also receiving emicizumab prophylaxis.

How Is the Trial Designed?

Group I: Nuwiq Experimental Treatment 1 Intervention

All patients receiving Nuwiq (recombinant FVIII). Nuwiq will be administered intravenously in accordance with the relevant prescribing information. Treatment will be repeated as necessary every 8-24 hours until adequate wound healing, then - if required - for at least another 7 days to maintain FVIII plasma levels of 30-60 IU/dL.

Nuwiq is already approved in United States, Canada for the following indications:

Approved in United States as Nuwiq for:

• Hemophilia A

Approved in Canada as Nuwiq for:

• Hemophilia A

Who Is Running the Clinical Trial?

Octapharma

Lead Sponsor

Trials

86

Recruited

11,300+

Wolfgang Marguerre

Octapharma

Chief Executive Officer since 1983

MBA from INSEAD

Wolfgang Frenzel

Octapharma

Chief Medical Officer since 2010

MD from University of Vienna

Published Research Related to This Trial

In a study of 66 previously untreated patients with severe hemophilia A, Nuwiq® showed a cumulative incidence of high-titre inhibitors at 12.8%, indicating a relatively low risk of developing inhibitors compared to other treatments.

Nuwiq® demonstrated excellent efficacy in managing bleeding episodes, with 91.8% of treated bleeds rated as 'excellent' or 'good', and no significant tolerability concerns were reported during the treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunogenicity, efficacy and safety of Nuwiq® (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study. Liesner, RJ., Abashidze, M., Aleinikova, O., et al.[2018]

Human-cl rhFVIII (Nuwiq®) is a new recombinant factor VIII produced in a human cell line, achieving high purity and recovery rates through a multi-step purification process that includes advanced techniques like affinity chromatography and pathogen clearance steps.

The final product demonstrates a specific activity of over 9000 IU/mg and is free from animal-derived products, ensuring a high safety margin against potential contaminants and pathogens.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development, upscaling and validation of the purification process for human-cl rhFVIII (Nuwiq®), a new generation recombinant factor VIII produced in a human cell-line. Winge, S., Yderland, L., Kannicht, C., et al.[2015]

In the NuProtect study involving 108 previously untreated patients with severe hemophilia A, simoctocog alfa demonstrated high efficacy, with a median annualized bleeding rate of 0 during continuous prophylaxis, indicating effective prevention of bleeding episodes.

The treatment was well tolerated, with no safety concerns reported, including no thromboembolic events, and a high success rate in treating bleeding episodes (92.9% rated as excellent or good) and surgical prophylaxis (94.7% rated as excellent or good).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study. Mathias, M., Abraham, A., Belletrutti, MJ., et al.[2023]

In a phase III study involving 59 children aged 2-12 with severe hemophilia A, Nuwiq® (Human-cl rhFVIII) demonstrated excellent efficacy in preventing and treating bleeding episodes, with 91.5% of children showing 'excellent' or 'good' results for all bleeds.

The treatment was safe, with no major or life-threatening bleeds reported, and no serious adverse events or FVIII inhibitors observed, indicating a favorable safety profile for this new recombinant factor VIII therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Novel, human cell line-derived recombinant factor VIII (Human-cl rhFVIII, Nuwiq® ) in children with severe haemophilia A: efficacy, safety and pharmacokinetics. Klukowska, A., Szczepański, T., Vdovin, V., et al.[2019]

In a long-term study involving 49 children with severe hemophilia A, Nuwiq® was found to be well tolerated with no patients developing drug-related adverse events or inhibitors, indicating a strong safety profile.

Nuwiq® significantly reduced annualized bleeding rates, especially in younger children, with a 71% reduction in spontaneous bleeds compared to the previous study, demonstrating its efficacy in preventing bleeding episodes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term tolerability, immunogenicity and efficacy of Nuwiq® (human-cl rhFVIII) in children with severe haemophilia A. Klukowska, A., Szczepański, T., Vdovin, V., et al.[2018]

Simoctocog alfa (Nuwiq®) is a recombinant FVIII treatment for severe hemophilia A that has shown efficacy and safety in clinical trials involving 201 previously treated patients, with a mean half-life of 15.1-17.1 hours in adults and adolescents.

