Achondroplasia
Conditions
Keywords
achondroplasia, dwarfism, bone diseases, development, bone diseases, Musculoskeletal Diseases, Osteochondrodysplasias, Genetic Diseases, Inborn, Skeletal Dysplasia
Brief summary
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.
Interventions
BIOLOGICALRecifercept
Recifercept
Sponsors
Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
15 Months to 12 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months) | 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. |
| Change From Baseline in Height at Month 24 | Baseline, 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9 | Baseline and 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. |
| Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9 | Baseline and 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 |
| Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9 | Baseline and 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). |
| Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9 | Baseline, 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. |
| Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9 | Baseline, 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. |
| Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9 | Baseline, 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. |
| Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 | Baseline and 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. |
| Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | — |
| Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | From baseline up to end of study/early termination (for a maximum duration of 11 months) | Laboratory parameters that were assessed included lymphocytes, neutrophils, eosinophils, monocytes and potassium. Clinically significant abnormal laboratory findings were determined based on investigator's decision. |
| Number of Participants With Clinically Significant Findings in Vital Signs Through the Study | From baseline up to end of study/early termination (for a maximum duration of 11 months) | 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. |
| Number of Participants With Clinically Significant Findings in Physical Examination Through the Study | From baseline up to end of study/early termination (for a maximum duration of 11 months) | 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. |
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | From Month 3 up to end of study/early termination (up to Month 11) | — |
| Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | — |
| Clearance (CL/F) of Recifercept | Predose on Day 91, 181, 271, 361 and 451. | Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes. |
Countries
Australia, Belgium, Denmark, Italy, Portugal, Spain, United States
Participant flow
Recruitment details
Eligible participants aged greater than or equal to (\>=)15 months to 12 years (inclusive) diagnosed with achondroplasia from study C4181005 (NCT04638153) were enrolled in this extension study.
Pre-assignment details
A total of 35 participants were enrolled and assigned to study treatment.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 16 |
| 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 |
| 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 |
| Total | 35 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Study terminated by sponsor | 14 | 16 | 1 |
| Overall Study | Withdrawal by parent/guardian | 2 | 1 | 1 |
Baseline characteristics
| Characteristic | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Recifercept 2 mg/kg Twice Weekly | Recifercept 1.5 mg/kg Once Daily | Total |
|---|---|---|---|---|
| Age, Continuous | 7.5 Years STANDARD_DEVIATION 2.66 | 6.6 Years STANDARD_DEVIATION 2.5 | 9.5 Years STANDARD_DEVIATION 0.71 | 7.2 Years STANDARD_DEVIATION 2.56 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 0 Participants | 1 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 14 Participants | 17 Participants | 1 Participants | 32 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 14 Participants | 17 Participants | 2 Participants | 33 Participants |
| Sex: Female, Male Female | 9 Participants | 9 Participants | 2 Participants | 20 Participants |
| Sex: Female, Male Male | 7 Participants | 8 Participants | 0 Participants | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 16 | 0 / 17 | 0 / 2 |
| other Total, other adverse events | 9 / 16 | 11 / 17 | 0 / 2 |
| serious Total, serious adverse events | 0 / 16 | 0 / 17 | 0 / 2 |
Outcome results
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.
Time frame: Baseline, Month 24
Population: 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.
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.
Time frame: From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months)
Population: FAS consisted of all participants who received at least one dose of recifercept. Participants were analyzed according to the dose they actually received.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | SAEs | 0 Participants |
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | AEs | 9 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | AEs | 11 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | SAEs | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | AEs | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | SAEs | 0 Participants |
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.
Time frame: Baseline and Months 3, 6, 9
Population: 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.
Secondary
Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9
Time frame: Baseline, Months 3, 6, 9
Population: 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.
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.
Time frame: Baseline, Months 3, 6, 9
Population: 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.
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
Time frame: Baseline and Months 3, 6, 9
Population: 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.
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).
Time frame: Baseline and Months 3, 6, 9
Population: 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.
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.
Time frame: Baseline, Months 3, 6, 9
Population: 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.
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.
Time frame: Baseline and Months 3, 6, 9
Population: 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.
Secondary
Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9
Time frame: Baseline, Months 3, 6, 9
Population: 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.
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.
Time frame: Baseline, Months 3, 6, 9
Population: 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.
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.
Time frame: Predose on Day 91, 181, 271, 361 and 451.
Population: 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.
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.
Time frame: From baseline up to end of study/early termination (for a maximum duration of 11 months)
Population: 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.'
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Monocytes | 2 Participants |
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Eosinophils | 1 Participants |
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Neutrophils | 0 Participants |
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Lymphocytes | 0 Participants |
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Chemistry: Potassium | 2 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Eosinophils | 3 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Neutrophils | 1 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Lymphocytes | 1 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Monocytes | 0 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Chemistry: Potassium | 1 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Chemistry: Potassium | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Monocytes | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Lymphocytes | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Eosinophils | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Hematology: Neutrophils | 0 Participants |
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.
Time frame: From baseline up to end of study/early termination (for a maximum duration of 11 months)
Population: FAS included all participants who were planned to receive at least one dose of recifercept.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Significant Findings in Physical Examination Through the Study | 0 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Significant Findings in Physical Examination Through the Study | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Significant Findings in Physical Examination Through the Study | 0 Participants |
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.
Time frame: From baseline up to end of study/early termination (for a maximum duration of 11 months)
Population: FAS included all participants who were planned to receive at least one dose of recifercept.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Clinically Significant Findings in Vital Signs Through the Study | 0 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Clinically Significant Findings in Vital Signs Through the Study | 0 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Clinically Significant Findings in Vital Signs Through the Study | 0 Participants |
Secondary
Number of Participants With Positive Anti-Drug Antibodies (ADA)
Time frame: From Month 3 up to end of study/early termination (up to Month 11)
Population: FAS included all participants who were planned to receive at least one dose of recifercept.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Number of Participants With Positive Anti-Drug Antibodies (ADA) | 12 Participants |
| Recifercept 2 mg/kg Twice Weekly | Number of Participants With Positive Anti-Drug Antibodies (ADA) | 15 Participants |
| Recifercept 1.5 mg/kg Once Daily | Number of Participants With Positive Anti-Drug Antibodies (ADA) | 2 Participants |