Pozelimab
- Generic Name
- Pozelimab
- Brand Names
- Veopoz
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 2096328-94-6
- Unique Ingredient Identifier
- 0JJ21K6L2I
- Background
CD55-deficient protein-losing enteropathy (PLE), or CHAPLE disease, is an ultra-rare hereditary disease, with fewer than 100 patients diagnosed worldwide or fewer than 10 patients in the US. The pathophysiology of this disease is mainly attributed to the deficiency of the CD55 protein, which is the main regulator of the complement cascade.. Under normal circ...
- Indication
Pozelimab is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.
- Associated Conditions
- CHAPLE disease
- Associated Therapies
- -
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
- Conditions
- Interventions
- First Posted Date
- 2024-08-07
- Last Posted Date
- 2024-12-18
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 750
- Registration Number
- NCT06541704
- Locations
- πΊπΈ
Colorado Retina - Lakewood, Lakewood, Colorado, United States
πΊπΈGeorgia Retina - Marietta, Marietta, Georgia, United States
πΊπΈPalmetto Retina Center, West Columbia, South Carolina, United States
Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
- Conditions
- First Posted Date
- 2023-09-08
- Last Posted Date
- 2023-09-08
- Lead Sponsor
- Regeneron Pharmaceuticals
- Registration Number
- NCT06028594
Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
- First Posted Date
- 2023-08-22
- Last Posted Date
- 2023-08-22
- Lead Sponsor
- Regeneron Pharmaceuticals
- Registration Number
- NCT06003881
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works.
- Conditions
- Interventions
- First Posted Date
- 2023-02-27
- Last Posted Date
- 2024-12-18
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 202
- Registration Number
- NCT05744921
- Locations
- π¨π³
Chang Gung Memorial Hospital- Linkou Branch, Taoyuan, Taiwan
π¨π¦Toronto General Hospital, Toronto, Ontario, Canada
ππΊSemmelweis University/Semmelweis Egyetem, Budapest, Hungary
Compassionate Use of Pozelimab
- First Posted Date
- 2022-02-09
- Last Posted Date
- 2024-11-08
- Lead Sponsor
- Regeneron Pharmaceuticals
- Registration Number
- NCT05232110
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
- Conditions
- Interventions
- First Posted Date
- 2021-11-24
- Last Posted Date
- 2024-12-19
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 190
- Registration Number
- NCT05133531
- Locations
- π΅πͺ
Clinica San Felipe, Lima, Peru
π΅πSt Lukes Medical Center, Quezon City, Central Luzon, Philippines
π¨π³Taichung Veterans General Hospital (VGHTC), Taichung City, Taiwan
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
- Conditions
- Interventions
- First Posted Date
- 2021-11-23
- Last Posted Date
- 2023-08-16
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 2
- Registration Number
- NCT05131204
- Locations
- πΊπΈ
Regeneron Research Facility, Whittier, California, United States
A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis
- Conditions
- Interventions
- First Posted Date
- 2021-10-07
- Last Posted Date
- 2024-12-05
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 235
- Registration Number
- NCT05070858
- Locations
- πΊπΈ
HonorHealth Neurology 2018, Scottsdale, Arizona, United States
πΊπΈUniversity of California Irvine, Irvine, California, United States
πΊπΈUniversity of Southern California, Los Angeles, California, United States
A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers
- First Posted Date
- 2021-06-25
- Last Posted Date
- 2022-07-06
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 30
- Registration Number
- NCT04940364
- Locations
- π¬π§
Regeneron Research Site, London, United Kingdom
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
- Conditions
- Interventions
- First Posted Date
- 2021-05-17
- Last Posted Date
- 2023-06-13
- Lead Sponsor
- Regeneron Pharmaceuticals
- Target Recruit Count
- 6
- Registration Number
- NCT04888507
- Locations
- π¬π§
Regeneron Study Site, Leeds, United Kingdom
Drug Development Updates
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