Congenital Protein C Deficiency MedDRA version: 20.1 Level: PT Classification code 10051298 Term: Protein C deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: CEPROTIN
Product Name: Ceprotin
Product Code: TAK-662
Pharmaceutical Form: Lyophilisate and solvent for solution for injection
INN or Proposed INN: Human Protein C
Current Sponsor code: TA
Sponsors
Takeda
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: PK Part: 1. Male and female participants with Japanese nationality. 2. A diagnosis of congenital protein C deficiency (homozygous or compound heterozygous). 3. Asymptomatic participant. 4. Oral anticoagulants allowed to be received. Extension part: 1. Participants who participated in the PK part of this study (TAK-662-1501). 2. Participant who are; a. Diagnosed with PF, CISN/WISN, and/or other acute thromboembolic episode for on-demand treatment only; b. Requiring treatment with TAK-662 for short-term prophylaxis for surgical procedures; c. Requiring treatment with TAK-662 for long-term prophylaxis. Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 2 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: PK Part: 1. Current or recurrent disease that could affect the action, or disposition of the investigational product (IP), or clinical or laboratory assessments. 2. A body weight less than 8 kg. 3. Serious liver dysfunction, judged by the investigator. 4. Any thrombosis within 2 weeks prior to administration of the IP. 5. Other investigational product than TAK-662 received within 60 days prior to the administration of the IP. 6. Current or relevant history of physical or psychiatric illness, or any medical disorder that may require treatment or make the participant unlikely to fully complete the study, or any condition that presents undue risk from the IP or procedures. 7. Current use of any medication (including over-the-counter, herbal, or homeopathic preparations) that could affect (improve or worsen) the condition being studied, or could affect the action or disposition of the IP, or clinical or laboratory assessment. 8. Known or suspected intolerance or hypersensitivity to the IP, closely-related compounds, or any of the stated ingredients. 9. Known history of alcohol or other substance abuse within the last year. 10. Within 30 days prior to the first dose of IP, a participant has been enrolled in a clinical study (including vaccine studies) that, in the investigator's opinion, may impact this sponsored study. Extension Part: 1. New serious medical conditions which could affect participant's safety or treatment were observed during participation in the PK part of this study (TAK-662-1501).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To measure the pharmacokinetic (PK) parameters of TAK-662 in asymptomatic participants with homozygous or double heterozygous congenital protein C deficiency in Japanese participants.;Secondary Objective: - To assess safety profile of TAK-662. - To assess efficacy of TAK-662 in following: a. for on-demand treatment such as purpura fulminans (PF), coumarin-induced skin necrosis/warfarin-induced skin necrosis (CISN/WISN), and other vascular thromboembolic events; b. for short-term thromboembolic prophylaxis during surgical procedures; and c. for long-term prophylactic treatment of acute thrombotic episodes (Extension part).;Primary end point(s): 1. Protein C activity 2. Terminal Phase Elimination Half-life (t1/2) for TAK-662 3. Incremental recovery (IR) for TAK-662 4. In-vivo recovery (IVR) for TAK-662 5. AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for TAK-662 6. AUC8: Area Under the Plasma Concentration-time Curve from Time 0 to Infinity for TAK-662 7. Cmax: Maximum Observed Plasma Concentration for TAK-662 8. Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for TAK-662 ;Timepoint(s) of evaluation of this end point: Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Number of Participants with Treatment-Related Adverse Experiences (AEs) 2. Extension Part: Treatment of Acute Episodes Rated by Efficacy Rating Scale 3. Extension Part: Percentage of Participants who Experience Surgical Episodes during Short-Term Prophylaxis 4. Extension Part: Number of Episodes of PF and/or Thrombotic Episodes during Long-Term Prophylaxis;Timepoint(s) of evaluation of this end point: Up to 1.5 years | — |
Countries
Japan
Contacts
Public ContactStudy Director
Takeda
Outcome results
None listed