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Trial record 1 of 5 for:    recifercept
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A Study to Learn About Recifercept in Patients With Achondroplasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT05659719
Recruitment Status : Active, not recruiting
First Posted : December 21, 2022
Last Update Posted : March 3, 2023
Information provided by (Responsible Party):

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

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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

Resource links provided by the National Library of Medicine

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

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT05659719

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United States, New York
New York, New York, United States, 10017
Sponsors and Collaborators
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Study Director: Pfizer Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer Identifier: NCT05659719    
Other Study ID Numbers: C4181010
First Posted: December 21, 2022    Key Record Dates
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:
Additional relevant MeSH terms:
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Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn