ADYNOVATE Drug Use-Results Survey

NCT03169972 | Completed Results

• 1 other identifier: 261601
• Study Type: observational
• Target: 135
• Locations: 1 country
• Sites: 52

Brief Summary

The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients:

1. 1.Unexpected adverse drug reactions
2. 2.Occurrence of adverse drug reactions in the actual clinical use
3. 3.Factors that may affect safety and efficacy
4. 4.Occurrence of Factor VIII inhibitor development in patients with coagulation factor VIII deficiency (hereinafter hemophilia A)
5. 5.Safety and efficacy for hemophilia A patients who received routine prophylactic therapy and on-demand therapy

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (10)

• Number of Participants Who Discontinued the Use of Study Drug

  Number of previously treated patients (PTPs) and previously untreated patients (PUPs) who discontinued the use of ADYNOVATE were reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Annual Bleed Rate (ABR) of Spontaneous Bleeding Episodes on a Prophylaxis Regimen

  Annual bleed rate (ABR) of spontaneous bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen

  Annual bleed rate (ABR) of breakthrough bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Duration of Treatment of Study Drug on a Prophylaxis Regimen

  Duration of treatment of study drug on a prophylaxis regimen was reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs.

• Duration of Treatment of Study Drug an On-Demand Regimen

  Duration of treatment of study drug on an on-demand regimen was reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Dose Per Administration of Study Drug on a Prophylaxis Regimen

  Dose per administration of study drug on a prophylaxis regimen was reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Dose Per Administration of Study Drug an On-Demand Regimen

  Dose per administration of study drug on an on-demand regimen was reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen

  Number of doses per a bleeding episode of study drug on an on-demand regimen was reported.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen

  Percentage of each category of hemostatic effectiveness for treatment of breakthrough bleeding episodes in a prophylaxis regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Hemostatic Effectiveness of Study Drug on an On-Demand Regimen

  Percentage of each category of hemostatic effectiveness for an on-demand regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

Secondary Outcomes (2)

• Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

• Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE)

  Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs

Study Arms (2)

Previously treated patients (PTPs)

PTPs: patients who had 4 or more days to other Factor VIII (FVIII) products

Biological: ADYNOVATE

Previously untreated patients (PUPs)

PUPs: patients who had 3 or less previous exposure days to other products

Biological: ADYNOVATE

Interventions

ADYNOVATE BIOLOGICAL

Antihemophilic Factor (Recombinant), PEGylated

Also known as: BAX 855, BAX855, Recombinant Factor VIII (FVIII) PEGylated

Previously treated patients (PTPs); Previously untreated patients (PUPs)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia A (congenital blood coagulation factor VIII deficiency) who receive ADYNOVATE

You may qualify if:

• Hemophilia A patients who receive ADYNOVATE, including previously treated patients with Factor VIII deficiency (PTPs), and previously untreated patients with Factor VIII deficiency (PUPs) who are treated with ADYNOVATE.

You may not qualify if:

• Patients not administered ADYNOVATE.

Sponsors & Collaborators

• Takeda: lead

Study Sites (52)

