Cipaglucosidase alfa
- Generic Name
- Cipaglucosidase alfa
- Brand Names
- Pombiliti
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 2359727-71-0
- Unique Ingredient Identifier
- 4SED7F4BSG
- Background
Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA. Other types of enzyme replacement therapy for the treatment of Pompe disease include alglucosidase alfa and avalglucosidase alfa. Cipag...
- Indication
In Europe, cipaglucosidase alfa is a long-term enzyme replacement therapy used in combination with the enzyme stabilizer miglustat for the treatment of adults with late-onset Pompe disease, also known as acid α-glucosidase (GAA) deficiency.
- Associated Conditions
- Late-onset Pompe Disease
- Associated Therapies
- -
A Global Prospective Observational Registry of Patients With Pompe Disease
- Conditions
- Interventions
- First Posted Date
- 2023-11-07
- Last Posted Date
- 2024-10-30
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 500
- Registration Number
- NCT06121011
- Locations
- 🇺🇸
Cincinnati Children's Hospital, Cincinnati, Ohio, United States
🇩🇪Universitätsklinikum Gießen und Marburg GmhH, Marburg, Germany
🇺🇸University of Arkansas Medical Science, Little Rock, Arkansas, United States
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
- Conditions
- Interventions
- First Posted Date
- 2021-03-22
- Last Posted Date
- 2024-11-06
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 36
- Registration Number
- NCT04808505
- Locations
- 🇺🇸
University of Florida Clinical Research Center, Gainesville, Florida, United States
🇺🇸The Emory Clinic, Atlanta, Georgia, United States
🇺🇸Duke University Early Phase Research Unit, Durham, North Carolina, United States
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
- Conditions
- Interventions
- First Posted Date
- 2019-04-11
- Last Posted Date
- 2024-07-01
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 22
- Registration Number
- NCT03911505
- Locations
- 🇺🇸
Woodruff Memorial Research Building, Atlanta, Georgia, United States
🇺🇸UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇺🇸Arkansas Children's Hospital, Little Rock, Arkansas, United States
Drug Development Updates
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