Trial Outcomes & Findings for A Study of TAK-660 in Surgical Procedures for People With Hemophilia A. (NCT NCT04941898)

NCT ID: NCT04941898

Last Updated: 2024-11-15

Results Overview

An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. Adverse drug reaction refers to AE related to administered drug.

• Recruitment status: COMPLETED
• Target enrollment: 16 participants
• Primary outcome timeframe: Up to 30 Days
• Results posted on: 2024-11-15

Participant Flow

Participants took part in the survey at 10 investigative sites in Japan, from 30 June 2021 to 15 March 2024 .

Participants with coagulation factor VIII (FVIII) deficiency who received Recombinant FVIII PEGylated (ADYNOVATE) for surgery/procedure were enrolled. Participants received ADYNOVATE Intravenous Infusion as part of a routine medical care.

Participant milestones

Measure	Recombinant FVIII PEGylated Intravenous Infusion Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Overall Study STARTED	16
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Recombinant FVIII PEGylated Intravenous Infusion Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Overall Study Protocol Violation	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Age, Continuous	41.4 Years STANDARD_DEVIATION 17.13 • n=15 Participants
Sex: Female, Male Female	1 Participants n=15 Participants
Sex: Female, Male Male	14 Participants n=15 Participants

PRIMARY outcome

Timeframe: Up to 30 Days

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to the medicinal product.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Number of Participants With Adverse Events	2 Participants

PRIMARY outcome

Timeframe: Up to 30 Days

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Number of Participants With Serious Adverse Events	1 Participants

PRIMARY outcome

Timeframe: Up to 30 Days

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. Adverse drug reaction refers to AE related to administered drug.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Number of Participants With Adverse Drug Reaction	0 Participants

PRIMARY outcome

Timeframe: Up to 30 Days

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria. Adverse drug reaction refers to AE related to administered drug.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Number of Participants With Serious Adverse Drug Reaction	0 Participants

PRIMARY outcome

Timeframe: Up to 30 Days

Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. Number of participants with adverse reactions categorized as inhibitor development, shock, or anaphylaxis was reported.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Number of Participants With Adverse Reactions Categorized as Inhibitor Development, Shock, or Anaphylaxis	0 Participants

SECONDARY outcome

Timeframe: Day 1 (date of surgery)

Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis.

The number of surgeries with each rating on hemostatic efficacy was counted and the percentage was reported. Intraoperative hemostatic efficacy was assessed by following four rating and criteria. Excellent: Intraoperative blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population (≤100%)), Good: Intraoperative blood loss was up to 50% more than expected for the type: of procedure performed in a non-hemophilic population (101-150%), Fair: Intraoperative blood loss was more than 50% of that expected for the type of procedure performed in a non-hemophilic population (>150%) None: Uncontrolled hemorrhage that was the result of inadequate therapeutic response despite proper dosing, necessitating rescue therapy with study drug or other replacement therapy.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=14 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Intraoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion Excellent	93.3 percentage in total surgeries
Intraoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion Good	6.7 percentage in total surgeries
Intraoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion Fair	0 percentage in total surgeries
Intraoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion Poor	0 percentage in total surgeries

SECONDARY outcome

Timeframe: 1 Day post-surgery

Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.

The number of surgeries with each rating on hemostatic efficacy was counted and the percentage was reported. Postoperative hemostatic efficacy 1 day after surgery was assessed by following four rating and criteria. Excellent: Postoperative hemostasis achieved with study drug was as good or better than that expected for the type of surgical procedure performed in a nonhemophilic population, Good: Postoperative hemostasis achieved with study drug was probably as good as that expected for the type of surgical procedure performed in a nonhemophilic population, Fair: Postoperative hemostasis with study drug was clearly less than optimal for the type of procedure performed but was maintained without necessitating rescue therapy with study drug or other replacement therapy, None: Participant experienced uncontrolled bleeding that was the result of inadequate therapeutic response despite proper dosing, necessitating rescue therapy with study drug or other replacement therapy.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion 1 Day After Surgery Excellent	56.3 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion 1 Day After Surgery Good	37.5 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion 1 Day After Surgery Fair	6.3 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion 1 Day After Surgery Poor	0 percentage in total surgeries

SECONDARY outcome

Timeframe: At completion of perioperative management (approximately 30 days after surgery)

Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis.

Postoperative hemostatic efficacy at completion of perioperative management was assessed by following four rating and criteria. Excellent: Postoperative hemostasis achieved after Day 1 with study drug was as good or better than that expected for the type of surgical procedure performed in a non-hemophilic population, Good: Postoperative hemostasis achieved after Day 1 with study drug was probably as good as that expected for the type of surgical procedure performed in a non-hemophilic population, Fair: Postoperative hemostasis with study drug after Day 1 was clearly less than optimal for the type of procedure performed but was maintained without necessitating rescue therapy with study drug or other replacement therapy, None: Participant experienced uncontrolled bleeding after Day 1 that was the result of inadequate therapeutic response despite proper dosing, necessitating rescue therapy with study drug or other replacement therapy.

Outcome measures

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 Participants Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion at Completion of Perioperative Management Excellent	50.0 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion at Completion of Perioperative Management Good	43.8 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion at Completion of Perioperative Management Fair	6.3 percentage in total surgeries
Postoperative Hemostatic Efficacy of Recombinant FVIII PEGylated Intravenous Infusion at Completion of Perioperative Management Poor	0 percentage in total surgeries

Adverse Events

Recombinant FVIII PEGylated Intravenous Infusion

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 participants at risk Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Gastrointestinal disorders Melaena	6.7% 1/15 • Up to 30 days At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Other adverse events

Measure	Recombinant FVIII PEGylated Intravenous Infusion n=15 participants at risk Participants with coagulation factor VIII (FVIII) deficiency received Recombinant FVIII PEGylated (ADYNOVATE) Intravenous Infusion for surgery/procedure as part of a routine medical care.
Psychiatric disorders Insomnia	6.7% 1/15 • Up to 30 days At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Constipation	6.7% 1/15 • Up to 30 days At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Injury, poisoning and procedural complications Procedural pain	6.7% 1/15 • Up to 30 days At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Additional Information

Medical Director

Takeda

Phone: +1-877-825-3327

Email: trialdisclosures@takeda.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place