Nagoya City, Japan

Nagoya, Aichi-ken, 466-8560, Japan

Location

Hirosaki City

Hirosaki, Aomori, 036-8004, Japan

Location

Chiba-City, Japan

Chiba, Chiba, 260-8677, Japan

Location

Matsudo City, Japan

Matsudo, Chiba, 271-8511, Japan

Location

Narita City

Narita, Chiba, 286-8523, Japan

Location

Matsuyama City, Japan

Matsuyama, Ehime, 790-8524, Japan

Location

Toon City

Tōon, Ehime, 791-0295, Japan

Location

Fukuoka-City, Japan

Fukuoka, Fukuoka, 812-8582, Japan

Location

Kitakyusyu City, Japan

Kitakyushu, Fukuoka, 805-0050, Japan

Location

Kitakyusyu City, Japan

Kitakyushu, Fukuoka, 807-8556, Japan

Location

Koriyama City

Kōriyama, Fukushima, 963-8585, Japan

Location

Sukagawa City

Sukagawa, Fukushima, 962-8507, Japan

Location

Ogaki City, Japan

Ōgaki, Gifu, 503-8502, Japan

Location

Maebashi City

Maebashi, Gunma, 371-8511, Japan

Location

Hiroshima City, Japan

Hiroshima, Hiroshima, 734-8551, Japan

Location

Kudou-Gun, Japan

Kudou-Gun, Hokkaido, 049-4501, Japan

Location

Sapporo City

Sapporo, Hokkaido, 060-8648, Japan

Location

Kobe City

Kobe, Hyōgo, 650-0047, Japan

Location

Kobe City

Kobe, Hyōgo, 651-2273, Japan

Location

Nishinomiya City, Japan

Nishinomiya, Hyōgo, 633-8501, Japan

Location

Morioka City, Japan

Morioka, Iwate, 020-8560, Japan

Location

Zentuji City, Japan

Zentuji City, Kagawa-ken, 765-8501, Japan

Location

Kagoshima City, Japan

Kagoshima, Kagoshima-ken, 890-0046, Japan

Location

Kawasaki City, Japan

Kawasaki, Kanagawa, 216-8511, Japan

Location

Yokohama City, Japan

Yokohama, Kanagawa, 232-8555, Japan

Location

Yokohama City, Japan

Yokohama, Kanagawa, 241-0811, Japan

Location

Koti City, Japan

Kochi, Koti Prefecture, 781-8555, Japan

Location

Minamata City, Japan

Minamata, Kumamoto, 867-0041, Japan

Location

Kyoto City

Kyoto, Kyoto, 605-0981, Japan

Location

Tsu City, Japan

Tsu, Mie-ken, 514-8507, Japan

Location

Sendai City

Sendai, Miyagi, 983-8520, Japan

Location

Tome City, Japan

Tome, Miyagi, 987-0511, Japan

Location

Nichinan City

Nichinan, Miyazaki, 887-0013, Japan

Location

Matsumoto City

Matsumoto, Nagano, 390-8621, Japan

Location

Nagano City, Japan

Nagano, Nagano, 380-0928, Japan

Location

Jyoetsu City

Jyoetsu City, Niigata, 943-0147, Japan

Location

Kashiwazaki City

Kashiwazaki, Niigata, 945-0035, Japan

Location

Kurasiki City, Japan

Kurashiki, Okayama-ken, 701-0192, Japan

Location

Okayama City, Japan

Okayama, Okayama-ken, 700-8558, Japan

Location

Higashiosaka City

Higashiosaka, Osaka, 578-8588, Japan

Location

Hirakata City

Hirakata, Osaka, 573-1191, Japan

Location

Nishi-ku

Nishi-ku, Osaka, 593-8304, Japan

Location

Osaka City, Japan

Osaka, Osaka, 540-0006, Japan

Location

Osaka-City, Japan

Osaka, Osaka, 554-0012, Japan

Location

Koshigaya City, Japan

Koshigaya, Saitama, 343-8555, Japan

Location

Saitama-City, Japan

Saitama, Saitama, 330-8777, Japan

Location

Tokushima City, Japan

Tokushima, Tokushima, 770-8503, Japan

Location

Shinjuku-Ku, Japan

Shinjuku-Ku, Tokyo, 160-0023, Japan

Location

Suginami-ku, Japan

Suginami-ku, Tokyo, 167-0035, Japan

Location

Setagaya-ku, Japan

Setagaya-ku, Tokyo Metropolitan, 157-8535, Japan

Location

Sakata City, Japan

Sakata, Yamagata, 998-8501, Japan

Location

Shunan City, Japan

Shunan City, Yamagata, 745-8522, Japan

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Hemophilia A

Interventions

BAX 855; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 25, 2017
• First Posted: May 30, 2017
• Study Start: February 1, 2017
• Primary Completion: September 15, 2023
• Study Completion: September 15, 2023
• Last Updated: February 4, 2025
• Results First Posted: February 4, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

JP (52)