65. 原発性免疫不全症候群
［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | EUCTR2016-003438-26-HU (EUCTR) | 28/08/2019 | 20/06/2019 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Hungary;Slovakia;Greece;Denmark;United Kingdom;Sweden | |||
| 2 | EUCTR2016-003438-26-GR (EUCTR) | 13/11/2017 | 11/10/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 3 | NCT03277313 (ClinicalTrials.gov) | September 25, 2017 | 7/9/2017 | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects | Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: GAMMAGARD LIQUID | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Active, not recruiting | 2 Years | 15 Years | All | 44 | Phase 3 | United States |
| 4 | NCT03116347 (ClinicalTrials.gov) | June 13, 2017 | 12/4/2017 | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD Subjects | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects With Primary Immunodeficiency Diseases | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA;Biological: KIOVIG;Biological: Cuvitru | Baxalta now part of Shire | Baxalta Innovations GmbH, now part of Shire | Active, not recruiting | 2 Years | 17 Years | All | 42 | Phase 4 | Czechia;Denmark;France;Greece;Slovakia;Sweden;United Kingdom |
| 5 | EUCTR2016-003438-26-CZ (EUCTR) | 18/05/2017 | 27/01/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 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 |
| 6 | EUCTR2016-003438-26-DK (EUCTR) | 08/05/2017 | 19/01/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 7 | EUCTR2016-003438-26-FR (EUCTR) | 18/04/2017 | 21/03/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;United Kingdom;Sweden | ||
| 8 | EUCTR2016-003438-26-SE (EUCTR) | 04/04/2017 | 08/11/2016 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Hungary;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 9 | EUCTR2016-003438-26-GB (EUCTR) | 14/02/2017 | 01/11/2016 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden | ||
| 10 | NCT03054181 (ClinicalTrials.gov) | December 22, 2016 | 13/2/2017 | Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO) | Facilitated Immunoglobulin Administration Registry and Outcomes Study | Primary Immunodeficiency;Secondary Immune Deficiency | Biological: HyQvia | GWT-TUD GmbH | NULL | Recruiting | N/A | N/A | All | 100 | France;Germany;Italy | |
| 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 |
| 11 | NCT02881437 (ClinicalTrials.gov) | November 11, 2016 | 17/8/2016 | IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQvia | Assessment of the IgG Trough Level in Subjects With Primary Immunodeficiency Switching From Standard Subcutaneous Immunoglobulin (SCIG) to Every Other Week HyQvia | Primary Immunodeficiency | Drug: IgHy10 | University Hospital, Lille | Shire | Completed | 18 Years | N/A | All | 22 | Phase 4 | France |
| 12 | EUCTR2016-001480-36-FR (EUCTR) | 11/07/2016 | 22/11/2016 | Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQvia | Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQvia - HyQvia | Primary immunodeficiencies MedDRA version: 19.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Centre Hospitalier Régional et Universitaire de Lille | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | France | |||
| 13 | NCT02593188 (ClinicalTrials.gov) | November 12, 2015 | 29/10/2015 | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global) | Primary Immunodeficiency Diseases (PID) | Biological: HYQVIA | Baxalta now part of Shire | NULL | Active, not recruiting | 16 Years | N/A | All | 264 | United States | |
| 14 | EUCTR2016-003438-26-SK (EUCTR) | 01/02/2017 | Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. | Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases | Primary Immunodeficiency Disease (PIDD) MedDRA version: 19.1;Level: PT;Classification code 10064859;Term: Primary immunodeficiency syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use Product Name: HyQvia INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN Trade Name: KIOVIG 100 mg/ml solution for infusion Product Name: KIOVIG INN or Proposed INN: Human normal immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV) Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection Product Name: Cuvitru INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Baxalta US Inc. | NULL | NA | Female: yes Male: yes | 40 | Phase 4 | France;Czech Republic;Slovakia;Denmark;United Kingdom;Sweden |