11 completed, 3 terminated, 2 recruiting, 1 active not recruiting. 18 Phase 2, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 18 | Completed 11, Terminated 3, Recruiting 2, Active not recruiting 1, Withdrawn 1 |
| Phase 3 | 3 | Completed 2, Terminated 1 |
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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 |
|---|---|---|---|---|---|---|
| Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease Napazimone - Mitochondrial Diseases | Phase 2 | Recruiting | Pharming Technologies B.V. | 140 | 2026-06-12 | 2026-09-16 |
| Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation Leniolisib - PIDs Linked to PI3K | Phase 2 | Active not recruiting | Pharming Technologies B.V. | 12 | 2025-04-29 | 2026-09-16 |
| Leniolisib for Immune Dysregulation in CVID Leniolisib - Common Variable Immunodeficiency (CVID) | Phase 2 | Completed | Pharming Technologies B.V. | 27 | 2025-02-12 | 2026-09-18 |
| Leniolisib for Immune Dysregulation in PIDs Leniolisib - Primary Immunodeficiency Disorders (PIDs) | Phase 2 | Completed | Pharming Technologies B.V. | 13 | 2024-10-21 | 2026-10-19 |
| Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease KL1333, Placebo - Primary Mitochondrial Disease | Phase 2 | Recruiting | Pharming Technologies B.V. | 180 | 2022-12-13 | 2026-06-26 |
| Recombinant Human C1 Esterase Inhibitor (Conestat Alfa) in the Prevention of Acute Ischemic Cerebral and Renal Events After Transcatheter Aortic Valve Implantation Conestat alfa (Ruconest®), NaCl 0.9%) - Acute Ischemic Stroke, Acute Renal Injury | Phase 2 | Completed | University Hospital, Basel, Switzerland | 141 | 2022-03-16 | 2026-04-22 |
| Prevention of Acute Kidney Injury in Patients With NSTEMI conestat alfa or placebo - Non-ST Elevation Myocardial Infarction (NSTEMI) | Phase 2 | Terminated | Pharming Technologies B.V. | 29 | 2021-04-21 | 2024-04-03 |
| Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19 Ruconest - Confirmed Coronavirus Disease | Phase 2 | Completed | Pharming Technologies B.V. | 38 | 2020-11-30 | 2024-02-16 |
| Conestat Alfa in the Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19 Conestat alfa - Coronavirus Infections | Phase 2 | Terminated | University Hospital, Basel, Switzerland | 80 | 2020-08-06 | 2021-11-09 |
| Recombinant Human C1 Esterase Inhibitor in the Prevention of Contrast-induced Nephropathy in High-risk Subjects Conestat alfa, Sodium chloride 0.9% - Acute Kidney Injury | Phase 2 | Completed | University Hospital, Basel, Switzerland | 80 | 2017-01-01 | 2018-08-08 |
| Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI CDZ173 - Activated PI3Kdelta Syndrome (APDS); PASLI Disease | Phase 2 / Phase 3 | Terminated | Pharming Technologies B.V. | 37 | 2016-09-08 | 2026-03-30 |
| A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor Recombinant human C1 inhibitor, Placebo - Hereditary Angioedema | Phase 2 | Completed | Pharming Technologies B.V. | 32 | 2014-12-01 | 2017-12-08 |
| Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients rhC1INH - Hereditary Angioedema | Phase 2 | Completed | Pharming Technologies B.V. | 57 | 2012-01-17 | 2024-03-29 |
| Recombinant Human C1 Inhibitor for the Treatment of Early Antibody-Mediated Rejection in Renal Transplantation plasmapheresis and IVIG, recombinant C1 inhibitor - Graft Rejection, Kidney Transplantation | Phase 2 | Withdrawn | Pharming Technologies B.V. | 2010-12-01 | 2012-02-17 | |
| 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 Recombinant Human C1 Inhibitor, placebo - Hereditary Angioedema, Angioneurotic Edema | Phase 2 / Phase 3 | Completed | Pharming Technologies B.V. | 77 | 2005-07-01 | 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 / 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 |
Angioedemas, Hereditary (5) · Genetic Diseases, Inborn (3) · Acute Kidney Injury (2) · Angioedema (2) · Activated PI3K-delta Syndrome (1) · Aortic Valve Stenosis (1) · COVID-19 (1) · Common Variable Immunodeficiency (1) · Coronavirus Infections (1) · Cytokine Release Syndrome (1) · Diabetes Mellitus (1) · Heart Valve Diseases (1) · Immunologic Deficiency Syndromes (1) · Inflammation (1) · Ischemic Stroke (1)
conestat alfa (10) · Sodium Chloride (4) · leniolisib (4) · Complement C1 Inhibitor Protein (3) · Immunoglobulins, Intravenous (1) · Plasmapheresis (1)
United States (10) · Netherlands (7) · Czechia (5) · Italy (5) · Germany (4) · Spain (4) · Switzerland (4) · Romania (3) · United Kingdom (3) · Belgium (2) · Denmark (2) · France (2) · North Macedonia (2) · Australia (1) · Belarus (1)
Anurag Relan, MD (3) · Jan Nuijens, MD, PhD (2) · Amel Karaa, MD (1) · Bruno Giannetti, MD (1) · Gulbu Uzel, M.D. (1) · Hans Sollinger, MD, PhD (1) · Jonathan Bernstein, MD (1) · Michael Osthoff, M.D. (1) · Michael Osthoff, PD Dr. med. (1) · Michael Osthoff, Prof. Dr. med. (1) · Raban Jeger, Prof. Dr. med. (1)
PMID 20920772, PMID 23535096, PMID 28284978, PMID 28754491, PMID 30993784, PMID 32171721, PMID 33397449, PMID 36326435, PMID 37797893, PMID 37965347, PMID 38593221, PMID 41608120, PMID 42159953
2016 (1), 2025 (1)