A Study to Learn About Recifercept in Patients With Achondroplasia

• ClinicalTrials.gov Identifier: NCT05659719
• Recruitment Status: Active, not recruiting
• First Posted: December 21, 2022
• Last Update Posted: March 3, 2023
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

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.

Condition or disease	Intervention/treatment
Achondroplasia	Other: Recifercept

Study Design

• Study Type: Observational
• Actual Enrollment: 250 participants
• Observational Model: Cohort
• Time Perspective: Retrospective
• Official Title: A Pilot Project to Evaluate the Feasibility of Constructing a Concurrent External Control for Recifercept
• Actual Study Start Date: October 19, 2022
• Estimated Primary Completion Date: March 31, 2023
• Estimated Study Completion Date: March 31, 2023

Groups and Cohorts

Group/Cohort	Intervention/treatment
Recifercept cohort; Achondroplasia patients enrolled in the recifercept phase 2 clinical trial	Other: Recifercept; Patients received recifercept intervention in the phase 2 clinical trial
Natural history cohort; Achondroplasia patients enrolled in the achondroplasia natural history study

Outcome Measures

Primary Outcome Measures :

1. Height growth [ Time Frame: 6 months ]
   Increase in height growth above expected growth in reference population, defined as the height growth in the achondroplasia reference population +50%

Secondary Outcome Measures :

1. Standing height [ Time Frame: 6 months ]
   standing height in centimeters
2. Sitting height [ Time Frame: 6 months ]
   sitting height in centimeters
3. Knee height [ Time Frame: 6 months ]
   knee height in centimeters
4. Arm span [ Time Frame: 6 months ]
   arm span in centimeters
5. Length of the legs [ Time Frame: 6 months ]
   length of the legs in centimeters
6. Sitting height to standing height ratio [ Time Frame: 6 months ]
   sitting height to standing height ratio (unitless)
7. arm span to height or length difference [ Time Frame: 6 months ]
   arm span to height or length difference in centimeters
8. knee height to low segment ratio [ Time Frame: 6 months ]
   knee height to low segment ratio (unitless)
9. Achondroplasia-related orthopaedic complications [ Time Frame: 6 months ]
   lordosis, kyphoscoliosis, small foramen magnum, spinal stenosis
10. Other chondroplasia-related complications [ Time Frame: 6 months ]
    sleep disordered breathing, acute otitis media, hydrocephalus

Eligibility Criteria

• Ages Eligible for Study: 3 Months to 15 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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.

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

Contacts and Locations

Locations

United States, New York

Pfizer

New York, New York, United States, 10017

Sponsors and Collaborators

Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

More Information

Additional Information:

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

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT05659719
• Other Study ID Numbers: C4181010
• First Posted: December 21, 2022
• Last Update Posted: March 3, 2023
• Last Verified: March 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Additional relevant MeSH terms:

Achondroplasia; Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn