TBD ( DrugBank: - )
5 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 3 |
| 86 | Pulmonary arterial hypertension | 3 |
| 90 | Retinitis pigmentosa | 1 |
| 276 | Achondroplasia | 1 |
| 299 | Cystic fibrosis | 1 |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | EUCTR2014-003480-37-IT (EUCTR) | 06/05/2015 | 07/01/2015 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2). MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1) | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | United States;Germany;United Kingdom;Japan;Italy | |||
| 2 | EUCTR2012-005430-11-IT (EUCTR) | 14/05/2014 | 20/01/2014 | Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease | Neuronal Ceroid Lipofuscinosis Type 2 (CLN2). MedDRA version: 16.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: N/A Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | Germany;United Kingdom;Italy | ||
| 3 | EUCTR2012-005430-11-DE (EUCTR) | 11/09/2013 | 10/04/2013 | Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease | Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2) MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: N/A Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | Germany;United Kingdom |
86. Pulmonary arterial hypertension
Clinical trials : 1,205 / Drugs : 684 - (DrugBank : 124) / Drug target genes : 100 - Drug target pathways : 193
| 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 | EUCTR2016-001411-20-GB (EUCTR) | 12/01/2018 | 09/01/2017 | Long-term extension study of the safety and pharmacokinetics of QCC374 in PAH patients | Long-term, open label, multicenter, extension study to evaluate the safety and tolerability of QCC374 in patients with PAH | Pulmonary arterial hypertension MedDRA version: 20.0;Level: PT;Classification code 10064911;Term: Pulmonary arterial hypertension;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: QCC374 0.015 mg INN or Proposed INN: TBD Product Code: QCC374 0.06 mg INN or Proposed INN: TBD | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 38 | Phase 2 | United States;Taiwan;Germany;United Kingdom;Korea, Republic of | ||
| 2 | EUCTR2016-001411-20-DE (EUCTR) | 17/10/2017 | 07/08/2017 | Long-term extension study of the safety and pharmacokinetics of QCC374 in PAH patients | Long-term, open label, multicenter, extension study to evaluate the safety and tolerability of QCC374 in patients with PAH | Pulmonary arterial hypertension MedDRA version: 20.0;Level: PT;Classification code 10064911;Term: Pulmonary arterial hypertension;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: QCC374 0.015 mg INN or Proposed INN: TBD Product Code: QCC374 0.06 mg INN or Proposed INN: TBD | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 38 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Taiwan;Germany;United Kingdom;Korea, Republic of | ||
| 3 | EUCTR2016-001412-38-DE (EUCTR) | 27/07/2017 | 03/04/2017 | Safety, pharmacokinetics and efficacy study of QCC374 in PAH patients | A randomized, parallel-group, placebo-controlled subject and investigator blinded study to assess the safety, tolerability, pharmacokinetics and efficacy of QCC374 in the treatment of pulmonary arterial hypertension | Pulmonary arterial hypertension MedDRA version: 20.0;Level: PT;Classification code 10064911;Term: Pulmonary arterial hypertension;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Code: QCC374 0.015 mg INN or Proposed INN: TBD Product Code: QCC374 0.06 mg INN or Proposed INN: TBD | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 38 | Phase 2 | United States;Taiwan;Germany;United Kingdom;Korea, Republic of |
90. Retinitis pigmentosa
Clinical trials : 147 / Drugs : 176 - (DrugBank : 43) / Drug target genes : 49 - Drug target pathways : 110
| 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 | EUCTR2016-002696-10-SE (EUCTR) | 09/05/2018 | 21/12/2017 | Explore the safety and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene | An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene | retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: CPK850 INN or Proposed INN: TBD | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Sweden |
276. Achondroplasia
Clinical trials : 51 / Drugs : 34 - (DrugBank : 6) / Drug target genes : 4 - Drug target pathways : 26
| 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 | EUCTR2013-004137-32-FR (EUCTR) | 18/06/2015 | A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia | achondroplasia MedDRA version: 18.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: TBD Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;France;Australia;United Kingdom |
299. Cystic fibrosis
Clinical trials : 1,695 / Drugs : 1,527 - (DrugBank : 268) / Drug target genes : 111 - Drug target pathways : 174
| 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 | EUCTR2019-000750-63-NL (EUCTR) | 31/10/2019 | 05/08/2019 | Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutation | A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation | Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ABBV-2222 Product Code: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 Product Name: ABBV-3067 Product Code: ABBV-3067 INN or Proposed INN: TBD Other descriptive name: ABBV-3067 | AbbVie Deutschland | NULL | Not Recruiting | Female: yes Male: yes | 189 | Phase 2 | Serbia;United States;Slovakia;Spain;United Kingdom;France;Czech Republic;Hungary;Canada;Puerto Rico;Belgium;Poland;Netherlands |