Continuation Study of Long-term Safety, Tolerability, Pha... | NCT05116046

NCT05116046

ID	Type	Description	Link
2021-003149-39	EudraCT Number

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Low Dose	Experimental	Low Dose	Biological: Recifercept
Medium Dose	Experimental	Medium Dose	Biological: Recifercept
High Dose	Experimental	High Dose	Biological: Recifercept

Name	Type	Description	Arm Group Labels	Other Names
Recifercept	Biological	Recifercept	High Dose Low Dose Medium Dose

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs	An AE was any untoward medical occurrence that did not necessarily have a causal relationship with study treatment. TEAE was an AE that occurred after initiation of study treatment that was not present at the time of treatment start or an AE that increased in severity after the initiation of medication, if the event was present at the time of treatment start emerges. SAE was an AE resulting in any of the following outcomes or considered medically significant: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly or birth defect. Severe AEs were AEs that were medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated disabling, limiting self-care activities of daily living.	From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months)
Change From Baseline in Height at Month 24	Height was measured using anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.	Baseline, Month 24

Secondary Outcomes

Measure	Description	Time Frame
Clearance (CL/F) of Recifercept	Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes.	Predose on Day 91, 181, 271, 361 and 451.
Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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).

Exclusion Criteria:

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].
Known closure of long bone growth plates (cessation of height growth).
Body weight >45 kg.
History of hypersensitivity to study intervention or any excipients.
History of any prior treatment with human growth hormone or related products (including insulin-like growth factor 1 [IGF-1]).
History of receipt of any treatment that are known to potentially affect growth (including oral steroids >5 days in the last 6 months, high dose inhaled corticosteroids (>800 mcg/day beclometasone equivalent) and medication for attention deficit hyperactivity disorder).
History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
Presence of any internal guided growth plates/devices.
History of removal of internal guided growth plates/devices within less than 6 months.
History of receipt of any other (except recifercept) investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
History of receipt of an investigational drug (not for achondroplasia/growth affecting) within the last 30 days (or as determined by the local requirement) or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer).

Contacts/Locations Module

Name	Affiliation	Role
Pfizer CT.gov Call Center	Pfizer	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Ocean Sleep Medicine		Irvine	California	92604	United States
Long Beach Memorial Medical Center

References Module

Label	URL
To obtain contact information for a study center near you, click here.	View source

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
FG001	Recifercept 2 mg/kg Twice Weekly

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	May 17, 2022	Dec 11, 2023

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

No data available

No data is available for this block.

Masking Info

Full Analysis Set (FAS) included all participants who received at least 1 dose of recifercept. Participants were analyzed according to the dose they actually received.

Arm/Group Information

ID	Title	Description
BG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
BG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
BG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.
BG003	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

Years

Title

Denominators

Categories

Title	Measurements
	BG0007.5± 2.66 BG0016.6± 2.50 BG0029.5± 0.71 BG003

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0009 BG0019 BG002

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0002 BG0010 BG002

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000 BG0010 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs

An AE was any untoward medical occurrence that did not necessarily have a causal relationship with study treatment. TEAE was an AE that occurred after initiation of study treatment that was not present at the time of treatment start or an AE that increased in severity after the initiation of medication, if the event was present at the time of treatment start emerges. SAE was an AE resulting in any of the following outcomes or considered medically significant: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly or birth defect. Severe AEs were AEs that were medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated disabling, limiting self-care activities of daily living.

FAS consisted of all participants who received at least one dose of recifercept. Participants were analyzed according to the dose they actually received.

Posted

Count of Participants

Participants

From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months)

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Units	Counts
Participants	OG00016 OG00117 OG0022

Title

Denominators

Categories

AEs

Title	Measurements
	OG0009 OG00111 OG0020

SAEs

Primary

Change From Baseline in Height at Month 24

Height was measured using anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Month 24

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Clearance (CL/F) of Recifercept

Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Predose on Day 91, 181, 271, 361 and 451.

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9

Sitting height to standing height ratio was calculated based upon the anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline and Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9

Height and length difference was calculated with anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline and Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9

Knee height was defined as the distance from the sole of the foot to the most anterior surface of the femoral condyles of the thigh (medial being more anterior), with the ankle and knee each flexed to a 90-degree angle. Lower segment of the leg included tibia and foot height

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline and Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9

Occipito-frontal circumference was measured by anthropometric measurements. It was measured over the most prominent part on the back of the head (occiput) and just above the eyebrows (supraorbital ridges).

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline and Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9

Occipito-frontal circumference was measured by anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9

The Z-score described how many standard deviations a given measurement lies above or below a size or age-specific population mean. A Z-score above the population mean indicates a positive value, whereas a Z-score below the population mean indicates a negative value. The greater the deviation of the Z-score from zero (in a positive or negative direction), the greater the magnitude of deviation from the mean.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily

Secondary

Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9

Fixed Flexion Angles was measured by anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Units	Counts

Secondary

Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9

Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Posted

Baseline, Months 3, 6, 9

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Units	Counts

Secondary

Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study

Laboratory parameters that were assessed included lymphocytes, neutrophils, eosinophils, monocytes and potassium. Clinically significant abnormal laboratory findings were determined based on investigator's decision.

