A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

NCT ID: NCT01731600

Last Updated: 2020-11-23

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

68 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-02-20

Study Completion Date

2018-09-28

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Congenital Bleeding Disorder Haemophilia A

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

N8-GP

Group Type EXPERIMENTAL

turoctocog alfa pegol

Intervention Type DRUG

Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

turoctocog alfa pegol

Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

NNC 0129-0000-1003 N8-GP

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
* Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion Criteria

\- Any history of FVIII inhibitors
Minimum Eligible Age

0 Years

Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Novo Nordisk Investigational Site

Phoenix, Arizona, United States

Site Status

Novo Nordisk Investigational Site

Long Beach, California, United States

Site Status

Novo Nordisk Investigational Site

Orange, California, United States

Site Status

Novo Nordisk Investigational Site

Washington D.C., District of Columbia, United States

Site Status

Novo Nordisk Investigational Site

Orlando, Florida, United States

Site Status

Novo Nordisk Investigational Site

Tampa, Florida, United States

Site Status

Novo Nordisk Investigational Site

Boise, Idaho, United States

Site Status

Novo Nordisk Investigational Site

Iowa City, Iowa, United States

Site Status

Novo Nordisk Investigational Site

New Orleans, Louisiana, United States

Site Status

Novo Nordisk Investigational Site

Boston, Massachusetts, United States

Site Status

Novo Nordisk Investigational Site

Minneapolis, Minnesota, United States

Site Status

Novo Nordisk Investigational Site

Omaha, Nebraska, United States

Site Status

Novo Nordisk Investigational Site

New Hyde Park, New York, United States

Site Status

Novo Nordisk Investigational Site

Charlotte, North Carolina, United States

Site Status

Novo Nordisk Investigational Site

Cincinnati, Ohio, United States

Site Status

Novo Nordisk Investigational Site

Dayton, Ohio, United States

Site Status

Novo Nordisk Investigational Site

Philadelphia, Pennsylvania, United States

Site Status

Novo Nordisk Investigational Site

Philadelphia, Pennsylvania, United States

Site Status

Novo Nordisk Investigational Site

Charleston, South Carolina, United States

Site Status

Novo Nordisk Investigational Site

Nashville, Tennessee, United States

Site Status

Novo Nordisk Investigational Site

Dallas, Texas, United States

Site Status

Novo Nordisk Investigational Site

Houston, Texas, United States

Site Status

Novo Nordisk Investigational Site

Charlottesville, Virginia, United States

Site Status

Novo Nordisk Investigational Site

Spokane, Washington, United States

Site Status

Novo Nordisk Investigational Site

Rio de Janeiro, , Brazil

Site Status

Novo Nordisk Investigational Site

Toronto, Ontario, Canada

Site Status

Novo Nordisk Investigational Site

Bron, , France

Site Status

Novo Nordisk Investigational Site

Lille, , France

Site Status

Novo Nordisk Investigational Site

Paris, , France

Site Status

Novo Nordisk Investigational Site

Athens, , Greece

Site Status

Novo Nordisk Investigational Site

Thessaloniki, , Greece

Site Status

Novo Nordisk Investigational Site

Tel Litwinsky, , Israel

Site Status

Novo Nordisk Investigational Site

Vicenza, , Italy

Site Status

Novo Nordisk Investigational Site

Kitakyusyu, Fukuoka, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Vilnius, , Lithuania

Site Status

Novo Nordisk Investigational Site

Kuala Lumpur, , Malaysia

Site Status

Novo Nordisk Investigational Site

Porto, , Portugal

Site Status

Novo Nordisk Investigational Site

San Juan, , Puerto Rico

Site Status

Novo Nordisk Investigational Site

Bellinzona, , Switzerland

Site Status

Novo Nordisk Investigational Site

Lucerne, , Switzerland

Site Status

Novo Nordisk Investigational Site

Zurich, , Switzerland

Site Status

Novo Nordisk Investigational Site

Antalya, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

Bornova-IZMIR, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

İzmit, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

Samsun, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

Donetsk, , Ukraine

Site Status

Novo Nordisk Investigational Site

Lviv, , Ukraine

Site Status

Novo Nordisk Investigational Site

Leicester, , United Kingdom

Site Status

Novo Nordisk Investigational Site

London, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Oxford, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Germany United States Brazil Canada France Greece Israel Italy Japan Lithuania Malaysia Portugal Puerto Rico Switzerland Turkey (Türkiye) Ukraine United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. 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. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.

Reference Type RESULT
PMID: 32293786 (View on PubMed)

Saulyte Trakymiene S, Economou M, Kenet G, Landorph A, Shen C, Kearney S. Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):15-25. doi: 10.1111/jth.15036.

Reference Type RESULT
PMID: 32940955 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Related Links

Access external resources that provide additional context or updates about the study.

http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

U1111-1129-6009

Identifier Type: OTHER

Identifier Source: secondary_id

2012-001711-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

JapicCTI-132214

Identifier Type: REGISTRY

Identifier Source: secondary_id

NN7088-3885

Identifier Type: -

Identifier Source: org_study_id