In a study of personalized prophylaxis, 83% of adult patients experienced no spontaneous bleeds over six months, and no FVIII inhibitors were detected after over 43,000 injections, indicating a strong safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program. Lissitchkov, T., Klukowska, A., Pasi, J., et al.[2020]

In a study involving 111 previously treated subjects with hemophilia A, the recombinant antihemophilic factor rAHF-PFM was found to be bioequivalent to the standard treatment RECOMBINATE rAHF, demonstrating similar pharmacokinetic properties and safety profiles.

rAHF-PFM effectively managed 93% of bleeding events with just one or two infusions, and showed no significant increase in immunogenicity, indicating it is a safe and effective treatment option for hemophilia A.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Tarantino, MD., Collins, PW., Hay, CR., et al.[2018]

In a phase 3 trial involving 81 previously untreated patients with hemophilia A, long-term treatment with the extended half-life factor VIII product N8-GP showed a 30% incidence of FVIII inhibitors, which is consistent with previous findings.

During the prophylaxis regimen, patients experienced a median annualized bleeding rate of 1.4, with 13% having no bleeding episodes and a high hemostatic success rate of 87.6% for treating bleeding episodes, indicating effective management of hemophilia A.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The immunogenicity, safety, and efficacy of N8-GP in previously untreated patients with severe hemophilia A: pathfinder6 end-of-trial results. Kenet, G., Young, G., Chuansumrit, A., et al.[2023]

Simoctocog alfa (Nuwiq), a new recombinant factor VIII product, shows a high efficacy and safety profile in treating hemophilia A, particularly in previously treated patients (PTPs).

Preliminary data from phase III trials suggest that simoctocog alfa may reduce the risk of inhibitor development in previously untreated patients (PUPs), which is a significant concern in hemophilia A treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa). Franchini, M., Mannucci, PM.[2017]

Citations

1. nuwiqusa.com nuwiqusa.com/safety/

Proven Hemophilia A Treatment with Low Risk Inhibitors Learn about NUWIQ® [Antihemophilic Factor (Recombinant)] safety in treated and untreated patients with Hemophilia A. Discover how it controls bleeding.

2. octapharma.com octapharma.com/news/press-release/2023/new-nuwiq-study-published-in-the-european-journal-of-haematology

New Nuwiq® study published in the European Journal of ... The efficacy of Nuwiq® was similar to that of Adynovate® in the 8–12% arm (77% vs 62%). The mean annualised bleeding rate was significantly ...

3. fda.gov fda.gov/files/vaccines,%20blood%20&%20biologics/published/Clinical-Review---NUWIQ.pdf

Clinical Review - NUWIQ Based on my review of the submitted, data NUWIQ® appears safe and effective in pediatric and adult patients with hemophilia A. An approval is recommended.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/26315974/

Novel, human cell line-derived recombinant factor VIII ... Efficacy was 'excellent' or 'good' in treating 28 (100%) of 28 bleeds. Overall efficacy was rated as 'excellent' during four surgical procedures ...

5. rarediseaseadvisor.com rarediseaseadvisor.com/therapies/nuwiq-simoctocog-alfa-antihemophilic-factor-recombinant/

Nuwiq (Simoctocog Alfa - Hemophilia In a study of 20 adults and 2 adolescents treated on-demand with Nuwiq, the effectiveness of the treatment was excellent or good in 931 of 986 ( ...

6. fda.gov fda.gov/media/93485/download

Package Insert - NUWIQ The safety profile of NUWIQ was evaluated in seven prospective, open-label clinical studies in previously treated patients (PTPs - exposed to a Factor VIII ...

7. nuwiqusa.com nuwiqusa.com/hcp/clinical-evidence/nuwiq-safety-in-previously-untreated-patients-pups/

NUWIQ® – Safety in Previously Untreated Patients (PUPs) Learn about NUWIQ® safety in previously untreated patients (PUPs) with hemophilia A. Final NuProtect results show a low incidence of inhibitors.

8. nuwiq.com nuwiq.com/safefy-information/

Safefy information – Nuwiq Nuwiq is a recombinant antihemophilic factor [coagulation factor VIII (Factor VIII)] indicated in pediatric and adult patients with Hemophilia A

9. factormyway.com factormyway.com/html/pdf/nuwiq-patient-brochure.pdf

NUWIQ Patient Brochure FVIII replacement therapy is the standard of care for hemophilia A.1. FVIII replacement is a trusted therapy that has been proven to be safe and ...