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临床试验/NCT03277313
NCT03277313已完成3 期

Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency Diseases

Baxalta now part of Shire38 个研究点 分布在 1 个国家目标入组 44 人开始时间: 2017年9月25日最近更新:
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适应症

试验速览

阶段
3 期
状态
已完成
发起方
入组人数
44
试验地点
38
主要终点
Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year
研究概览研究设计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

The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary immunodeficiency disease (PIDD).

研究设计

研究类型
Interventional
分配方式
Non Randomized
干预模型
Single Group
主要目的
Prevention
盲法
None

入排标准

年龄范围
2 Years 至 15 Years（Child）
性别
All
接受健康志愿者
否

入选标准

  • •Participant must have a documented diagnosis of a form of primary immunodeficiency (PI) involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies (IUIS) Scientific Committee 2015 (Picard et al., 2015) prior to enrollment. The diagnosis must be confirmed by the sponsor´s Medical Director prior to first treatment with IP in the study.
  • •Participant is at least two and below 16 years of age at the time of screening.
  • •Participant has been receiving a consistent dose of Immunoglobulin G (IgG), administered in compliance with the respective product information for a period of at least three months prior to screening. The average minimum pre-study dose over that interval was equivalent to 300 mg/kg BW / 4 weeks and a maximum dose equivalent to 1000 mg/kg body weight (BW) / 4 weeks.
  • •Participant has a serum trough level of IgG > 5 g/L at screening.
  • •If female of childbearing potential, participant presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  • •Participant /legally authorized representative is willing and able to comply with the requirements of the protocol.

排除标准

  • •Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/
  • •Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
  • •Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 2.5 times the upper limit of normal (ULN) for the testing laboratory
  • •Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] ≤ 500/mm^3)
  • •Participant has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site.
  • •Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following intravenous (IV) immunoglobulin, subcutaneous (SC) immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
  • •Participant has severe Immunoglobulin A (IgA) deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity.
  • •Participant has a known allergy to hyaluronidase.
  • •Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
  • •Participant has a bleeding disorder or a platelet count less than 20,000/μL, or who, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of SC therapy.
  • •Participant has severe dermatitis that would preclude adequate sites for safe product administration in the opinion of the investigator.
  • •Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • •Participant is a family member or employee of the investigator.
  • •If female, participant is pregnant or lactating at the time of enrollment.

结局指标

主要结局

Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year

时间窗: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

The rate of ASBI was defined as the mean number of ASBI per participant-year based on the United States (U.S.) Food and drugs Administration (FDA) guidance for industry to support marketing of human immune globulin intravenous (IGIV) as replacement therapy for primary humoral immunodeficiency and the European Medicines Agency (EMA) guideline on the clinical investigation of human normal immunoglobulin for SC and /or intramuscular administration. ASBI included bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Acute Serious Bacterial Infections.

次要结局

  • Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus(Study Epoch 2, Year 2: Months 6, 24, and 36)
  • Rate Represented as Mean Number of All Infections Per Participant-year(From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months))
  • Epoch 2: Minimum Concentration (Cmin)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Rate of SAEs Excluding Infections, Related and Not Related, Per Infusion(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Infusion Volume Per Site(Study Epoch 2: Up to 36 months)
  • Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses(Study Epoch 2, Year 1: Months 0, 6, and 12; Year 2: Months 18, 24 and 36)
  • Epoch 2: Duration of Infusion(Study Epoch 2: Up to 36 months)
  • HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score(Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36)
  • Treatment Preference and Satisfaction: Number of Participants Who Completed Treatment Preference Questionnaire(Study Epoch 2: Up to Month 36)
  • Percentage of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Area Under the Curve Over the Infusion Interval (AUCTau)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Epoch 2: Apparent Clearance (CL/F)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Epoch 2: Maximum Concentration (Cmax)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Epoch 2: Terminal Half-life (T 1/2)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Number of Participants With Serious Adverse Events (SAEs) Excluding Infections, Related and Not Related(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Number of Participants With All Temporally Associated TEAEs Excluding Infections(From beginning of infusion up to 72 hours post infusion)
  • Epoch 2: Number of Infusion Sites (Needle Sticks) Per Month(Study Epoch 2: Up to 36 months)
  • Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score(Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36)
  • Health Resource Utilization: Days on Antibiotics(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score(Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36)
  • Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score(Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36)
  • Health Resource Utilization: Number of Days Hospitalized Per Participant-Year(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B(Study Epoch 2, Year 2: Months 6, 24, and 36)
  • Epoch 2: Area Under the Curve Normalized for Week (AUCweek)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Rate of All TEAEs Excluding Infections, Related and Not Related, Per Infusion(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Number of Participants With Local TEAEs Excluding Infections, Related and Not Related(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Rate of Local TEAEs Excluding Infections, Related and Not Related, Per Infusion(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Number of Participants With Systemic TEAEs Excluding Infections, Related and Not Related(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Number of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections(From beginning of infusion up to 72 hours post infusion)
  • Rate of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections Per Infusion(From beginning of infusion up to 72 hours post infusion)
  • Number of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Time to Maximum Concentration (Tmax)(Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion)
  • Rate of All Temporally Associated TEAEs Excluding Infections Per Infusion(From beginning of infusion up to 72 hours post infusion)
  • Percentage of Participants With Any TEAEs Excluding Infections(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Number of Infusions Per Month(Study Epoch 2: Up to 36 months)
  • Epoch 1: Number of Weeks to Reach Final Dose Interval(Epoch 1 (up to 6 weeks))
  • Epoch 2: Percentage of Participants Who Achieved a Treatment Interval of Three or Four Weeks in Epoch 2(Study Epoch 2: Up to 36 months)
  • Epoch 2: Percentage of Participants Who Maintained a Treatment Interval of Three or Four Weeks in Epoch 2 for 12 Months(Study Epoch 2: Up to 12 months)
  • Health Resource Utilization: Days Not Able to go to School or Work, or to Perform Normal Daily Activities(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Health Resource Utilization: Number of Hospitalizations(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Number of Participants With All Treatment Emergent Adverse Events (TEAEs) Excluding Infections, Related and Not Related(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Rate of Systemic TEAEs Excluding Infections, Related and Not Related, Per Infusion(From first dose of study drug up to EOS (up to 4 years 9 months))
  • Epoch 2: Number of Infusion Sites (Needle Sticks) Per Infusion(Study Epoch 2: Up to 36 months)
  • Epoch 2: Maximum Infusion Rate Per Site(Study Epoch 2: Up to 36 months)
  • Epoch 2: Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE(Study Epoch 2: Up to 36 months)
  • Epoch 2: Percentage of Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE(Study Epoch 2: Up to 36 months)

研究者

发起方
Baxalta now part of Shire
申办方类型
Industry
责任方
Sponsor

研究点 (38)

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