NL-OMON54973已完成3 期
Protocol EFC16293: A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc von Willebrand Factor XTEN Fusion Protein (rFVIIIFc VWF XTEN; BIVV001) in Previously Treated Patients *12 Years of Age With Severe Hemophilia A. - XTEND-1
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 16 至 99（—）
入选标准
- •Participants are eligible to be included in the study only if all of the
- •following criteria apply:
- •01. Participant must be equal to or greater than 12 years of age inclusive, at
- •the time of signing the informed consent.
- •02. Severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as
- •documented either by central laboratory testing at Screening or in historical
- •medical records from a clinical laboratory demonstrating <1% FVIII coagulant
- •activity (FVIII:C) or a documented genotype
- •known to produce severe hemophilia A.
- •03. Previous treatment for hemophilia A (prophylaxis or on demand) with any
- •recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150
- •04. Current regimen includes one of the following: * Prophylactic treatment
- •regimen with a marketed FVIII product or prophylactic emicizumab therapy for at
- •least 6 months during the previous 12 months. Appropriate washout time needs to
- •be taken into account. * On-demand regimen with a marketed FVIII product with a
- •history of at least 12 bleeding episodes in the previous 12 months or at least
- •6 bleeding episodes in the previous 6 months prior to study enrollment. -
- •On-demand participant is accepting to move to a prophylaxis treatment regimen
- •after 26-week on-demand period.
- •05. Platelet count *100,000 cells/*L at Screening.
- •06. A participant known to be human immunodeficiency virus (HIV) antibody
- •positive, either previously documented or identified from screening
- •assessments, must have the following results prior to enrollment.
- •a. CD4 lymphocyte count >200 cells/mm³
- •b. Viral load of <400 copies/mL
- •Documented results of CD4 lymphocyte count and viral load will be accepted if
- •samples were collected within 26 weeks prior to Screening or if samples were
- •collected during Screening and evaluated by the central laboratory.
- •Participants who have previously tested negative for HIV must have a repeat
- •test by the central laboratory during Screening
- •07. Willingness and ability of the participant or surrogate (a caregiver or a
- •family member *18 years of age) to complete training in the use of the study
- •electronic Patient Diary (ePD) and to use the ePD throughout the study.
- •08. Male or Female
- •Contraceptive use by men or women should be consistent with local regulations
- •regarding the methods of contraception for those participating in clinical
- •studies. a) Male participants - No contraceptive measures required for this
- •study. b) Female participants - A female participant is eligible to participate
- •if she is not pregnant or breastfeeding, and at least one of the following
- •conditions applies: - Is not a woman of childbearing potential (WOCBP), as
- •defined in Section 10.4
- •or - Is a WOCBP and using an acceptable contraceptive method as described
- •Section 10.4 during the intervention period (at a minimum until Safety
- •Follow-up Call or Visit). The investigator should evaluate the effectiveness of
- •the contraceptive method in relationship to the first dose of study
- •intervention. and - A WOCBP must have a negative highly sensitive pregnancy
- •test before the first dose of study intervention as described in Section 10.2.
- •A serum pregnancy test should be performed at screening. For all other time
- •points, serum or urine pregnancy testing may be performed at the discretion of
排除标准
- •Participants are excluded from the study if any of the following criteria
- •Medical conditions
- •01. Any concurrent clinically significant liver disease that, in the opinion of
- •the Investigator, would make the participant unsuitable for enrollment. This
- •may include, but is not limited to cirrhosis, portal hypertension, and acute
- •02. Serious active bacterial or viral infection (other than chronic hepatitis
- •or HIV) present within 30 days of Screening.
- •03. Other known coagulation disorder(s) in addition to hemophilia A.
- •04. History of hypersensitivity or anaphylaxis associated with any FVIII
- •05. History of a positive inhibitor test defined as *0.6 BU/mL, or any value
- •greater than or equal to the lower sensitivity cut-off for laboratories with
- •cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs
- •or symptoms of decreased response to FVIII administrations. Family history of
- •inhibitors will not exclude the participant.
