Suspended

A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA

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What is being tested

Recifercept

Biological
Who is being recruted

Achondroplasia+16

+ Bone Diseases

+ Bone Diseases, Developmental

From 15 Months to 12 Years
+19 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: December 2021
See protocol details

Summary

Principal SponsorPfizer
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: December 24, 2021

Actual date on which the first participant was enrolled.

All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this open-label extension (OLE) study for up to an additional 24 months of treatment. Approximately 63 participants will be offered to continue at the previously received dose of Recifercept either Low Dose Medium Dose High Dose or at the therapeutic dose once it is identified. Participants will attend the clinic monthly for 24 months. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires.

Official TitleA PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
NCT05116046
Principal SponsorPfizer
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

35 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 15 Months to 12 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AchondroplasiaBone DiseasesBone Diseases, DevelopmentalCarbohydrate Metabolism, Inborn ErrorsConnective Tissue DiseasesDwarfismEndocrine System DiseasesMetabolic DiseasesMetabolism, Inborn ErrorsMucopolysaccharidosesMucopolysaccharidosis IVMusculoskeletal DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic DiseasesOsteochondrodysplasiasLysosomal Storage DiseasesSkin and Connective Tissue DiseasesMucinosesGenetic Diseases, Inborn

Criteria

4 inclusion criteria required to participate
Male and female participants between the ages of >15 months to <12 years inclusive, at Visit 1 (Screen 1).
Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests lifestyle considerations and other study procedures.
Completed the C4181005 Phase 2 study.
Able to stand independently for height measurements (if ≥2 years of age at enrollment).
15 exclusion criteria prevent from participating
Body weight >45 kg.
Presence of co-morbid conditions or circumstances that, in the opinion of the investigator, would affect interpretation of growth data or ability to complete the trial procedures.
Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
Presence of severe obesity (body mass index (BMI) >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

3 intervention groups are designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
High Dose

Group II

Experimental
Low Dose

Group III

Experimental
Medium Dose

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 14 locations

Suspended

Ocean Sleep Medicine

Irvine, United StatesOpen Ocean Sleep Medicine in Google Maps
Suspended

Long Beach Memorial Medical Center

Long Beach, United States
Suspended

MemorialCare Sleep Disorders Center at Long Beach Memorial Medical Center

Long Beach, United States
Suspended

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, United States
Suspended14 Study Centers