FAS included all participants who were planned to receive at least one dose of recifercept. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure.'

Posted

Count of Participants

Participants

From baseline up to end of study/early termination (for a maximum duration of 11 months)

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Secondary

Number of Participants With Clinically Significant Findings in Vital Signs Through the Study

Absolute values and changes from baseline in supine systolic and diastolic blood pressure, oral temperature, and pulse rate were planned to be summarized by treatment in accordance with the sponsor reporting standards. Clinically significant abnormal laboratory findings were those which were not associated with the underlying disease, unless judged by the investigator to be more severe than expected for the participant's condition.

FAS included all participants who were planned to receive at least one dose of recifercept.

Posted

Count of Participants

Participants

From baseline up to end of study/early termination (for a maximum duration of 11 months)

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily

Secondary

Number of Participants With Clinically Significant Findings in Physical Examination Through the Study

A complete physical examination included cardiovascular, respiratory, gastrointestinal systems, and skin. Height and weight will also be measured and recorded as part of the anthropometric measurements collected. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual.

FAS included all participants who were planned to receive at least one dose of recifercept.

Posted

Count of Participants

Participants

From baseline up to end of study/early termination (for a maximum duration of 11 months)

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily

Secondary

Number of Participants With Positive Anti-Drug Antibodies (ADA)

FAS included all participants who were planned to receive at least one dose of recifercept.

Posted

Count of Participants

Participants

From Month 3 up to end of study/early termination (up to Month 11)

ID	Title	Description
OG000	Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
OG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
OG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

Units	Counts

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Recifercept 1 mg/kg Once Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.	16	16	9	16
EG001	Recifercept 2 mg/kg Twice Weekly	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.	17	17	11	17
EG002	Recifercept 1.5 mg/kg Once Daily	Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.	2	2	0	2

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Lymphopenia	Blood and lymphatic system disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Ear pain	Ear and labyrinth disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Eustachian tube dysfunction	Ear and labyrinth disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Tympanic membrane perforation	Ear and labyrinth disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Odynophagia	Gastrointestinal disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Toothache	Gastrointestinal disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Vomiting	Gastrointestinal disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Fatigue	General disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Injection site rash	General disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Pyrexia	General disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Hypersensitivity	Immune system disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
COVID-19	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0002 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Conjunctivitis	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Injection site haematoma	General disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Ear infection	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Gastroenteritis viral	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Influenza	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0002 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Nasopharyngiti	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0003 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Oral herpes	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Otitis externa	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Otitis media	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Otitis media acute	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Rhinitis	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0014 affected17 at risk EG0020 affected2 at risk
Skin candida	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Viral infection	Infections and infestations	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Clavicle fracture	Injury, poisoning and procedural complications	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Vaccination complication	Injury, poisoning and procedural complications	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Blood phosphorous increased	Investigations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Blood urea increase	Investigations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Platelet count increased	Investigations	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Headache	Nervous system disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Nystagmus	Nervous system disorders	MedDRA v25.1	Non-systematic Assessment	EG0001 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Breast pain	Reproductive system and breast disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Catarrh	Respiratory, thoracic and mediastinal disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA v25.1	Non-systematic Assessment	EG0003 affected16 at risk EG0010 affected17 at risk EG0020 affected2 at risk
Dermatitis	Skin and subcutaneous tissue disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0011 affected17 at risk EG0020 affected2 at risk
Haematoma	Vascular disorders	MedDRA v25.1	Non-systematic Assessment	EG0000 affected16 at risk EG0012 affected17 at risk EG0020 affected2 at risk

Data was not collected for efficacy endpoints as study was terminated early based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.

Certain Agreements

First MCP Posted Date

ID	Term
D000130	Achondroplasia
D004392	Dwarfism
D001847	Bone Diseases
D009140	Musculoskeletal Diseases
D010009	Osteochondrodysplasias
D030342	Genetic Diseases, Inborn
D009085	Mucopolysaccharidosis IV

Ancestor Terms

ID	Term
D001848	Bone Diseases, Developmental
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D004700	Endocrine System Diseases
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D016464	Lysosomal Storage Diseases
D017520	Mucinoses
D003240	Connective Tissue Diseases
D017437	Skin and Connective Tissue Diseases
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

Title	Measurements
	OG0000 OG0010 OG0020

Units	Counts
Participants	OG0000 OG0010 OG0020

Units	Counts
Participants	OG0000 OG0010 OG0020

Participants with achondroplasia who completed study C4181005 (NCT04638153) received recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.