- •06. Positive inhibitor result, defined as *0.6 BU/mL at Screening.
- •07. Abnormal renal function, defined as serum creatinine >2.0 mg/dL taken at
- •08. Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5
- •x upper limit of normal (ULN) taken at Screening.
- •09. Serum total bilirubin >3 x ULN, taken at Screening.
- •Prior/concomitant therapy
- •10. Vaccination within 30 days of Screening
- •11. Treatment with acetylsalicylic acid (ASA) within 2 weeks prior to screening
- •12. Treatment with non-steroidal anti-inflammatory drugs (NSAIDs) above the
- •maximum dose specified in the regional prescribing information within 2 weeks
- •prior to Screening.
- •13. Systemic treatment within 12 weeks prior to Screening with chemotherapy
- •and/or other immunosuppressive drugs (except for the treatment of hepatitis C
- •virus [HCV] or HIV). Use of corticosteroids is allowed, except for systemic
- •corticosteroid treatment given daily or on alternate days for >14 days. Local,
- •topical, and/or inhaled steroid use is permitted.
- •Prior/concurrent clinical study experience
- •14. Emicizumab use within the 20 weeks prior to Screening
- •15. Previous enrolment in this study; participants who fail Screening may
- •6. Treatment with an investigational product within 30 days or 5.5 half-lives
- •prior to Screening, whichever is longer. For investigational products with a
- •pharmacodynamic effect that persists longer than the half-life, the maximal
- •pharmacodynamic effect must return to baseline prior to Screening.
- •Other exclusions
- •17. Major surgery within 8 weeks prior to Screening. Major surgery is defined
- •as any surgical procedure (elective or emergent) that usually, but not always,
- •involves general anesthesia and/or respiratory assistance, in which a major
- •body cavity is penetrated and exposed, or a substantial impairment of physical
- •or physiological functions is produced (eg, laparotomy, thoracotomy,
- •craniotomy, joint replacement, or limb amputation).
- •18. Individuals accommodated in an institution because of regulatory or legal
- •order; prisoners or participants who are legally institutionalized
- •19. Any country-related specific regulation that would prevent the participant
- •from entering the study * see Appendix 10 (Section 10.10) (country specific
- •requirements)
- •20. Partic
研究者
相似试验
已完成
3 期
A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich*s Ataxia PatientsFriedreichs ataxia100103941002839610010335NL-OMON32601Santhera Pharmaceuticals (Switzerland) Limited12
进行中（未招募）
1 期
An extension of the CL-503012 study, which involved an investigation into the safety and efficacy of Kiacta™ in preventing kidney function decline in patients with AA amyloidosis, a disease associated with long-standing inflammatory disease, which can lead to the buildup of protein (amyloid A) inside the kidney and can cause kidney failureAA AmyloidosisMedDRA version: 17.1Level: PTClassification code 10002022Term: AmyloidosisSystem Organ Class: 10021428 - Immune system disordersEUCTR2013-004150-16-PLA.T. Development Switzerland SAR24
进行中（未招募）
1 期
An extension of the CL-503012 study, which involved an investigation into the safety and efficacy of Kiacta™ in preventing kidney function decline in patients with AA amyloidosis, a disease associated with long-standing inflammatory disease, which can lead to the buildup of protein (amyloid A) inside the kidney and can cause kidney failureEUCTR2013-004150-16-LTA.T. Development Switzerland SAR70
进行中（未招募）
1 期
An extension of the CL-503012 study, which involved an investigation into the safety and efficacy of Kiacta™ in preventing kidney function decline in patients with AA amyloidosis, a disease associated with long-standing inflammatory disease, which can lead to the buildup of protein (amyloid A) inside the kidney and can cause kidney failureEUCTR2013-004150-16-GBA.T. Development Switzerland SAR24
进行中（未招募）
不适用
A Phase III Open-Label, Single-Group Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients - MICONOS EXTENSIOFriedreich's AtaxiaEUCTR2007-001646-40-NLSanthera Pharmaceuticals (Switzerland) Ltd204