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 | EUCTR2013-002290-21-HU (EUCTR) | 13/10/2014 | 14/07/2014 | Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases - Study to compare Gammaplex 10% & 5% in Primary Immunodeficiency | Primary immunodeficiency diseases MedDRA version: 17.0;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: Gammaplex Product Name: Gammaplex INN or Proposed INN: Human Normal Immunoglobulin Product Name: Gammaplex 10 INN or Proposed INN: Human Normal Immunoglobulin | Bio Products Laboratory Limited | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;Hungary;United Kingdom | ||
| 2 | EUCTR2013-002290-21-GB (EUCTR) | 30/04/2014 | 21/10/2013 | Study to compare the blood levels and safety of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases - Study to compare Gammaplex 10% & 5% in Primary Immunodeficiency | Primary immunodeficiency diseases MedDRA version: 17.1;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: Gammaplex Product Name: Gammaplex INN or Proposed INN: Human Normal Immunoglobulin Product Name: Gammaplex 10 INN or Proposed INN: Human Normal Immunoglobulin | Bio Products Laboratory Limited | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;Hungary;Israel;United Kingdom | ||
| 3 | NCT01963143 (ClinicalTrials.gov) | February 2014 | 13/9/2013 | Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | A Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyper-IgM Syndrome | Biological: Gammaplex (5%);Biological: Gammaplex 10 | Bio Products Laboratory | NULL | Completed | 2 Years | 55 Years | All | 48 | Phase 3 | United States;Hungary;United Kingdom |
| 4 | NCT01289847 (ClinicalTrials.gov) | March 2011 | 27/1/2011 | A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency | A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents | Primary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Hyper-IgM Syndrome;Wiskott-Aldrich Syndrome | Biological: Gammaplex | Bio Products Laboratory | NULL | Completed | 2 Years | 16 Years | All | 25 | Phase 4 | United States;Chile;Israel |
| 5 | NCT00278954 (ClinicalTrials.gov) | January 2006 | 18/1/2006 | Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. | A Phase III, Multicenter, Open-Label Study To Evaluate The Efficacy, Safety, and Pharmacokinetics of Gammaplex® in Primary Immunodeficiency Diseases | Primary Immunodeficiency;Common Variable Hypogammaglobulinemia;X-linked Hypogammaglobulinemia;Hypogammaglobulinemia;Immunodeficiency With Hyper-IgM;Wiskott-Aldrich Syndrome | Biological: Gammaplex (Intravenous immunoglobulin) | Bio Products Laboratory | NULL | Completed | 3 Years | N/A | All | 50 | Phase 3 | United States |
| 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 | EUCTR2011-005679-18-Outside-EU/EEA (EUCTR) | 07/12/2011 | A research study to determine if Gammaplex®, is safe, tolerable, and effective when given to children and adolescents who have Primary Immunodeficiency Diseases (PID) | A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents - n/a | Primary immunodeficiency diseases MedDRA version: 14.0;Level: SOC;Classification code 10005329;Term: Blood and lymphatic system disorders;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Gammaplex Product Name: Gammaplex Product Code: n/a INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN | Bio Products Laboratory Limited | NULL | NA | Female: yes Male: yes | 25 | Phase 4 | Chile;United States |