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

Sponsor
Takeda (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04984889
Collaborator
(none)
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4
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Study Details

Study Description

Brief Summary

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 7days after the infusion to check side effect from the study treatment.

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

Detailed Description

A Phase 1/2 Single-Dose Study of TAK-662 in Japanese Patients with Congenital Protein C Deficiency

Study Design

Study Type:
Interventional
Anticipated Enrollment :
3 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official 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
Actual Study Start Date :
Aug 30, 2021
Actual Primary Completion Date :
Mar 29, 2022
Anticipated Study Completion Date :
May 31, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: TAK-662 80 IU/kg

TAK-662 80 international unit (IU)/kg, single intravenous infusion over 15 minutes on Day 1. In the extension part, dose of TAK-662 will be modified per participants. TAK-662 is Protein C Concentrate, which is a lyophilized, sterile concentrate of human protein C.

Drug: TAK-662
Lyophilized, sterile concentrate of human protein C
Other Names:
  • Protein C Concentrate
  • Outcome Measures

    Primary Outcome Measures

    1. Protein C activity [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    2. Terminal Phase Elimination Half-life (t1/2) for TAL-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    3. Incremental recovery (IR) for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    4. In-vivo recovery (IVR) for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    5. AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    6. AUC∞: Area Under the Plasma Concentration-time Curve from Time 0 to Infinity for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    7. Cmax: Maximum Observed Plasma Concentration for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    8. Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for TAK-662 [Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, 36 hours Post-dose]

    Secondary Outcome Measures

    1. Number of Participants with Treatment-Related Adverse Experiences (AEs) [Up to Day 7]

      An AE is defined as any untoward medical occurrence in a clinical investigation subject administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g. a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug.

    2. Extension Part: Treatment of Acute Episodes Rated by Efficacy Rating Scale [Up to Day 10 (Duration may vary depending on symptoms)]

      The treatment of acute episodes (purpura fulminans [PF] / coumarin-induced skin necrosis [CISN] / warfarin-induced skin necrosis [WISN] and/or other vascular thromboembolic events) will be rated according to the efficacy rating scale; as effective, effective with complications, or not effective, when it is determined that the next infusion will be unnecessary.

    3. Extension Part: Percentage of Participants who Experience Surgical Episodes during Short-Term Prophylaxis [Up to Day 10 (Duration may vary depending on symptoms)]

      Percentage of participants who will experience surgical episodes during short-term prophylaxis in extension part will be reported. TAK-662 will be utilized as short-term prophylaxis, that will be free of presentations of PF or thromboembolic complications.

    4. Extension Part: Number of Episodes of PF and/or Thrombotic Episodes during Long-Term Prophylaxis [Up to 1.5 years]

      Number of episodes of PF and/or thrombotic episodes during long-term prophylaxis in extension part will be reported.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    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).

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Nara Medical University Hospital Kashihara Nara Japan
    2 Chiba Children's Hospital Chiba Japan
    3 Chiba University Hospital Chiba Japan
    4 Saitama Prefectural Children's Medical Center Saitama Japan

    Sponsors and Collaborators

    • Takeda

    Investigators

    • Study Director: Study Director, Takeda

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Takeda
    ClinicalTrials.gov Identifier:
    NCT04984889
    Other Study ID Numbers:
    • TAK-662-1501
    • U1111-1267-4412
    • jRCT2031210209
    First Posted:
    Aug 2, 2021
    Last Update Posted:
    Apr 11, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Apr 11, 2022