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NCT01489111: pathfinder™3

Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.

Completed Phase 3 Results posted Last updated 10 August 2020
What this trial tests

Phase 3 trial testing turoctocog alfa pegol in Congenital Bleeding Disorder in 36 participants. Completed in 10 December 2018.

Timeline
3 August 2012
Primary endpoint
10 December 2018
10 December 2018

Quick facts

Lead sponsorNovo Nordisk A/S
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment36
Start date3 August 2012
Primary completion10 December 2018
Estimated completion10 December 2018
Sites70 locations across Italy, Japan, Malaysia, Taiwan, Croatia, Denmark, Netherlands, Sweden

Drugs / interventions tested

Conditions studied

Sponsor

Novo Nordisk A/S — full company profile →

Who can join

12 and older, male only, with Congenital Bleeding Disorder or Haemophilia A. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or None Primary · Assessed by the Investigator/surgeon at the day of surgery

Haemostatic effect during surgery was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. This was assessed after completion of surgery (defined as "last stitch"). Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.

Excellent
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)25
Good
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)22
Moderate
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)2
None
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
Average Consumption of N8-GP During Surgery Secondary · During surgery, defined as the time from "knife to skin" until "last stitch"

Average consumption of N8-GP, during surgery is presented. The time during surgery is defined from 'knife to skin' until 'last stitch'.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)20.7± 0.0
Haemostatic Effect of N8-GP During the Post-operative Period Days 1-6 Secondary · During the post-operative period, days 1-6

Haemostatic effect during post-operative period days 1-6 as evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.

Excellent
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
Good
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)1
Moderate
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
None
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
Missing
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)1
Haemostatic Effect of N8-GP During the Post-operative Period Days 7-14 Secondary · During the post-operative period, days 7-14

Haemostatic effect during post-operative period days 1-6 and days 7-14 was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.

Excellent
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)1
Good
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)1
Moderate
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
None
GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
Average Consumption of N8-GP During the Post-operative Period Days 1-6 Secondary · During the post-operative period, days 1-6

Average consumption of N8-GP during post operative period days 1-6 is presented. Analysis population: Full analysis set included all subjects exposed to the trial drug (N8-GP) and completed surgery.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)33.0± 10.2
Incidence Rate of Inhibitors Against Factor VIII (FVIII) (≥0.6 BU/mL) Secondary · during the trial (2-5 weeks)

Incidence rate of inhibitors is the number of newly developed inhibitors per surgery. Development of FVIII inhibitors was measured by a validated Nijmegen modified Bethesda assay. A positive inhibitor test was defined as ≥0.6 bethesda unit. Number of participants with inhibitors at the end of trial is presented.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0
Estimated Blood Loss During Surgery Secondary · During surgery

The mean estimated blood loss following surgery is presented. Estimated blood loss (mL) was evaluated post surgery.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)322.6± 745.0
Number of Transfusions During the Post-operative Period Days 1-6 Secondary · Post-operative period, days 1-6

Number of blood product transfusions (transfusion of red blood cells) during the post-surgery period, Days 1-6 is presented.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)9
Length of Stay in the Hospital Secondary · During the trial (2-5 weeks)

Mean number of days stayed at the hospital during the trial.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)10.00± 8.9
Number of Days in Intensive Care Secondary · During the trial (2-5 weeks)

Mean number of days in the intensive care due to surgery during the trial is presented.

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)0.02± 0.14
Adverse Events Reported During the Trial Period Secondary · During the trial (2-5 weeks)

Number of adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)127
Serious Adverse Events Reported During the Trial Period Secondary · During the trial (2-5 weeks)

Number of serious adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).

GroupValue95% CI
NNC 0129-0000-1003 (Turoctocog Alfa Pegol)5

Adverse events — posted to ClinicalTrials.gov

Time frame: From the first trial related activity (day 0) after the patient has signed the informed consent until the end of trial (earliest at day 14).. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

NNC 0129-0000-1003 (Turoctocog Alfa Pegol)
Serious: 4/53 (8%)
Deaths: 0/53

Serious adverse events (5 terms)

ReactionSystemNNC 0129-0000-1003 (Turoct…
HaemorrhageVascular disorders
IschaemiaVascular disorders
Mobility decreasedMusculoskeletal and connective tissue disorders
Pancreatitis acuteGastrointestinal disorders
Tooth extractionSurgical and medical procedures
Other adverse events (10 terms — click to expand)

ReactionSystemNNC 0129-0000-1003 (Turoct…
ConstipationGastrointestinal disorders
NauseaGastrointestinal disorders
C-reactive protein increasedInvestigations
Haemoglobin decreasedInvestigations
Procedural painInjury, poisoning and procedural complications
PyrexiaGeneral disorders
Alanine aminotransferase increasedInvestigations
DiarrhoeaGastrointestinal disorders
Post procedural inflammationInjury, poisoning and procedural complications
VomitingGastrointestinal disorders

Most-reported serious reactions: Haemorrhage, Ischaemia, Mobility decreased, Pancreatitis acute, Tooth extraction.

Data from ClinicalTrials.gov NCT01489111 adverse events section.

Sponsor's own description

This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates.
    Mancuso ME, Santagostino E. · · 2017 · cited 72× · PMID 28350322 · DOI 10.3390/jcm6040039
  2. Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2.
    Giangrande P, Abdul Karim F, Nemes L, You CW, et al · · 2020 · cited 22× · PMID 32544297 · DOI 10.1111/jth.14959
  3. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials.
    Tosetto A, Neff A, Lentz SR, Santagostino E, et al · · 2020 · cited 10× · PMID 32293786 · DOI 10.1111/hae.13980
  4. An overview of the pathfinder clinical trials program: Long-term efficacy and safety of N8-GP in patients with hemophilia A.
    Matsushita T, Mangles S. · · 2020 · cited 9× · PMID 32558236 · DOI 10.1111/jth.14958
  5. Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response.
    Chowdary P, Hampton K, Jiménez-Yuste V, Young G, et al · · 2022 · cited 6× · PMID 34865209 · DOI 10.1055/s-0041-1739514
  6. Illustrative Cases from the Pathfinder Clinical Trials of Patients with Hemophilia A Treated with Turoctocog Alfa Pegol (N8-GP).
    Klamroth R, Hampton K, Saulyte Trakymienė S, Korsholm L, et al · · 2021 · cited 1× · PMID 34764641 · DOI 10.2147/ppa.s326282

Verify or expand the search:

Other trials of turoctocog alfa pegol

Trials testing the same drug.

Other recruiting trials for Congenital Bleeding Disorder

Currently open trials in the same condition.

Other Novo Nordisk A/S trials

Trials by the same sponsor.

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