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
Showing 1 to 10 of 500 diseases
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 | NCT05754450 (ClinicalTrials.gov) | April 21, 2023 | 6/2/2023 | An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion ... | A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II) A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Su ... | Leukocyte Adhesion Deficiency | Drug: AVTX-803 | Avalo Therapeutics, Inc. | NULL | Not yet recruiting | 6 Months | 75 Years | All | 2 | Phase 3 | United States |
2 | NCT05193552 (ClinicalTrials.gov) | April 1, 2023 | 6/8/2021 | Usage of Spirometry in Managing IgG Therapy in CVID With Airway Disease | A Prospective Study of the Utility of Spirometry to Identify and Manage Immunoglobulin Replacement Dosage in Primary Antibody Deficiency in Patients With Potentially Reversible Airway Disease A Prospective Study of the Utility of Spirometry to Identify and Manage Immunoglobulin Replacement D ... | Common Variable Immunodeficiency | Drug: Hizentra | University of Alabama at Birmingham | NULL | Not yet recruiting | 21 Years | N/A | All | 22 | Phase 4 | NULL |
3 | NCT05593588 (ClinicalTrials.gov) | March 1, 2023 | 21/10/2022 | Senolytics Treatment of Interstitial Lung Disease in Common Variable Immunodeficiency | Senolytics as a Novel Treatment for Interstitial Lung Disease in Common Variable Immunodeficiency (CVID) Senolytics as a Novel Treatment for Interstitial Lung Disease in Common Variable Immunodeficiency(CV ... | Common Variable Immunodeficiency;Interstitial Lung Disease Due to Systemic Disease | Drug: Fisetin;Drug: Placebo | Avni Joshi | NULL | Not yet recruiting | 18 Years | N/A | All | 20 | Phase 2 | United States |
4 | EUCTR2020-004734-37-HU (EUCTR) | 24/01/2023 | 17/11/2022 | Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Imm ... | Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, eff ... | Primary Immunodeficiency Diseases MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] Primary ImmunodeficiencyDiseases MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: P ... | Product Name: Newnorm INN or Proposed INN: Newnorm Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Product Name: Newnorm INN or Proposed INN: Newnorm Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN ... | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 1;Phase 3 | United States;Hungary;Slovakia;Poland;Ukraine;Germany;Netherlands;Italy | ||
5 | NCT05398809 (ClinicalTrials.gov) | January 18, 2023 | 26/5/2022 | Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyend ... | A Phase 2 Open-Label Study to Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata A Phase 2 Open-Label Study to Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Pa ... | Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced);Alopecia Areata | Drug: Ruxolitinib | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 12 Years | 65 Years | All | 70 | Phase 2 | United States |
6 | NCT05600907 (ClinicalTrials.gov) | January 2, 2023 | 26/10/2022 | Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD) Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomato ... | Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD) Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granu ... | Chronic Granulomatous Disease;CGD | Biological: JSP191 | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 30 | Early Phase 1 | United States |
7 | NCT05071222 (ClinicalTrials.gov) | January 2023 | 27/8/2021 | Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Le ... | A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating the Safety and Efficacy of Gene Therapy of the Severe Combined Immunodeficiency (SCID) Caused by Mutations in the Human DCLRE1C Gene (Artemis) by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating the Safety and Effi ... | Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | Genetic: ARTEGENE drug product | Assistance Publique - Hôpitaux de Paris | NULL | Not yet recruiting | N/A | 1 Year | All | 5 | Phase 1/Phase 2 | France |
8 | 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 Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Heredi ... | 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) A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema ... | 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 Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioede ... | Drug: PHA-022121 low dose;Drug: PHA-022121 medium dose;Drug: PHA-022121 high dose;Drug: PHA-022121 selected dose Drug: PHA-022121 low dose;Drug: PHA-022121 medium dose;Drug: PHA-022121 high dose;Drug: PHA-022121 s ... | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | N/A | All | 72 | Phase 2/Phase 3 | United States;Bulgaria;Czechia;France;Germany;Hungary;Spain |
9 | JPRN-jRCT2031210309 | 20/12/2022 | 13/09/2021 | RALPID | Phase 2 study of the efficacy and safety of sirolumus in patients with primary immunodeficiency | Primary immunodeficiency PID, ALPS, APDS, CTLA4, IPEX, lymphoproliferative disease, enterocolitis, cytopenia;D000081207 Primary immunodeficiency PID, ALPS, APDS, CTLA4, IPEX, lymphoproliferative disease, enterocolitis, c ... | Administer sirolimus tablets or granules once a daily. Initial dose: 2mg (tablet), 0.049 mg/kg or 0.035 mg/kg (granule) Administer sirolimustablets or granules once a daily. Initial dose: 2mg (tablet), 0.049 mg/kg or 0.03 ... | Endo Akifumi | NULL | Recruiting | >= 2month old | Not applicable | Both | 10 | Phase 2 | Japan |
10 | EUCTR2021-001226-21-DE (EUCTR) | 01/12/2022 | 08/03/2022 | A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with Hereditary Angioedema. A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patie ... | 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 - KONFIDENT A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the E ... | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] Hereditary Angioedema Type Ior II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: ... | Product Name: KVD900 300 mg Film Coated Tablet INN or Proposed INN: sebetralstat Other descriptive name: KVD900 Product Name: KVD900300 mg Film Coated Tablet INN or Proposed INN: sebetralstat Other descriptive name ... | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 114 | Phase 3 | Portugal;United States;Slovakia;Greece;Spain;North Macedonia;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Romania;Australia;Bulgaria;Germany;Netherlands;Japan;New Zealand Portugal;United States;Slovakia;Greece;Spain;North Macedonia;Israel;United Kingdom;Italy;France;Hung ... |