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Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

2020년 12월 21일 업데이트: Octapharma

Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A

The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

57

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

  • 러시아 연방
      • Barnaul, 러시아 연방
        • Barnaul Branch of RAMS hematology center
      • Moscow, 러시아 연방
        • Federal Scientific Hematology Center
  • 루마니아
      • Bucharest, 루마니아
        • Centrul Medical Unirea -Policlinica Enescu
  • 불가리아
      • Sofia, 불가리아
        • Specialized Hospital for Active Treatment "Joan Pavel"
  • 폴란드
      • Krakow, 폴란드
        • Krakowskie Centrum Medyczne
      • Rzeszow, 폴란드
        • Korczowski Bartosz Gabinet Lekarski
  • 헝가리
      • Budapest, 헝가리
        • National Haemophilia Centre

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

12년 이상 (어린이, 성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

남성

설명

Inclusion Criteria:

  1. Severe hemophilia A (<1% FVIII:C) according to medical history
  2. Male patients aged ≥12 years
  3. Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
  4. Immunocompetence (CD4+ count >200/µL)
  5. Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
  6. Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted

Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).

Exclusion Criteria:

  1. Any coagulation disorders other than hemophilia A
  2. History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
  3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
  4. Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
  5. Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: All patients
All patients will receive Wilate for prophylactic treatment
의약품: Wilate
다른 이름들:
  • 폰 빌레브란트 인자 / 인자 VIII(혈장 유래)

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Total Annualized Bleeding Rate (TABR)
기간: 6 months
The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
6 months

2차 결과 측정

결과 측정
측정값 설명
기간
Spontaneous Annualized Bleeding Rate (SABR)
기간: 6 months
The number of spontaneous bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
6 months
Efficacy of Wilate in the Treatment of Breakthrough BEs
기간: 6 months
The proportion of BEs successfully treated with Wilate were documented by the patient (together with the investigator in case of on-site treatments) in the patient diary for all BEs according to a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none', where 'excellent' was defined as "Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection" (best outcome) and 'none' was defined as "No improvement within 12 hours, or worsening of symptoms, requiring more than two injections for complete resolution" (worst outcome). All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated.'
6 months
Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis
기간: 6 months
The average consumption of Wilate per month of study (IU/kg) for all patients receiving prophylaxis.
6 months
Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C
기간: Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The value of the AUCnorm of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C
기간: Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The in vivo half-life of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C
기간: Initial PK assessment (Day -1) and 6 months
PK assessments of FVIII:C were conducted using the one-stage (OS) assay. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Initial PK assessment (Day -1) and 6 months
Incremental in Vivo Recovery (IVR) of Wilate Over Time
기간: Baseline, 3 and 6 months
The rise in FVIII activity in IU/dl per unit dose administered in IU/kg was determined from all patients at baseline, 3 and 6 months, using the OS assay.
Baseline, 3 and 6 months
Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
기간: 6 months
Analysis of variance (ANOVA) was used in an exploratory sense to assess an association between ABO blood type and the FVIII:C half-life of Wilate. This was analyzed by calculating the mean square in a one-stage assay.
6 months
Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
기간: 6 months
ANOVA was used in an exploratory sense to assess an association between VWF:Ag with the FVIII:C half-life of Wilate. This was analyzed by calculating the mean square in a one-stage assay.
6 months
Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study
기간: 6 months
At each (scheduled or unscheduled) study visit, AEs were documented by the investigator throughout the study.
6 months
Immunogenicity of Wilate by Testing for FVIII Inhibitors
기간: 6 months
FVIII inhibitor activity was determined at each study visit before the injection of Wilate using the modified Bethesda assay (Nijmegen modification).
6 months
Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
기간: 6 months
Virus safety was evaluated by taking a plasma sample for parvovirus B19 antibody testing before the first injection of Wilate. All patients negative at screening were tested again at the study completion visit. The number with Parvovirus B19 seroconversions between BL and end of study was recorded.
6 months

기타 결과 측정

결과 측정
측정값 설명
기간
Efficacy of Wilate in Surgical Prophylaxis
기간: 6 months
Hemostatic efficacy was assessed at the end of surgery by the surgeon and at end of the postoperative period by the hematologist, using a 4-point hemostatic efficacy scale including the four items: 'excellent' (best possible outcome), 'good', 'moderate' and 'none' (worst outcome). Overall efficacy was assessed by the investigator, taking both the intra and postoperative assessments into account, and using the 'excellent,' 'good,' moderate,' and 'none' scale.
6 months

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Octapharma

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2016년 12월 1일

기본 완료 (실제)

2018년 3월 29일

연구 완료 (실제)

2018년 3월 29일

연구 등록 날짜

최초 제출

2016년 10월 27일

QC 기준을 충족하는 최초 제출

2016년 11월 1일

처음 게시됨 (추정)

2016년 11월 3일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2021년 1월 19일

QC 기준을 충족하는 마지막 업데이트 제출

2020년 12월 21일

마지막으로 확인됨

2020년 12월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • WIL-27

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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