ISRCTN14762022已完成3 期
A randomized, double-blind, placebo-controlled, phase 3, three-way crossover trial to evaluate the efficacy and safety of two dose levels of KVD900, an oral plasma kallikrein inhibitor, for on-demand treatment of angioedema attacks in adolescent and adult patients with hereditary angioedema type I or II
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 136
研究概览
简要总结
2024 Results article in https://pubmed.ncbi.nlm.nih.gov/38819658/ (added 10/06/2024)
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •Current inclusion criteria as of 26/10/2023:
- •1. Male or female patients 12 years of age and older with a weight of >30 kg
- •2. Confirmed diagnosis of HAE type I or II at any time in the medical history.
- •3. Patient has access to and ability to use conventional on-demand treatment for HAE attacks.
- •4. If a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must be on a stable dose and regimen for at least 3 months prior to the Screening Visit and be willing to remain on a stable dose and regimen for the duration of the trial.
- •5. Patient’s last dose of attenuated androgens was at least 28 days prior to randomization.
- •6. Patient:
- •6.1. Has had a least two documented HAE attacks within 3 months prior to screening or randomisation; or
- •6.2. Is a completer of the KVD824-201 trial within 3 months prior to randomization and meets all other entry criteria to enroll in KVD900-301.7. Patients must meet the contraception requirements.
- •8. Patients must be able to swallow trial tablets whole.
- •9. Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the electronic diary (eDiary).
- •10. Investigator believes that the patient is willing and able to adhere to all protocol requirements.
- •11. Patient provides signed informed consent or assent (when applicable). A parent or legally authorized representative (LAR) must also provide signed informed consent when required.
- •Previous inclusion criteria as of 04/11/2022:
- •1. Male or female patients 12 years of age and older with a weight of >30kg
- •2. Confirmed diagnosis of HAE type I or II at any time in the medical history.
- •3. Patient has access to and ability to use conventional on-demand treatment for HAE attacks.
- •4. If a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must be on a stable dose and regimen for at least 3 months prior to the Screening Visit and be willing to remain on a stable dose and regimen for the duration of the trial.
- •5. Patient’s last dose of attenuated androgens was at least 28 days prior to randomization.
- •6. Patient:
- •6.1. Has had at least two documented HAE attacks within 3 months prior to randomization; or
- •6.2. Is a completer of the KVD824-201 trial within 3 months prior to randomization and meets all other entry criteria to enroll in KVD900-301.7. Patients must meet the contraception requirements.
- •8. Patients must be able to swallow trial tablets whole.
- •9. Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the electronic diary (eDiary).
- •10. Investigator believes that the patient is willing and able to adhere to all protocol requirements.
- •11. Patient provides signed informed consent or assent (when applicable). A parent or legally authorized representative (LAR) must also provide signed informed consent when required.
- •Previous inclusion criteria:
- •1. Male or female patients 12 years of age and older with a weight of >30kg
- •2. Confirmed diagnosis of HAE type I or II at any time in the medical history.
- •3. Patient has access to and ability to use conventional on-demand treatment for HAE attacks.
- •4. If a patient is receiving long-term prophylactic treatment with one of these medicines indicated for HAE: intravenous (iv) or sc plasma-derived C1-INH, and/or lanadelumab, they must be on a stable dose and re
排除标准
- •Current exclusion criteria as of 04/11/2022:
- •1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1-inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.
- •2. A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
- •3. Use of angiotensin-converting enzyme (ACE) inhibitors after the Screening Visit or within 7 days prior to randomization.
- •4. Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
- •5. Patients who require sustained use of strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers.
- •6. Inadequate organ function, including but not limited to:
- •6.1. Alanine aminotransferase (ALT) >2x upper limit of normal (ULN)
- •6.2. Aspartate aminotransferase (AST) >2x ULN
- •6.3. Bilirubin direct >1.25x ULN
- •6.4. International normalized ratio (INR) >1.2
- •6.5. Clinically significant hepatic impairment defined as a Child-Pugh B or C
- •7. Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial.
- •8. History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.
- •9. Known hypersensitivity to KVD900 or placebo or to any of the excipients.
- •10. Prior participation in trial KVD900-201.
- •11. Participation in any gene therapy treatment or trial for HAE.
- •12. Participation in any interventional investigational clinical trial, (with the exception of KVD824-201), including an investigational COVID-19 vaccine trial, within 4 weeks of the last dosing of the investigational drug prior to screening.
- •13. Any pregnant or breastfeeding patient.
- •Previous exclusion criteria:
- •1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1-inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.
- •2. A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
- •3. Use of angiotensin-converting enzyme (ACE) inhibitors after the Screening Visit or within 7 days prior to randomization.
- •4. Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
- •5. Use of strong cytochrome P450 3A4 (CYP3A4) inhibitors and inducers during participation in the trial, starting within 5 half-lives of the Screening Visit.
- •6. Inadequate organ function, including but not limited to:
- •6.1. Alanine aminotransferase (ALT) >2x upper limit of normal (ULN)
- •6.2. Aspartate aminotransferase (AST) >2x ULN
- •6.3. Bilirubin direct >1.25x ULN
- •6.4. International normalized ratio (INR) >1.2
- •6.5. Clinically significant hepatic impairment defined as a Child-Pugh B or C
- •7. Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the
研究者
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