65. Primary immunodeficiency
413 clinical trials, 581 drugs (DrugBank: 97 drugs), 68 drug target genes, 202 drug target pathways
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 | NCT03974178 (ClinicalTrials.gov) | September 29, 2019 | 23/5/2019 | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense | Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense: a Multicentre, Open-label Clinical Trial | Trypanosoma Brucei Rhodesiense; Infection | Drug: Fexinidazole | Drugs for Neglected Diseases | European and Developing Countries Clinical Trials Partnership (EDCTP) | Recruiting | 6 Years | N/A | All | 50 | Phase 2;Phase 3 | Malawi;Uganda |
| 2 | NCT03025789 (ClinicalTrials.gov) | November 17, 2016 | 11/11/2016 | Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | An Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole in Patients With Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage | Trypanosomiasis, African;Sleeping Sickness;Trypanosomiasis; Gambian | Drug: Fexinidazole | Drugs for Neglected Diseases | Sanofi | Active, not recruiting | 6 Years | N/A | All | 174 | Phase 3 | Congo, The Democratic Republic of the;Guinea |
| 3 | NCT02571062 (ClinicalTrials.gov) | March 2015 | 24/6/2015 | Bioequivalence Study - Reference Clinical Fexinidazole Tablet Versus Proposed Market Formulation | A Bioequivalence Study of the Reference Clinical Fexinidazole Tablet vs Proposed Market Formulation in Healthy Male Volunteers of African Sub-Saharan Origin:an Open-label,Randomized,Two-treatment,Single Dose,Replicate Design,Fed Condition | Trypanosomiasis, African | Drug: Fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 18 Years | 45 Years | Male | 30 | Phase 1 | France |
| 4 | NCT02498782 (ClinicalTrials.gov) | July 2014 | 20/8/2014 | Study to Evaluate Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chagas Disease | Phase 2, Randomized, Multicenter, Placebo-controlled, Safety and Efficacy Study to Evaluate Six Oral Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chronic Indeterminate Chagas Disease. | Chagas Disease;Trypanosomiasis, South American;South American Trypanosomiasis;Disease, Chagas | Drug: Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | NULL | Recruiting | 18 Years | 50 Years | Both | 140 | Phase 2 | Bolivia |
| 5 | NCT02184689 (ClinicalTrials.gov) | May 3, 2014 | 19/6/2014 | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 6 Years | 14 Years | All | 125 | Phase 2;Phase 3 | Congo, The Democratic Republic of the |
| 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 | NCT02169557 (ClinicalTrials.gov) | April 30, 2014 | 15/5/2014 | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study | Human African Trypanosomiasis (HAT) | Drug: Fexinidazole | Drugs for Neglected Diseases | NULL | Completed | 15 Years | N/A | All | 230 | Phase 2;Phase 3 | Congo, The Democratic Republic of the |
| 7 | 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 |
| 8 | NCT01483170 (ClinicalTrials.gov) | September 2011 | 29/11/2011 | Multiple Dose Study to Evaluate Security, Tolerance and Pharmacokinetic of Fexinidazole (Drug Candidate for Human African Trypanosomiasis) Administered With a Loading Dose and With Food | Double-blind, Placebo Controlled, Randomized Multiple Ascending Dose Study in Fed Conditions for Ten Days Dosing Regimen With a Loading Dose to Evaluate the Safety, the Tolerability and the Pharmacokinetics of Oral Fexinidazole in 36 Healthy Male Sub-Saharan Volunteers. | Trypanosomiasis, African | Drug: Tablets Fexinidazole;Drug: Placebo | Drugs for Neglected Diseases | NULL | Terminated | 18 Years | 45 Years | Male | 30 | Phase 1 | France |
| 9 | NCT00982904 (ClinicalTrials.gov) | September 2009 | 22/9/2009 | Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the Fexinidazole | Randomized, Double-blind, Placebo-controlled Study of the Tolerability, and Pharmacokinetics of Fexinidazole After Single and Repeated Oral Ascending Doses, Completed by a Comparative Bioavailability Study of an Oral Suspension Versus a Tablet and an Exploratory Assessment of Food Effect, in Healthy Male Volunteers | Human African Trypanosomiasis | Drug: Fexinidazole/Placebo | Drugs for Neglected Diseases | Sanofi | Completed | 18 Years | 45 Years | Male | 108 | Phase 1 | France |