pause_circle_filledNot Yet Recruiting

Severe Hemophilia A

Bayer Identifier:
23153
ClinicalTrials.gov Identifier:
NCT07446010
EudraCT Number:
Not Available
EU CT Number:
Not Available
Post Approval observational study to learn more about how safe Octocog alfa is and how well it works in patients with severe Hemophilia A in India

Trial purpose

Hemophilia A is a genetic condition that makes it hard for blood to clot properly. This happens because the body does not have enough of a protein called Factor VIII, which helps stop bleeding. The main goal of treating someone with hemophilia is to stop and prevent bleeding by giving them the missing Factor VIII. This treatment can be given when a person starts bleeding (called on-demand treatment), or it can be given regularly to prevent bleeding (called prophylactic therapy). In India, most people with hemophilia A get treatment only when they have a bleeding episode, and only a few receive regular preventive treatment. Octocog alfa (also known as BAY 81-8973) is a modern, laboratory-made version of Factor VIII. It is made without using any human or animal materials and has special features that help it work better in the body. In India, Octocog alfa is approved for use in adults and children with hemophilia A to:
- Treat and control bleeding episodes when they happen
- Manage bleeding during surgery
- Prevent bleeding by giving regular treatment
The safety and effectiveness of Octocog alfa have been shown in several global studies. This new study is required by Indian health authorities to collect information about how safe Octocog alfa is and how well it works in people with hemophilia A who have already received treatment. The study will look at how Octocog alfa is used in real-life medical practice in India, including how doctors prescribe it, how patients use it, and what treatment results they have.

Key Participants Requirements

Sex

Male

Age

18 - N/A
    - Male patients aged 18 years or older with a documented diagnosis of severe Hemophilia A, defined by a baseline Factor VIII (FVIII) activity level of less than 1% (<0.01 IU/mL) in accordance with the Hemophilia Severity Classification
    - Previously treated with FVIII concentrate(s) (plasma derived or recombinant, including Octocog alfa) either on-demand or prophylactically for at least 100 Exposure Days (EDs).
    - Patients for whom the decision to initiate on-demand treatment with Octocog alfa for acute bleeding was made as per the investigator’s routine treatment practice. This will include patients who are already on on-demand treatment with Octocog alfa as well.
    - Written informed consent from the patient or legal representative
    - Known contraindication according to the local prescriber information
    - Patients who are participating in an investigational program with interventions outside of routine clinical practice.
    - Patients with any other diagnosis of bleeding/coagulation disorder other than Hemophilia A.
    - Patients who are on ongoing prophylactic treatment with any FVIII concentrate or non-factor treatments like emicizumab.
    - Patients exhibiting any of the following laboratory abnormalities at screening:
    – Known platelet count <100,000 mm3
    – Known Serum Creatinine >2 folds upper the normal limit
    – Known Hepatic AST or ALT >5 folds upper the normal limit
    - Patients who have received an on-demand infusion with any other FVIII (different to Octocog alfa), FVII or Activated prothrombin complex concentrate product (aPCC/FEIBA) 72 hours before the enrollment.
    - History or presence of FVIII inhibitor with a titer ≥ 0.6 with Nijmegen modified Bethesda Assay (NBA), or a clinical history suggestive of an inhibitor necessitating changes to treatment
    - Patient concerns or other barriers precluding adequate understanding or cooperation.

Trial summary

Enrollment Goal
33
Trial Dates
May 2026 - December 2026
Phase
N/A
Could I Receive a placebo
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Not yet recruiting
Department of Medicine Assam Medical College & HospitalDibrugarh, India
Not yet recruiting
All India Institute Of Medical SciencesNew Delhi, India
Not yet recruiting
Christian Medical College & HospitalLudhiana, India
Not yet recruiting
Government of Medical College KozhikodeKozhikode, India
Not yet recruiting
Sahyadri Super Speciality HospitalPune, India
Not yet recruiting
Sanjay Gandhi Post Graduate Institute & Medical SciencesLucknow, India

Primary Outcome

  • Duration of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
    date_rangeTime Frame:
    12 weeks
  • Severity of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
    The severity (or intensity) of an AE will be evaluated by the Investigator in accordance with the CTCAE v 5.0. - Grade 1 (Milde): Asymptomatic or mild symptoms ; clinical or diagnostic observations only ; intervention not indicated. - Grade 2 (Moderate): Minimal, local or invasive intervention indicated, limiting age-appropriate instrumental activities of daily living. - Grade 3 (Severe): Severe or medically significant but not immediately life threatening; hospitalization or prolongation of existing hospitalization indicated ; disabling; limiting self-care activities of daily living. - Grade 4 (Life threatening Consequences): Urgent intervention indicated. -Grade 5 (Death): Related to Adverse Event
    date_rangeTime Frame:
    12 weeks
  • Outcome of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
    The outcome is defined by the following categories. - Recovered or Resolved: The subject has completely recovered or resolved from the Serious Adverse Event. - Recovered or Resolved with sequelae: As a result of AE , the subject suffered persistent and significant disability / incapacity (e.g., blind, deaf and paralysed ). Any AE recovered with sequelae should be rated as an SAE . - Recovering or Resolving: The subject has begin to recover from the condition or injury , but the event has considered ongoing at a reduced intensity. - Not Recovered or Not Resolved: The AE itself is still present and observable. - Fatal: Death due to SAE, mention the cause of death. Unknown: This term should only be used in cases where the subject is lost to follow up .
    date_rangeTime Frame:
    12 weeks
  • Treatment administered for treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
    date_rangeTime Frame:
    12 weeks
  • Laboratory test results related to adverse events of special interest (AESIs)rse events (TESAEs)
    hypersensitivity, inhibitor development Tests used will be as per the routine clinical practice followed by the investigator at his/her hospital
    date_rangeTime Frame:
    12 weeks

Secondary Outcome

  • Total number of infusions per bleed
    date_rangeTime Frame:
    12 weeks
  • Dose of Octocog alfa (IU/kg) per bleed
    date_rangeTime Frame:
    12 weeks
  • Location of bleeds
    date_rangeTime Frame:
    12 weeks
  • Type of bleeds
    date_rangeTime Frame:
    12 weeks
  • Severity of bleeds
    Severity is defined and measured as follows. - Mild: Spontaneous bleeding into joints or muscles, predominantly in the absence of identifiable hemostatic challenge. The bleeding stops on its own or with pressure or the bleeding stops or slows to an ooze or trickle after 15 minutes of pressure. It may ooze or trickle for up to 45 min. - Moderate: Occasional spontaneous bleeding; prolonged bleeding with minor trauma or surgery, the bleeding slows or stops wtih presure, but starts again if you remove the pressure or the blood may soak through a few bandages, but it is not fast or out of control. - Severe: blood is pumping from the wound or the bleeding does not stopr or slow down with pressure or blood is quickly soaking through bandage after bandage
    date_rangeTime Frame:
    12 weeks
  • Duration of bleeds
    date_rangeTime Frame:
    12 weeks
  • Investigator rating of response
    poor, moderate, good, excellent
    date_rangeTime Frame:
    12 weeks

Trial design

A prospective, multicenter, open-label, Phase IV post-authorization safety study conducted in India to assess the safety and treatment outcomes of Octocog alfa in real-world practice for on-demand treatment of acute bleeds in previously treated severe Hemophilia A patients in India
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
N/A
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A