Clinical trial NCT05659719: A Study to Learn About Recifercept in Patients With Achondroplasia
Official title: A Pilot Project to Evaluate the Feasibility of Constructing a Concurrent External Control for Recifercept
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Achondroplasia
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-12-21
Enrollment: 248
Sex: ALL
Minimum age (years): 3
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Achondroplasia patients, aged 3 months to 11 years old with documented and confirmed genetic diagnosis of achondroplasia, who have completed at least 2 valid height and length measurement in the natural history study and have at least 6 months of follow-up data in either the natural history study or the phase 2 recifercept trial.

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Mean Height Growth at Month 3
timeFrame: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Mean Height Growth at Month 6
timeFrame: Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Mean Height Growth at Month 9
timeFrame: Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Mean Height Growth at Month 12
timeFrame: Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Secondary outcomes:
measure: Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
timeFrame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
timeFrame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
timeFrame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Number of Participants With Achondroplasia Related Orthopedic Complications
timeFrame: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
measure: Number of Participants With Other Achondroplasia Related Orthopedic Complications
timeFrame: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Other outcomes:
Locations:
city: New York
country: United States
facility: Pfizer
state: New York
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: