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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05116046
Registration number
NCT05116046
Ethics application status
Date submitted
18/10/2021
Date registered
10/11/2021
Titles & IDs
Public title
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
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Scientific title
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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Secondary ID [1]
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2021-003149-39
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Secondary ID [2]
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C4181008
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Achondroplasia
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - Recifercept
Experimental: Low Dose - Low Dose
Experimental: Medium Dose - Medium Dose
Experimental: High Dose - High Dose
Treatment: Other: Recifercept
Recifercept
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Intervention code [1]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs
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Assessment method [1]
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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.
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Timepoint [1]
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From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months)
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Primary outcome [2]
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Change From Baseline in Height at Month 24
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline, Month 24
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Secondary outcome [1]
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Clearance (CL/F) of Recifercept
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Assessment method [1]
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Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes.
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Timepoint [1]
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Predose on Day 91, 181, 271, 361 and 451.
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Secondary outcome [2]
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Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline and Months 3, 6, 9
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Secondary outcome [3]
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Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline and Months 3, 6, 9
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Secondary outcome [4]
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Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9
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Assessment method [4]
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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
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Timepoint [4]
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Baseline and Months 3, 6, 9
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Secondary outcome [5]
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Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9
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Assessment method [5]
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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).
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Timepoint [5]
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Baseline and Months 3, 6, 9
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Secondary outcome [6]
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Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline, Months 3, 6, 9
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Secondary outcome [7]
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Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9
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Assessment method [7]
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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.
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Timepoint [7]
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Baseline, Months 3, 6, 9
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Secondary outcome [8]
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Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline, Months 3, 6, 9
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Secondary outcome [9]
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Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9
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Assessment method [9]
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Timepoint [9]
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Baseline, Months 3, 6, 9
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Secondary outcome [10]
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Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9
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Assessment method [10]
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Timepoint [10]
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Baseline, Months 3, 6, 9
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Secondary outcome [11]
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Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study
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Assessment method [11]
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Laboratory parameters that were assessed included lymphocytes, neutrophils, eosinophils, monocytes and potassium. Clinically significant abnormal laboratory findings were determined based on investigator's decision.
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Timepoint [11]
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From baseline up to end of study/early termination (for a maximum duration of 11 months)
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Secondary outcome [12]
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Number of Participants With Clinically Significant Findings in Vital Signs Through the Study
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Assessment method [12]
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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.
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Timepoint [12]
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From baseline up to end of study/early termination (for a maximum duration of 11 months)
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Secondary outcome [13]
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Number of Participants With Clinically Significant Findings in Physical Examination Through the Study
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Assessment method [13]
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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.
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Timepoint [13]
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From baseline up to end of study/early termination (for a maximum duration of 11 months)
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Secondary outcome [14]
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Number of Participants With Positive Anti-Drug Antibodies (ADA)
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Assessment method [14]
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Timepoint [14]
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From Month 3 up to end of study/early termination (up to Month 11)
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Eligibility
Key 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).
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Minimum age
15
Months
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Maximum age
12
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/12/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
30/03/2023
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Sample size
Target
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Accrual to date
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Final
35
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Delaware
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Country [3]
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United States of America
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State/province [3]
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Texas
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Country [4]
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Belgium
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State/province [4]
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Vlaams - Gewest
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Country [5]
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Belgium
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State/province [5]
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Edegem
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Country [6]
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Denmark
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State/province [6]
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Copenhagen
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Country [7]
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Denmark
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State/province [7]
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Kobenhavn
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Country [8]
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Italy
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State/province [8]
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Roma
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Country [9]
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Portugal
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State/province [9]
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Coimbra
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Country [10]
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Spain
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State/province [10]
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Alava
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT05116046
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Pfizer CT.gov Call Center
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Address
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Pfizer
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/46/NCT05116046/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/46/NCT05116046/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05116046