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 | NCT01685827 (ClinicalTrials.gov) | October 2012 | 12/9/2012 | Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2 | Efficacy and Safety of Fexinidazole Compared to Nifurtimox-Eflornithine Combination Therapy (NECT) in Patients With Late-stage Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: Pivotal, Non-inferiority, Multicentre, Randomised, Open-label Study | Human African Trypanosomiasis (HAT);Sleeping Sickness | Drug: Fexinidazole;Drug: Nifurtimox;Drug: Eflornithine | Drugs for Neglected Diseases | NULL | Completed | 15 Years | N/A | All | 394 | Phase 2;Phase 3 | Central African Republic;Congo, The Democratic Republic of the;Congo |
| 2 | NCT00906880 (ClinicalTrials.gov) | April 2009 | 19/5/2009 | Clinical Study to Assess the Tolerability, Feasibility and Effectiveness of Nifurtimox and Eflornithine (NECT) for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Phase | Clinical Study to Assess the Clinical Tolerability, Feasibility and Effectiveness Under Field Conditions of the Combination of Nifurtimox and Eflornithine (NECT) for the Treatment of T.b.Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Stage | Human African Trypanosomiasis | Drug: Nifurtimox-Eflronithine Combination Treatment (NECT) | Drugs for Neglected Diseases | Ministry of Public Health, Democratic Republic of the Congo;Swiss Tropical & Public Health Institute | Completed | N/A | N/A | Both | 630 | Phase 4 | Congo |
| 3 | NCT00146627 (ClinicalTrials.gov) | September 2004 | 6/9/2005 | Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis | Clinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic Phase | Trypanosomiasis, African | Drug: Eflornithine;Drug: Nifurtimox | Drugs for Neglected Diseases | Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization;Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization | Completed | 15 Years | 70 Years | Both | 280 | Phase 3 | Congo, The Democratic Republic of the;Congo;The Democratic Republic of the Congo;Uganda |
| 4 | NCT00489658 (ClinicalTrials.gov) | October 2002 | 20/6/2007 | Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, Uganda | Efficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, Uganda | Trypanosomiasis, African | Drug: Eflornithine plus Nifurtimox combination therapy | Epicentre | Medecins Sans Frontieres | Terminated | N/A | N/A | Both | 31 | Phase 2;Phase 3 | Uganda |
| 5 | NCT00330148 (ClinicalTrials.gov) | March 2001 | 24/5/2006 | Randomized Clinical Trial of Three Drug Combinations for Late-Stage Gambiense Human African Trypanosomiasis | Clinical Trial Comparing the Therapeutic Combinations Melarsoprol-Nifurtimox, Melarsoprol-Eflornithine and Eflornithine-Nifurtimox in the Treatment of Gambiense Human African Trypanosomiasis in the Meningo-Encephalitic Phase | Trypanosomiasis, African | Drug: melarsoprol 1.8 mg/kg/d, 10d + nifurtimox 15/20 mg/kg/d, 10d;Drug: melarsoprol 1.8 mg/kg/d, 10d + eflornithine 400 mg/kg/d, 7d;Drug: nifurtimox 15/20 mg/kg/d 10d + eflornithine 400 mg/kg/d 7d | Epicentre | Medecins Sans Frontieres;Embassy of France in Uganda;National Sleeping Sickness Control Program, Uganda | Terminated | N/A | N/A | Both | 435 | Phase 3 | Uganda |