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临床试验/2023-506831-13-00
2023-506831-13-00招募中3 期

Efficacy and Safety of Concizumab prophylaxis in patients with haemophilia A or B without inhibitors

Novo Nordisk A/S16 个研究点 分布在 11 个国家目标入组 41 人开始时间: 2024年2月7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
41
试验地点
16
主要终点
For haemophilia A patients without inhibitors: the number of treated spontaneous and traumatic bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
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 compare the effect of concizumab prophylaxis to no prophylaxis (on demand treatment with factor) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia A without inhibitors. To compare the effect of concizumab prophylaxis to no prophylaxis (on demand treatment with factor) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia B without inhibitors.

入排标准

年龄范围
0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
性别
Male
接受健康志愿者
否

入选标准

  • •Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  • •Male aged ≥12 years at the time of signing informed consent
  • •Body weight >25 kg at screening
  • •Congenital severe haemophilia A (FVIII < 1%) or moderate/severe B (FIX ≤ 2%).
  • •Documented treatment with coagulation factor containing product in the last 24 weeks (not applicable for NN7415-4255 (explorer5) patients enrolled prior to the treatment pause).

排除标准

  • •Known or suspected hypersensitivity to any constituent of the trial product or related products
  • •A known systemic inflammatory condition requiring systemic treatment at screening
  • •Treatment with emicizumab within 180 days before screening
  • •Presence of confirmed inhibitor ≥0.6 BU at screening
  • •Known history of inhibitors ≥0.6 BU in the last 5 years according to the medical records.
  • •Any disorder, except for conditions associated with haemophilia, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol
  • •Previous participation in this trial. Participation is defined as signed informed consent. However, this is not applicable for patients who were screen failed at Sponsor’s decision due to the treatment pause
  • •Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer (not applicable for NN7415-4255 patients enrolled prior to the treatment pause).
  • •Platelets ≤100x109/L at screening
  • •Fibrinogen below laboratory lower normal limit at screening
  • •Hepatic dysfunction defined as AST and/or ALT >3 times the upper limit combined with total bilirubin > 1,5 times the upper limit at screening
  • •Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) ≤30 ml/min/1.73 m2 for serum creatinine measured at screening
  • •Known inherited or acquired coagulation disorder other than congenital haemophilia
  • •History of thromboembolic disease. Current clinical signs of, or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events

结局指标

主要结局

For haemophilia A patients without inhibitors: the number of treated spontaneous and traumatic bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)

For haemophilia A patients without inhibitors: the number of treated spontaneous and traumatic bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)

For haemophilia B patients without inhibitors: the number of treated spontaneous and traumatic bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)

For haemophilia B patients without inhibitors: the number of treated spontaneous and traumatic bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)

次要结局

  • For haemophilia A patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes Arm 4 patients who have been on stable PPX at least 24 weeks in study 4322 For previous PPX (study 4322): From the point in time where PPX is stable and up until the end of study. For concizumab PPX (trial 4307): From the point in time where the concizumab maintenance dose is confirmed, increased or decreased and up until the confirmatory analyses cut-off (at least 24 weeks).
  • For haemophilia B patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes Arm 4 patients who have been on stable PPX at least 24 weeks in study 4322 For previous PPX (study 4322): From the point in time where PPX is stable and up until the end of study. For concizumab PPX (trial 4307): From the point in time where the concizumab maintenance dose is confirmed, increased or decreased and up until the confirmatory analyses cut-off (at least 24 weeks).
  • For haemophilia A patients without inhibitors: Number of treated spontaneous bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • For haemophilia B patients without inhibitors: Number of treated spontaneous bleeding episodes On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds On demand (arm 1) From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the confirmatory analyses cut-off (at least 32 weeks)
  • Number of thromboembolic events On demand (arm 1 main part) From randomisation to on demand treatment until start of concizumab treatment Concizumab (arms 2-4) Before the pause: From start of concizumab treatment up until 7 weeks after the treatment was paused and After the pause: From start of concizumab treatment up until the confirmatory analyses cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of concizumab treatment up until the confirmatory analysis cut-off
  • Number of thromboembolic events Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 384 weeks)
  • Number of hypersensitivity type reactions On demand (arm 1 main part) From randomisation to on demand treatment up until start of concizumab treatment Concizumab (arms 2-4) Before the pause: From start of concizumab treatment up until 7 weeks after the treatment was paused and After the pause: From start of concizumab treatment up until the confirmatory analyses cut-off (at least 32 weeks) Concizumab (arm 1 extension) From start of concizumab treatment up until the confirmatory analysis cut-off
  • Number of hypersensitivity type reactions Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 384 weeks)
  • Number of injection site reactions On demand (arm 1 main part) From randomisation to on demand treatment until start of concizumab treatment Concizumab (arms 2-4) Before the pause: From start of concizumab treatment up until 7 weeks after the treatment was paused and After the pause: From start of concizumab treatment up until the confirmatory analyses cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of concizumab treatment up until the confirmatory analysis cut-off
  • Number of injection site reactions Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 384 weeks)
  • Number of patients with antibodies to concizumab Concizumab (arms 2-4) Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment (week 0) up until the confirmatory analyses cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of concizumab treatment (visit 9a) up until the confirmatory analysis cut-off
  • Number of patients with antibodies to concizumab Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 384 weeks)
  • Pre-dose (trough) concizumab plasma concentration (Ctrough) prior to the concizumab administration at week 24 (after restart)
  • Pre-dose thrombin peak prior to the concizumab administration at week 24 (after restart)
  • Pre-dose free TFPI concentration prior to the concizumab administration at week 24 (after restart)
  • Maximum concizumab plasma concentration (Cmax) from 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)
  • Area under the concizumab plasma concentration-time curve (AUC) from 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)

研究者

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

EU Submission Hub

Scientific

Novo Nordisk A/S

研究点 (16)

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