E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital Protein C Deficiency |
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E.1.1.1 | Medical condition in easily understood language |
Congenital Protein C Deficiency |
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E.1.1.2 | Therapeutic area | Body processes [G] - Genetic Phenomena [G05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10051298 |
E.1.2 | Term | Protein C deficiency |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To measure the pharmacokinetic (PK) parameters of TAK-662 in asymptomatic participants with homozygous or double heterozygous congenital protein C deficiency in Japanese participants. |
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E.2.2 | Secondary objectives of the trial |
- 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). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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). |
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E.5 End points |
E.5.1 | 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. AUC∞: 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
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose |
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E.5.2 | 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 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
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E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 2 |