Phase 2 Clinical trial with Recifercept starts in December 2020
24th Nov 2020
The Phase 2 clinical trial with Recifercpet will recruit approximately 63 children with achondroplasia, that will be distributed randomly to one of three doses:
- Low Dose
- Medium Dose
- High Dose
The children will be divided into two cohorts (or groups) of 9 children between 0-2 years old and 54 children aged 2-10 years old (inclusive).
The younger group (0-2 yo) is the exploratory cohort and will be enrolled later in the study (n=3 per dose, low, medium, and high).1
The children that participate in this trial will attend the recruiting clinical center at baseline (day 0), day 1, 4, 8, 15, 29 & then Month 2, 3, 6, 9 & 12 (or 11 visits over a year) for assessments (physical and clinical evaluations) for safety, blood sampling, physical examination, vital signs, anthropometric body measurements & patient/caregiver quality of life questionnaires. 1
Children that participate will receive treatment with Recifercept for 12 months, and all that complete this study, and accordingly the opinion of the investigator (the healthcare professional leading the recruitment in each clinical center), if the child has a positive risk: benefit profile, will be offered to enroll in an open-label extension (OLE) study after the 52 weeks. 1
What is the risk:benefit assessment?
Pharmaceutical products have become the mainstay of prevention, treatment, and diagnosis of disease. However, modern medicines, while being biologically effective, also have the potential for harm. Balancing the desirable effects (benefits) and undesirable (risks) of drugs is the core task of drug regulatory agencies (benefit-risk assessment). 2 A positive risk:benefit profile relates to a participant that presents and experiences expected or unexpected positive results (benefits) over limited, minimal, or nill risks.
For children to be able to participate, need to be enrolled prior in the Dreambird study and be aged ≥3 months to <11 years (up to the day before 11th birthday inclusive) at the time of enrollment.
Starting period: Pfizer intends to start recruitment in 3 to 4 clinical centers in December 2020 and the remaining centers will follow in early 2021.
References
1. ClinicialTrials.gov
2. Risk: benefits - European Medicines Agency
