A Study to Learn About Recifercept in Patients With Achondroplasia

Achondroplasia Clinical Trial

Official title:

A Pilot Project to Evaluate the Feasibility of Constructing a Concurrent External Control for Recifercept

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05659719
• Other study ID #: C4181010
• Status: Completed
• Start date: October 19, 2022
• Est. completion date: May 16, 2023

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already collected from a recifercept clinical trial and data collected from a separate study of achondroplasia. This study will compare patient experiences and will help the investigators determine if the study medicine, recifercept, is effective.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 250
• Est. completion date: May 16, 2023
• Est. primary completion date: May 16, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months to 15 Years

Eligibility

Inclusion Criteria:

• All patients from Study C4181005 who have completed Visits 1 through 11 (at D183) will be included in this project.

To construct a concurrent external control, patients from Study C4181001 will need to meet the following inclusion criteria from Study C4181005 to be eligible for inclusion:

• Documented, confirmed genetic diagnosis of achondroplasia from historical medical records (test must have been performed at a laboratory fully accredited for genetic testing under local regulations)

• Aged = 3 months to <11 years (up to the day before 11th birthday inclusive) at time of enrollment into the observational natural history study.

• Havecompleted at least 2 valid height/length measurements (at least 3 months apart)

• Assessed for Tanner stage 1 during physical examination before or at enrollment (must include assessment of breast development for females, testicular stage for males)

• Able to stand independently for height measurements (if = 2 years of age at enrollment); If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.

• Have at least 6 months of available follow-up data after enrollment into the natural history study

Exclusion Criteria:

• Patients meeting any of the following criteria will not be included in the study:

• Presence of severe obesity (BMI>95% percentile on Hoover-Fong BMI charts);

• Body weight <7kg or >30kg

• History of chronic kidney disease (CKD) or renal impairment

• History of receipt of any treatment that are known to potentially affect growth (including oral steroids > 5 days in the last 6 months before enrollment, high dose inhaled corticosteroids (>800 mcg/day beclametasone equivalent) and medication for attention deficient hyperactivity disorder.

• Less than 6 months since fracture or surgical procedure of any bone determined from the baseline visit date.

• Presence of any internal guided growth plates/devices

• History of removal of internal guided growth plates/devices within 6 months prior to enrollment

Related Conditions & MeSH terms

• Achondroplasia

Intervention

Other:
• Recifercept
Patients received recifercept intervention in the phase 2 clinical trial

Locations

Country	Name	City	State
United States	Pfizer	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height growth	Increase in height growth above expected growth in reference population, defined as the height growth in the achondroplasia reference population +50%	6 months
Secondary	Standing height	standing height in centimeters	6 months
Secondary	Sitting height	sitting height in centimeters	6 months
Secondary	Knee height	knee height in centimeters	6 months
Secondary	Arm span	arm span in centimeters	6 months
Secondary	Length of the legs	length of the legs in centimeters	6 months
Secondary	Sitting height to standing height ratio	sitting height to standing height ratio (unitless)	6 months
Secondary	arm span to height or length difference	arm span to height or length difference in centimeters	6 months
Secondary	knee height to low segment ratio	knee height to low segment ratio (unitless)	6 months
Secondary	Achondroplasia-related orthopaedic complications	lordosis, kyphoscoliosis, small foramen magnum, spinal stenosis	6 months
Secondary	Other chondroplasia-related complications	sleep disordered breathing, acute otitis media, hydrocephalus	6 months

External Links

•   To obtain contact information for a study center near you, click here.