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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
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
Secondary ID [1] 0 0
2021-003149-39
Secondary ID [2] 0 0
C4181008
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs
Timepoint [1] 0 0
From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months)
Primary outcome [2] 0 0
Change From Baseline in Height at Month 24
Timepoint [2] 0 0
Baseline, Month 24
Secondary outcome [1] 0 0
Clearance (CL/F) of Recifercept
Timepoint [1] 0 0
Predose on Day 91, 181, 271, 361 and 451.
Secondary outcome [2] 0 0
Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9
Timepoint [2] 0 0
Baseline and Months 3, 6, 9
Secondary outcome [3] 0 0
Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9
Timepoint [3] 0 0
Baseline and Months 3, 6, 9
Secondary outcome [4] 0 0
Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9
Timepoint [4] 0 0
Baseline and Months 3, 6, 9
Secondary outcome [5] 0 0
Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9
Timepoint [5] 0 0
Baseline and Months 3, 6, 9
Secondary outcome [6] 0 0
Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9
Timepoint [6] 0 0
Baseline, Months 3, 6, 9
Secondary outcome [7] 0 0
Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9
Timepoint [7] 0 0
Baseline, Months 3, 6, 9
Secondary outcome [8] 0 0
Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9
Timepoint [8] 0 0
Baseline, Months 3, 6, 9
Secondary outcome [9] 0 0
Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9
Timepoint [9] 0 0
Baseline, Months 3, 6, 9
Secondary outcome [10] 0 0
Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9
Timepoint [10] 0 0
Baseline, Months 3, 6, 9
Secondary outcome [11] 0 0
Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study
Timepoint [11] 0 0
From baseline up to end of study/early termination (for a maximum duration of 11 months)
Secondary outcome [12] 0 0
Number of Participants With Clinically Significant Findings in Vital Signs Through the Study
Timepoint [12] 0 0
From baseline up to end of study/early termination (for a maximum duration of 11 months)
Secondary outcome [13] 0 0
Number of Participants With Clinically Significant Findings in Physical Examination Through the Study
Timepoint [13] 0 0
From baseline up to end of study/early termination (for a maximum duration of 11 months)
Secondary outcome [14] 0 0
Number of Participants With Positive Anti-Drug Antibodies (ADA)
Timepoint [14] 0 0
From Month 3 up to end of study/early termination (up to Month 11)

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).
Minimum age
15 Months
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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).

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/03/2023
Sample size
Target
Accrual to date
Final
35
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Delaware
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
Belgium
State/province [4] 0 0
Vlaams - Gewest
Country [5] 0 0
Belgium
State/province [5] 0 0
Edegem
Country [6] 0 0
Denmark
State/province [6] 0 0
Copenhagen
Country [7] 0 0
Denmark
State/province [7] 0 0
Kobenhavn
Country [8] 0 0
Italy
State/province [8] 0 0
Roma
Country [9] 0 0
Portugal
State/province [9] 0 0
Coimbra
Country [10] 0 0
Spain
State/province [10] 0 0
Alava

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT05116046