65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 482 / Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212
| 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 | EUCTR2013-005508-33-CZ (EUCTR) | 12/11/2014 | 29/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 mg, powder for solution for infusion Product Name: Ovastat 1000 INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 mg, powder for solution for infusion Product Name: Ovastat 5000 INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy |