Congenital Protein C DeficiencyPhase 1/2NCT04984889
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
A Phase 1/2 study testing TAK-662 in people with Congenital Protein C Deficiency, run by Takeda at 4 sites in Japan.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Protein C Deficiency
- What is being tested?
- TAK-662
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 4 sitesJapan
- Study dates
- 7 Sep 2021to 31 Oct 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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:
- 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).
Trial locations and contacts
4 sites in 1 country.
Japan4 sites
Chiba Children's Hospital
Chiba
Chiba University Hospital
Chiba
Nara Medical University Hospital
Kashihara, Nara
Saitama Prefectural Children's Medical Center
Saitama
About this study
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
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.
Read more
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.
- Conditions
- Congenital Protein C Deficiency
- Being tested
- TAK-662
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TAK-662Drug
Also called Protein C Concentrate
Lyophilized, sterile concentrate of human protein C
| Study group | Role | What they receive |
|---|---|---|
| TAK-662 80 IU/kg | Experimental | 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. Receives: Drug: TAK-662 |
What is being measured
The primary outcome is the main question the study is designed to answer.
PK Part: Protein C Activity Level of TAK-662
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.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Terminal Phase Elimination Half-life (t1/2) of TAK-662
t1/2 of TAK-662 was reported.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Incremental Recovery (IR) of TAK-662
IR of TAK-662 was reported measured in terms of international unit per milliliter/ international unit per kilogram (IU/mL)/(IU/kg).
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Percentage of In-vivo Recovery (IVR) of TAK-662
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.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) of TAK-662
AUClast of TAK-662 was reported measured in terms of international unit*hour per milliliter (IU*h/ml).
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity (AUC0-infinity) of TAK-662
AUC0-infinity of TAK-662 was reported.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Maximum Observed Plasma Concentration (Cmax) of TAK-662
Cmax of TAK-662 was reported.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
PK Part: Time to Reach the Maximum Plasma Concentration (Tmax) of TAK-662
Tmax of TAK-662 was reported.
Measured: Pre-infusion, 0.5, 1, 2, 4, 8, 12, 24, and 36 hours post-infusion
Secondary outcomes (4)
PK and Extension Parts: Number of Participants With Treatment-Related Adverse Experiences (AEs)
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.
Measured: 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
Extension Part: Number of Episode Rated as Effective, Effective With Complications, or Not Effective on Efficacy Rating Scale During On-Demand Treatment
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.
Measured: Extension Part: From the first dose of TAK-662 on-demand treatment in the Extension Part up to 35 months
Extension Part: Percentage of Surgical Episodes During Short-Term Prophylaxis That is Free of Presentations of PF or Thromboembolic Complications
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.
Measured: Extension Part: From the first dose of TAK-662 short-term prophylaxis treatment in the Extension Part up to 35 months
Extension Part: Number of Episodes of PF and/or Thrombotic Episodes During Long-Term Prophylaxis
Number of episodes of PF and/or thrombotic episodes during long-term prophylaxis was planned to be reported.
Measured: Extension Part: From the first dose of TAK-662 long-term prophylaxis treatment in the Extension Part up to 35 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2021
- Study start7 Sep 2021
- Primary completion29 Mar 2022
- Study completion31 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Protein C DeficiencyStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04984889
- Sponsor's protocol ID
- TAK-662-1501
- Other IDs
- U1111-1267-4412, jRCT2031210209
- Status last verified
- Apr 2025
- First submitted
- 29 Jul 2021
- Last updated
- 7 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be reidentified (due to the limited number of study participants/study sites).
Publications
Koh K, Nogami K, Kakuda H, Okimoto Y, Hino M, Suzuki C, Terashio S, Ueda H, Ohga S. Efficacy and Safety of Human Protein C Concentrate in Japanese Patients With Severe Congenital Protein C Deficiency. Pediatr Int. 2026 Jan-Dec;68(1):e70506. doi: 10.1111/ped.70506.
PubMed 42745610Koh K, Nogami K, Kakuda H, Okimoto Y, Hino M, Tanigawara Y, Li Z, Suzuki C, Terashio S, Ueda H, Ohga S. A Phase 1/2, Open-Label, Single-Dose, Multicenter Study to Evaluate the Pharmacokinetics and Safety of Human Plasma-Derived Protein C Concentrate in Japanese Patients with Severe Congenital Protein C Deficiency (SCPCD). J Blood Med. 2026 Mar 13;17:535025. doi: 10.2147/JBM.S535025. eCollection 2026.
PubMed 41859023
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04984889
- Acronym / sponsor ID
- TAK-662-1501
- Conditions
- Congenital Protein C Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 7 Sep 2021 (actual)
- Primary completion
- 29 Mar 2022 (actual)
- Study completion
- 31 Oct 2024 (actual)
- First posted
- 2 Aug 2021 (actual)
- Results
- Posted 5 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TAK-662 80 IU/kg | Experimental | Drug: TAK-662TAK-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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
- Participants who participated in the PK part of this study (TAK-662-1501).
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TAK-662Also: Protein C ConcentrateLyophilized, sterile concentrate of human protein C | Drug | TAK-662 80 IU/kg |
Primary outcome
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.
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.
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).
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.
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).
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.
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.
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.
4 secondary outcomes
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
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
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42745610Koh K, Nogami K, Kakuda H, Okimoto Y, Hino M, Suzuki C, Terashio S, Ueda H, Ohga S. Efficacy and Safety of Human Protein C Concentrate in Japanese Patients With Severe Congenital Protein C Deficiency. Pediatr Int. 2026 Jan-Dec;68(1):e70506. doi: 10.1111/ped.70506.
- Derived from this study: PubMed 41859023Koh K, Nogami K, Kakuda H, Okimoto Y, Hino M, Tanigawara Y, Li Z, Suzuki C, Terashio S, Ueda H, Ohga S. A Phase 1/2, Open-Label, Single-Dose, Multicenter Study to Evaluate the Pharmacokinetics and Safety of Human Plasma-Derived Protein C Concentrate in Japanese Patients with Severe Congenital Protein C Deficiency (SCPCD). J Blood Med. 2026 Mar 13;17:535025. doi: 10.2147/JBM.S535025. eCollection 2026.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be reidentified (due to the limited number of study participants/study sites).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TAK-662 in people with Congenital Protein C Deficiency, run by Takeda at 4 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04984889, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2025.