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临床试验/2023-509769-18-00
2023-509769-18-00招募中3 期

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease

Baxalta Innovations GmbH11 个研究点 分布在 5 个国家目标入组 8 人开始时间: 2024年5月30日最近更新:
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试验速览

阶段
3 期
状态
招募中
入组人数
8
试验地点
11
主要终点
The primary outcome measure is hemostatic efficacy, defined as the number of pediatric subjects with treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale). Bleeding episode treatment success is defined as a mean efficacy rating score of <2.5.
研究概览入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

To evaluate the hemostatic efficacy and safety of vonicog alfa, with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric subjects (<18 years of age) diagnosed with severe, hereditary VWD.

入排标准

年龄范围
0 years 至 17 years（0-17 Years）
接受健康志愿者
是

入选标准

  • •Diagnosis of severe VWD (defined as VWF:RCo <20%): a. Type 1 (VWF:RCo <20 IU/dL); or b. Type 2A (VWF:RCo <20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (FVIII:C <10% and historically documented genetics), Type 2M; or c. Type 3 (VWF:Ag ≤3 IU/dL).
  • •Age 0 to <18 years at the time of screening.
  • •The subject has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
  • •If female of childbearing potential, subject presents with a negative serum pregnancy test.
  • •If applicable, subject agrees to employ adequate birth control measures for the duration of the study.
  • •Subject and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a prescreening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the subject's compliance with the study requirements.
  • •Additional inclusion criteria for previously treated subjects as well as for subjects undergoing surgery:
  • •Unable to tolerate, inadequately responsive to not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of subjects who are not good candidates for DDAVP include subjects with type 2B or type 3 VWD.
  • •The subject has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (ie, treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.
  • •Additional inclusion criterion for previously untreated subjects: The subject has not received prior VWF coagulation factor replacement therapy.

排除标准

  • •Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio >1.4)
  • •Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C.
  • •Diagnosis of renal disease, with a serum creatinine level ≥2.5 mg/dL.
  • •Immunomodulatory drug treatment other than anti-retroviral chemotherapy (eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day (excluding topical treatment [eg, ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate).
  • •If female, subject is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained.
  • •Subject has participated in another clinical study involving an IP, other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study.
  • •Subject's legal representative is a family member or employee of the Investigator.
  • •History or presence of a VWF inhibitor at Screening.
  • •History or presence of a FVIII inhibitor with a titer ≥0.4 Bethesda units (BU) (by Nijmegen assay) or ≥0.6 BU (by Bethesda assay.)
  • •Documented history of a VWF:RCo half-life <6 hours.
  • •Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins.
  • •Medical history of immunological disorders, excluding seasonal allergic rhinitis/ conjunctivitis/ asthma, food allergies, or animal allergies
  • •Medical history of a thromboembolic event.
  • •Human immunodeficiency virus positive, with an absolute CD4 count <200/mm
  • •In the judgment of the Investigator, the subject has another clinically significant concomitant disease (eg, uncontrolled hypertension, cancer) that may pose additional risks for the subject.

结局指标

主要结局

The primary outcome measure is hemostatic efficacy, defined as the number of pediatric subjects with treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale). Bleeding episode treatment success is defined as a mean efficacy rating score of <2.5.

The primary outcome measure is hemostatic efficacy, defined as the number of pediatric subjects with treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale). Bleeding episode treatment success is defined as a mean efficacy rating score of <2.5.

次要结局

  • Efficacy: 1. Number of treated nonsurgical bleeding episodes with an efficacy rating of 'excellent' or 'good'.
  • 3. Area under the plasma concentration/time curve from 0 to 96 hours post-infusion (AUC0-96h) for FVIII activity. Point estimates per age cohort will be presented.
  • 2. Number of infusions, vonicog alfa units, and ADVATE units (if needed), per bleeding episode.
  • 3. For elective or emergency surgery: an overall assessment of hemostatic efficacy 24 hours after the last perioperative infusion of vonicog alfa, or on Day 14, whichever is earlier, assessed by the Investigator (hematologist) on a 4-point scale.
  • Safety: 1. Incidence and severity of adverse events (AEs) by system organ class (SOC) and preferred term.
  • 2. Incidence of thromboembolic events.
  • 3. Incidence of severe hypersensitivity reactions.
  • 4. Development of neutralizing antibodies to VWF and Factor VIII (FVIII).
  • 5. Development of total binding antibodies to VWF.
  • 6. Development of antibodies to CHO proteins, murine IgG, and rFurin.
  • PK/PD: 1. Area under the plasma concentration/time curve from 0 to 96 hours post-infusion (AUC0-96h), area under the plasma concentration/time curve from time 0 to infinity (AUC0-∞),
  • Mean residence time (MRT), maximal plasma concentration (Cmax), time to maximal plasma concentration (Tmax), clearance (CL), incremental recovery (IR), in-vivo recovery (IVR), elimination phase half-life (T1/2), and volume of distribution at steady state (Vss) for VWF:RCo., VWF:Ag and VWF:CB using non-compartmental analysis (NCA) methodology.
  • 2. Area under the plasma concentration/time curve from 0 to 96 hours post-infusion (AUC0-96h) for VWF:Ag and VWF:CB. Point estimates per age cohort will be presented.

研究者

申办方类型
Pharmaceutical company
责任方
Principal Investigator
主要研究者

Jingmei Zhang

Scientific

Baxalta Innovations GmbH

研究点 (11)

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