65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05396105 (ClinicalTrials.gov) | December 28, 2022 | 28/4/2022 | Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema | A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II) | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor [C1-INH] Deficiency;C1 Esterase Inhibitor Deficiency;C1 Esterase Inhibitor, Deficiency of;C1 Inhibitor Deficiency | Drug: PHA-022121 low dose;Drug: PHA-022121 medium dose;Drug: PHA-022121 high dose;Drug: PHA-022121 selected dose | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | N/A | All | 72 | Phase 2/Phase 3 | United States;Bulgaria;Czechia;France;Germany;Hungary;Spain |
| 2 | EUCTR2021-006906-58-HU (EUCTR) | 11/07/2022 | 13/05/2022 | An extension phase 2/3 study to test the safety of long term administration of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema | A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - RAPIDe-2 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2;Phase 3 | France;United States;Czechia;Hungary;Canada;Spain;Poland;Bulgaria;Israel;Germany;United Kingdom;Italy | ||
| 3 | NCT05047185 (ClinicalTrials.gov) | April 19, 2022 | 7/9/2021 | Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II) | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor Deficiency;C1 Inhibitor Deficiency | Drug: PHA-022121 low dose;Drug: PHA-022121 high dose;Drug: Placebo | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | 75 Years | All | 30 | Phase 2 | United States;Bulgaria;Canada;Germany;Israel;Italy;Poland;Spain;United Kingdom |
| 4 | EUCTR2021-000227-13-BG (EUCTR) | 07/03/2022 | 02/12/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | Canada;Austria;United States;Ireland;Poland;Italy;United Kingdom;Israel;Bulgaria;Germany;Spain | ||
| 5 | EUCTR2021-000227-13-PL (EUCTR) | 02/02/2022 | 18/11/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom | ||
| 6 | EUCTR2020-003445-11-BG (EUCTR) | 14/04/2021 | 02/03/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands | ||
| 7 | EUCTR2020-003445-11-NL (EUCTR) | 14/04/2021 | 01/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany | ||
| 8 | EUCTR2020-003445-11-DE (EUCTR) | 26/02/2021 | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | Spain;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands | ||
| 9 | EUCTR2020-003445-11-PL (EUCTR) | 23/02/2021 | 03/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Not Recruiting | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany | ||
| 10 | EUCTR2020-003445-11-HU (EUCTR) | 27/01/2021 | 30/11/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 11 | EUCTR2020-003445-11-FR (EUCTR) | 18/12/2020 | 22/10/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Czech Republic;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 12 | EUCTR2019-001693-28-DE (EUCTR) | 06/02/2020 | 02/09/2019 | Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older | Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with congenital C1-INH deficiency and hereditary angioedema - CONE-01 | Hereditary angioedema type I and type II MedDRA version: 20.0;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: C1 Esterase Inhibitor Human Product Code: OCTA-C1-INH Other descriptive name: C1 ESTERASE INHIBITOR (HUMAN) | Octapharma Pharmazeutika Produktionsges.m.b.H. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Czech Republic;Russian Federation;Hungary;Ukraine;Poland;Belarus;Bulgaria;Serbia;Germany | ||
| 13 | NCT03576469 (ClinicalTrials.gov) | June 13, 2018 | 15/6/2018 | A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | CVI - Common Variable Immunodeficiency | Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) | IMMUNOe Research Centers | NULL | Completed | 18 Years | N/A | All | 20 | Phase 4 | United States |
| 14 | EUCTR2020-003445-11-CZ (EUCTR) | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Belgium;Poland;Germany;Netherlands |