Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Nonacog beta pegol · 7 trials · 2 indications
Haemostatic effect of N9-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms.
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Haemostatic effect during surgery was evaluated immediately after surgery (last stitch) using a fourpoint response scale: \- Four-point response scale: Excellent, good, moderate, poor. The evaluation was done by the surgeon, anaesthesiologist and/or investigator based on experience as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure. 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. 4. Poor: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported.
The primary endpoint was incidence of inhibitors against coagulation factor nine (FIX) defined as titre ≥0.6 Bethesda unit (BU). Number of subjects who developed inhibitors against FIX are reported.
Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported.
| Arm | Type | Description |
|---|---|---|
| Arm A - Nonacog beta pegol (On-demand/Prophylaxis) | EXPERIMENTAL | Participants on on-demand treatment for 28 weeks, thereafter prophylactic treatment |
| Arm B - Nonacog beta pegol (Prophylaxis) | EXPERIMENTAL | Participants on prophylactic treatment only |
| 50 EDs (exposure days) | EXPERIMENTAL | - |
| Patients enrolled in trial | EXPERIMENTAL | New patients will be included as well as transferred patients from Paradigm™ 2 or Paradigm™ 4 trial |
| NNC-0156-000-0009 | EXPERIMENTAL | - |
| Prophylaxis, high dose (once weekly) | EXPERIMENTAL | - |
| Prophylaxis, low dose (once weekly) | EXPERIMENTAL | - |
| On-demand | EXPERIMENTAL | - |
| Prophylaxis, high dose (every second week) | EXPERIMENTAL | - |
| Prophylaxis, high dose (trial duration 52 weeks) | EXPERIMENTAL | - |
| Prophylaxis, low dose (trial duration 52 weeks) | EXPERIMENTAL | - |
| On-demand (trial duration 28 weeks) | EXPERIMENTAL | - |
| 25U/kg | EXPERIMENTAL | - |
| 50U/kg | EXPERIMENTAL | - |
| 100U/kg | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Nonacog beta pegol | DRUG | Nonacog beta pegol is administered as intravenous injections. Participants will receive nonacog beta pegol as prophylaxis, as on-demand for treatment of bleeding episodes and in relation to surgery. |
Inclusion Criteria: * Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial. * Male Chinese patient with moderate to severe congenital haemophi...
Nonacog beta pegol is an investigational therapy for congenital bleeding disorder and haemophilia B. It is being developed as a treatment for these conditions, which are characterized by a deficiency in clotting factor IX. The drug is intended for use in patients with haemophilia B to help manage bleeding episodes.
Nonacog beta pegol is being developed by Novo Nordisk A/S, a pharmaceutical company listed on the stock exchange under the ticker NVO. The company is conducting clinical trials to evaluate the safety and efficacy of this drug for the treatment of haemophilia B.
Nonacog beta pegol is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The Phase 3 trials are designed to assess its safety and efficacy in patients with haemophilia B, including previously untreated patients and those undergoing surgical procedures.
Nonacog beta pegol has been studied in several clinical trials, including NCT00956345, a Phase 1 safety study, and Phase 3 trials NCT01386528, NCT01467427, and NCT02141074. These trials evaluated the drug in non-bleeding patients, during surgical procedures, in previously treated children, and in previously untreated patients with haemophilia B.
Nonacog beta pegol is a small molecule therapy designed to address haemophilia B, a condition caused by a deficiency in clotting factor IX. While the specific molecular target is not detailed, the drug is intended to replace or supplement the missing clotting factor to help restore normal blood coagulation and reduce bleeding risk.
Yes, Nonacog beta pegol is also known as NNC-0156-0000-0009, as referenced in clinical trial titles. This alternative name is used in studies such as NCT01386528 and NCT01467427, which evaluate the drug's efficacy and safety in patients with haemophilia B.