Skip to main content
OpenTrials
Completed

NCT Number: NCT04984889

A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency

Pharmacokinetic Part:

This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give patients in the future.

Participants will receive 1 single infusion of TAK-662.

They will stay at the clinic until 3 days after the infusion. Then, participants will return to their clinic 7 days after the infusion to check side effects from the study treatment.

Extension Part:

Participants who will complete the PK part will be given an opportunity to continue TAK-662 administration as 3 different treatment options (on-demand therapy, short-term prophylaxis, and long-term prophylaxis) in the Extension part, until the commercial protein C concentrate is available at each study site or study termination.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Congenital Protein C Deficiency

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Nara Medical University Hospital, Kashihara, Nara, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

PK Part:

  • Male and female participants with Japanese nationality.
  • A diagnosis of congenital protein C deficiency (homozygous or compound heterozygous).
  • Asymptomatic participant.
  • Oral anticoagulants allowed to be received.

Extension part:

  • Participants who participated in the PK part of this study (TAK-662-1501).
  • 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.

Exclusion criteria

PK Part:

  • Current or recurrent disease that could affect the action, or disposition of the investigational product (IP), or clinical or laboratory assessments.
  • A body weight less than 8 kg.
  • Serious liver dysfunction, judged by the investigator.
  • Any thrombosis within 2 weeks prior to administration of the IP.
  • Other investigational product than TAK-662 received within 60 days prior to the administration of the IP.
  • 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.
  • 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.
  • Known or suspected intolerance or hypersensitivity to the IP, closely-related compounds, or any of the stated ingredients.
  • Known history of alcohol or other substance abuse within the last year.
  • 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:

  • 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).

Treatment and study plan

TAK-662

Drug

Lyophilized, sterile concentrate of human protein C

Other names: Protein C Concentrate

Primary outcomes

  1. PK Part: Protein C Activity Level of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    Protein C is a vitamin K-dependent plasma protein and is an important component of the coagulation system. Protein C activity level was measured by chromogenic assays. Protein C activity level of TAK-662 was reported.

  2. PK Part: Terminal Phase Elimination Half-life (t1/2) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    t1/2 of TAK-662 was reported.

  3. PK Part: Incremental Recovery (IR) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    IR of TAK-662 was reported measured in terms of international unit per milliliter/ international unit per kilogram (IU/mL)/(IU/kg).

  4. PK Part: Percentage of In-vivo Recovery (IVR) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    IVR corrected for plasma was determined using the formula: IVR (percentage [%])= (Maximum observed plasma concentration (Cmax) [IU/mL] - Concentration (C) pre-infusion [IU/mL]) * Plasma volume pre-infusion (PV) milliliter (mL)/ Dose (international unit [IU])*100 where Cmax was the observed Cmax value before baseline correction. IVR of TAK-662 measured in terms of percentage was reported.

  5. PK Part: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    AUClast of TAK-662 was reported measured in terms of international unit*hour per milliliter (IU*h/ml).

  6. PK Part: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity (AUC0-infinity) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    AUC0-infinity of TAK-662 was reported.

  7. PK Part: Maximum Observed Plasma Concentration (Cmax) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    Cmax of TAK-662 was reported.

  8. PK Part: Time to Reach the Maximum Plasma Concentration (Tmax) of TAK-662

    Time frame: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion

    Tmax of TAK-662 was reported.

Secondary outcomes

  1. PK and Extension Parts: Number of Participants With Treatment-Related Adverse Experiences (AEs)

    Time frame: PK Part: From the start of study drug administration up to Day 7; Extension Part: From the first dose of study drug administration in the Extension Part up to 35 months

    A treatment-related AE was defined as an adverse event that followed a reasonable temporal sequence from administration of a drug (including the course after withdrawal of the drug), or for which possible involvement of the drug was not able to be ruled out, although factors other than the drug, such as underlying diseases, complications, concomitant medications and concurrent treatments, might also be responsible. Number of participants with treatment-related AEs as assessed by the Investigator were reported.

  2. Extension Part: Number of Episode Rated as Effective, Effective With Complications, or Not Effective on Efficacy Rating Scale During On-Demand Treatment

    Time frame: Extension Part: From the first dose of TAK-662 on-demand treatment in the Extension Part up to 35 months

    The treatment of episodes of PF, CISN/ WISN, and/or other vascular thromboembolic events were rated as "effective", "effective with complications", or "not effective" according to the efficacy rating scale, as judged by investigators on the basis of following criteria, Effective: stabilization and regression of skin lesions/stabilization of thrombi; Effective with complications: treatment was effective but caused an adverse drug reaction that interfered with the regimen (resulted in change of dose or frequency of dosing) or forcing discontinuation of treatment or introducing pathogenic viral infection; Not effective: all other cases.

  3. Extension Part: Percentage of Surgical Episodes During Short-Term Prophylaxis That is Free of Presentations of PF or Thromboembolic Complications

    Time frame: Extension Part: From the first dose of TAK-662 short-term prophylaxis treatment in the Extension Part up to 35 months

    Percentage of surgical episodes for which TAK-662 was utilized as short-term prophylaxis that is free of presentations of PF or thromboembolic complications was reported.

  4. Extension Part: Number of Episodes of PF and/or Thrombotic Episodes During Long-Term Prophylaxis

    Time frame: Extension Part: From the first dose of TAK-662 long-term prophylaxis treatment in the Extension Part up to 35 months

    Number of episodes of PF and/or thrombotic episodes during long-term prophylaxis was planned to be reported.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

An Open-Label, Single-Dose, Phase 1/2 Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Human Protein C (TAK-662) for the Treatment of Congenital Protein C Deficiency in Japanese Subjects Followed by an Extension Part

Important dates

Study start
2021
Primary completion
2022
Study completion
2024
First posted
Aug 2, 2021
Registry last updated
May 7, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.