Nuwiq for Hemophilia A

Recruiting at 12 trial locations

Overseen By Sigurd Knaub

Age: Any Age

Sex: Female

Trial Phase: Phase 4

Sponsor: Octapharma

Must be taking: Factor VIII

Disqualifiers: Coagulation disorder, FVIII inhibitor, Severe liver/kidney disease, Pregnancy, 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

Duration: 30 ± 3 days

28 Participants Needed

Trial Summary

What is the purpose of this trial?

Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you have taken any investigational medicinal product within 30 days before the screening visit.

What data supports the effectiveness of the drug Nuwiq for Hemophilia A?

Clinical trials have shown that Nuwiq is effective in preventing and treating bleeds in patients with severe Hemophilia A, with 83% of patients experiencing no spontaneous bleeds during a 6-month period of personalized treatment. Additionally, it has a low rate of developing inhibitors, which are substances that can reduce the drug's effectiveness. ^{1 2 3 4 5}

Is Nuwiq safe for humans?

Nuwiq has been shown to be safe in clinical trials for both previously treated and untreated patients with hemophilia A, with no inhibitors detected in previously treated patients and a low rate of inhibitor development in previously untreated patients. ^{1 5 6 7 8}

What makes the drug Nuwiq unique for treating Hemophilia A?

Nuwiq is unique because it is a 4th generation recombinant factor VIII (rFVIII) produced in a human cell line, which helps ensure human-specific protein processing without chemical modifications or protein fusions. This approach may reduce the risk of developing inhibitors (immune responses) compared to other treatments. ^{1 2 4 5 9}

Research Team

Johannes Oldenburg

Principal Investigator

Experimental Haematology and Transfusion Medicine, University Clinic Bonn

Eligibility Criteria

This trial is for women and girls aged 12 or older with Hemophilia A, which means they have a Factor VIII level of at least 1% but less than 40%. They must be scheduled for major elective surgery that requires treatment with Factor VIII and agree to participate by signing a consent form.

Inclusion Criteria

Women/girls with haemophilia A (FVIII:C ≥1-<40%) according to medical history

I am 12 years old or older.

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

Exclusion Criteria

Pregnancy

Current participation in another interventional clinical trial

I have already undergone surgery as part of this study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Nuwiq is administered intravenously to provide haemostatic cover during major surgery, repeated every 8-24 hours until adequate wound healing, then for at least another 7 days if required

30 ± 3 days

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of adverse events and thrombotic events

30 ± 3 days

Treatment Details

Interventions

• Nuwiq

Trial Overview The study is testing Nuwiq, a recombinant factor VIII product. It's used to prevent bleeding in female patients with Hemophilia A who are about to undergo major surgery. The focus is on how well Nuwiq works and its safety during surgical procedures.

Participant Groups

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

Findings from Research

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 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&#174; ) in children with severe haemophilia A: efficacy, safety and pharmacokinetics. Klukowska, A., Szczepański, T., Vdovin, V., et al.[2019]

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]

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&#174; (human-cl rhFVIII) in children with severe haemophilia A. Klukowska, A., Szczepański, T., Vdovin, V., et al.[2018]

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&#174; (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]

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]

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 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]

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]

References

1. England 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. [2020]

2. England pubmed.ncbi.nlm.nih.gov

Novel, human cell line-derived recombinant factor VIII (Human-cl rhFVIII, Nuwiq&#174; ) in children with severe haemophilia A: efficacy, safety and pharmacokinetics. [2019]

3. England 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. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Long-term tolerability, immunogenicity and efficacy of Nuwiq&#174; (human-cl rhFVIII) in children with severe haemophilia A. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Immunogenicity, efficacy and safety of Nuwiq&#174; (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study. [2018]

6. England pubmed.ncbi.nlm.nih.gov

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

7. England 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. [2018]

8. England 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. [2023]

9. United States 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. [2015]