Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients

An Open Non-randomised Dose Escalation Trial Investigating the Safety and Pharmacokinetics of Single Intravenous Administrations of NNC128-0000-2011 in Patients With Haemophilia A or B

This trial is conducted in Europe and Japan. The aim of this trial is to assess the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of single doses of NNC128-0000-2011, when administered i.v. (intravenously) to haemophilia patients.

Study Overview

• Status: Completed
• Conditions: Congenital Bleeding Disorder; Haemophilia A; Haemophilia B
• Intervention / Treatment: Drug: NNC 0128-0000-2011; Drug: NNC 0128-0000-2011

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany, 10249
• Japan
  • Kitakyusyu,, Japan, 807-8555
• Spain
  • Madrid, Spain, 28046

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Patients diagnosed with haemophilia A or B (with or without inhibitors and irrespective of severity) based on medical records
• Japan: A legally acceptable representative (LAR) is required for patients between 18 and 19 years
• Body weight less than or equal to 100.0 kg
• Body Mass Index (BMI) less than or equal to 30.0 kg/m^2

Exclusion Criteria:

• Known or suspected allergy to trial product(s) or related products (including rFVIIa)
• Previous participation in this trial defined as administration of trial product
• The receipt of any investigational product within 30 days prior to trial start (screening)
• Congenital or acquired coagulation disorders other than haemophilia A or B
• Receipt of Immune Tolerance Induction (ITI) within the last 30 days prior to screening
• Any surgery within 30 days prior to screening
• Planned surgery within the trial period
• Platelet count below 50,000 platelets/mcL (based on medical records within the last 1 month or laboratory results at screening)
• Prothrombin time (PT) above 4 times Upper limit of normal (ULN) or International normalised ratio (INR) greater than 1.7
• Hepatic dysfunction or severe hepatic disease as evaluated by the investigator (trial physician)
• Renal dysfunction (dialysis) and/or creatinine levels more than or equal to 20% above upper normal limit (according to medical records or laboratory results at screening)
• Advanced atherosclerotic disease (defined as known history of ischemic heart disease, ischemic stroke, etc.)
• Any disease, condition, or medication which, according to the investigator's (trial physician) judgement, could imply a potential hazard to the patient or interfere with the trial participation or trial outcome
• Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 100 mcg/kg	Drug: NNC 0128-0000-2011; Single dose of 100 mcg/kg NNC128-0000-2011 administered i.v. (intravenously); Drug: NNC 0128-0000-2011; Single dose of 200 mcg/kg NNC128-0000-2011 administered i.v. (intravenously)
Experimental: 200 mcg/kg	Drug: NNC 0128-0000-2011; Single dose of 100 mcg/kg NNC128-0000-2011 administered i.v. (intravenously); Drug: NNC 0128-0000-2011; Single dose of 200 mcg/kg NNC128-0000-2011 administered i.v. (intravenously)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Frequency of Adverse Events (AEs)	from first trial product administration until 12 weeks after last trial product administration
Frequency of serious adverse events (SAEs)	from first trial product administration until 12 weeks after last trial product administration
Frequency of MESIs (Medical Event of Special Interest)	from first trial product administration until 12 weeks after last trial product administration

Secondary Outcome Measures

Outcome Measure	Time Frame
Neutralising antibodies against FVIIa and/or N7-GP	from first trial product administration until 12 weeks after last trial product administration

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S

Publications and helpful links

Helpful Links

• Clinical Trials at Novo Nordisk

Study record dates

Study Major Dates

• Study Start: January 1, 2011
• Primary Completion (Actual): July 1, 2011
• Study Completion (Actual): July 1, 2011

Study Registration Dates

• First Submitted: February 1, 2011
• First Submitted That Met QC Criteria: February 1, 2011
• First Posted (Estimate): February 2, 2011

Study Record Updates

• Last Update Posted (Estimate): May 13, 2016
• Last Update Submitted That Met QC Criteria: May 12, 2016
• Last Verified: May 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hemostatic Disorders; Hemophilia A; Hemophilia B; Blood Coagulation Disorders
• Other Study ID Numbers: NN7128-3840; 2010-021127-28 (EudraCT Number); U1111-1118-6995 (Other Identifier: WHO); JapicCTI-111455 (Registry Identifier: JAPIC)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No