5 completed. 5 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 5 |
| Phase 3 | 1 | Completed 1 |
Follow Genetic Disorders: new trials by email Literature Review of Genetic Disorders trials Research Report on Genetic Disorders
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A PXT3003 Low dose, PXT3003 Intermediate Dose, PXT3003 High Dose - Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies | Phase 2 | Completed | Pharnext S.C.A. | 80 | 2010-12-01 | 2017-11-22 |
| A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration Recombinant Human C1 Inhibitor - Genetic Disorders, Hereditary Angioedema | Phase 2 | Completed | Pharming Technologies B.V. | 25 | 2009-06-01 | 2018-06-26 |
| Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema i.v. recombinant human C1 inhibitor - Genetic Disorders | Phase 2 / Phase 3 | Completed | Pharming Technologies B.V. | 14 | 2004-04-01 | 2013-02-22 |
| Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema i.v. recombinant human C1 inhibitor - Genetic Disorders | Phase 2 | Completed | Pharming Technologies B.V. | 14 | 2003-06-01 | 2013-04-25 |
| Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis Busulfan, Cyclophosphamide, BMD - Genetic Disorders, Sickle Cell Anemia | Phase 2 | Completed | Memorial Sloan Kettering Cancer Center | 25 | 1994-01-01 | 2008-09-12 |
Genetic Diseases, Inborn (5) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Angioedemas, Hereditary (1) · Charcot-Marie-Tooth Disease (1) · Hereditary Sensory and Motor Neuropathy (1) · Thalassemia (1) · Tomaculous neuropathy (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Anemia (1) · Anemia, Aplastic (1) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1) · Anemia, Hypoplastic, Congenital (1) · Angioedema (1) · Bone Marrow Diseases (1) · Bone Marrow Failure Disorders (1) · Cardiovascular Diseases (1) · Congenital Abnormalities (1) · Congenital Bone Marrow Failure Syndromes (1) · Hematologic Diseases (1) · Hemic and Lymphatic Diseases (1) · Hemoglobinopathies (1)
Busulfan (1) · Complement C1 Inhibitor Protein (1) · Cyclophosphamide (1) · conestat alfa (1)
Pharming Technologies B.V. (3) · Memorial Sloan Kettering Cancer Center (1) · Pharnext S.C.A. (1)
Netherlands (3) · France (1) · Romania (1) · United States (1)
Jan Nuijens, MD, PhD (2) · Bruno Giannetti, MD (1) · Farid Boulad, MD (1)