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 | NCT03988426 (ClinicalTrials.gov) | March 7, 2017 | 13/6/2019 | Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases | Clinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases. | Primary Immune Deficiency Disorder | Biological: Octanorm | Octapharma | NULL | Completed | 18 Years | 70 Years | All | 25 | Phase 3 | Russian Federation |
| 2 | EUCTR2013-003877-87-SK (EUCTR) | 13/01/2017 | 17/03/2016 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1;Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland;Russian Federation | ||
| 3 | NCT03907241 (ClinicalTrials.gov) | March 1, 2016 | 7/12/2018 | CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL | Title for SCGAM-03: CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES WHO HAVE COMPLETED THE SCGAM-01 TRIAL Title for SCGAM-03 in Canada: CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL | Primary Immunodeficiency | Drug: Octanorm 16.5% | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 27 | Phase 3 | United States;Canada |
| 4 | NCT02627300 (ClinicalTrials.gov) | March 2016 | 3/12/2015 | Study of Octanorm Subcutaneous IG in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Clinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 Trial | Primary Immunodeficiency Disease | Drug: Octanorm 16.5% | Octapharma | NULL | Completed | N/A | N/A | All | 21 | Phase 3 | United States;Canada |
| 5 | EUCTR2013-003877-87-PL (EUCTR) | 11/03/2015 | 04/12/2014 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency MedDRA version: 20.0;Level: HLT;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland | ||
| 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 | EUCTR2013-003877-87-HU (EUCTR) | 14/03/2014 | 07/01/2014 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 3 | United States;Czech Republic;Hungary;Slovakia;Canada;Poland | ||
| 7 | EUCTR2013-003877-87-CZ (EUCTR) | 12/03/2014 | 19/12/2013 | Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system. | CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN(OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | Primary immunodeficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Octanorm 16.5% INN or Proposed INN: Human Normal Immunoglobulin Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Octapharma Pharmazeutika Prod.Ges.m.b.H | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1;Phase 3 | United States;Hungary;Czech Republic;Slovakia;Canada;Poland | ||
| 8 | NCT01888484 (ClinicalTrials.gov) | March 2014 | 21/6/2013 | Study of Octanorm Subcutaneous IG in Patients With PID | Clinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder | Biological: octanorm 16.5% | Octapharma | NULL | Completed | 2 Years | 75 Years | All | 75 | Phase 3 | United States;Canada;Czechia;Hungary;Poland;Russian Federation;Slovakia;Czech Republic |