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 | NCT04400045 (ClinicalTrials.gov) | May 22, 2020 | 19/5/2020 | Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome | Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome | Nijmegen Breakage Syndrome | Drug: Treosulfan | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 3 Months | 21 Years | All | 10 | Phase 2 | Russian Federation |
| 2 | EUCTR2013-005508-33-IT (EUCTR) | 13/07/2015 | 21/05/2015 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 18.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 18.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 18.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 3 | NCT02349906 (ClinicalTrials.gov) | April 2015 | 26/1/2015 | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases | Primary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure Syndromes | Drug: Treosulfan;Drug: Busilvex | medac GmbH | Celerion;Venn Life Sciences;Syneos Health | Active, not recruiting | N/A | 17 Years | All | 100 | Phase 2 | Czechia;Germany;Italy;Poland;Austria;Czech Republic |
| 4 | EUCTR2013-005508-33-PL (EUCTR) | 17/11/2014 | 08/08/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| 5 | EUCTR2013-005508-33-CZ (EUCTR) | 12/11/2014 | 29/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 mg, powder for solution for infusion Product Name: Ovastat 1000 INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 mg, powder for solution for infusion Product Name: Ovastat 5000 INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;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 |
| 6 | EUCTR2013-005508-33-DE (EUCTR) | 29/09/2014 | 06/06/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| 7 | EUCTR2013-005508-33-AT (EUCTR) | 08/08/2014 | 08/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 19.1;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 19.1;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 19.1;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft fuer klinische Spezialpräparate mbH | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 8 | EUCTR2013-003257-20-GB (EUCTR) | 02/05/2014 | 12/03/2014 | TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATION | Evaluation of Treosulfan pharmacokinetics (PK) in children undergoing allogeneic haematopoietic stem cell transplantation (HSCT) - Treosulfan PK in children | Any paediatric disease with an indication to an allogeneic stem cell transplantation (inclusing leukaemia, primary immunodeficiencies, metabolic disorders and autoimmune or genetic inflammatory bowel disorders).;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 4 | United Kingdom |