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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04638153




Registration number
NCT04638153
Ethics application status
Date submitted
15/09/2020
Date registered
20/11/2020
Date last updated
15/02/2024

Titles & IDs
Public title
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Scientific title
A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
Secondary ID [1] 0 0
2020-001189-13
Secondary ID [2] 0 0
C4181005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
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
Other interventions - Recifercept

Experimental: Low Dose - Low Dose

Experimental: Medium Dose - Medium Dose

Experimental: High Dose - High Dose

Experimental: PK Phase 2 Formulation - Phase 2 formulation [process 1c] 3mg/kg

Experimental: PK Phase 3 Formulation - Phase 3 formulation [process 2] 3mg/kg


Other interventions: Recifercept
Recifercept

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs)
Timepoint [1] 0 0
The first dose up to 28 to 35 days after the last dose of study intervention (13 months)
Primary outcome [2] 0 0
Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12
Timepoint [2] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [1] 0 0
Change From Baseline in Pulse Rate at Month 3, Month 6, Month 9, and Month 12
Timepoint [1] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [2] 0 0
Change From Baseline in Respiratory Rate at Month 3, Month 6, Month 9, and Month 12
Timepoint [2] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [3] 0 0
Change From Baseline in Blood Pressure at Month 3, Month 6, Month 9, and Month 12
Timepoint [3] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [4] 0 0
Change From Baseline in Temperature at Month 3, Month 6, Month 9, and Month 12
Timepoint [4] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [5] 0 0
Number of Participants With Abnormal Physical Examination Findings at Month 3, Month 6, Month 9, and Month 12
Timepoint [5] 0 0
Month 3, Month 6, Month 9, and Month 12
Secondary outcome [6] 0 0
Number of Participants With Laboratory Abnormalities (Without Regard to Baseline Abnormality)
Timepoint [6] 0 0
Baseline to Month 12
Secondary outcome [7] 0 0
Pre-Dose Serum Concentration (Ctrough) of Recifercept
Timepoint [7] 0 0
Pre-dose on Day(s) 4, 8, 15, 29, 61, 91, 183, 273, 365
Secondary outcome [8] 0 0
Number of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibody (NAb) of Recifercept
Timepoint [8] 0 0
The first dose up to 28 to 35 days after the last dose of study intervention (13 months)
Secondary outcome [9] 0 0
Change From Baseline in Sitting/Standing Height Ratio at Month 3, Month 6, Month 9, and Month 12
Timepoint [9] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [10] 0 0
Least Square Mean of Change From Baseline Arm Span to Standing Height/Length Difference at Month 3, Month 6, Month 9, and Month 12
Timepoint [10] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [11] 0 0
Change From Baseline in Knee Height : Lower Segment Ratio at Month 3, Month 6, Month 9, and Month 12
Timepoint [11] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [12] 0 0
Change From Baseline in Occipito-Frontal Circumference at Month 3, Month 6, Month 9, and Month 12
Timepoint [12] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [13] 0 0
Change From Baseline in Occipito-Frontal to Occipito-Mid-Face Ratio at Month 3, Month 6, Month 9, and Month 12
Timepoint [13] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [14] 0 0
Change From Baseline in Height Standard Deviation Score (Z-Score) at Month 3, Month 6, Month 9, and Month 12
Timepoint [14] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [15] 0 0
Change From Baseline in Fixed Flexion Angles at Elbow at Month 3, Month 6, Month 9, and Month 12
Timepoint [15] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [16] 0 0
Change From Baseline in Body Mass Index (BMI) at Month 3, Month 6, Month 9, and Month 12
Timepoint [16] 0 0
Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcome [17] 0 0
Change From Baseline in Waist : Chest Circumference Ratio at Month 9 and Month 12
Timepoint [17] 0 0
Baseline, Month 9, and Month 12
Secondary outcome [18] 0 0
Change From Baseline in Apnea-Hypopnea Index (AHI) at Month 12
Timepoint [18] 0 0
Baseline and Month 12
Secondary outcome [19] 0 0
Change From Baseline in Desaturation Index at Month 12
Timepoint [19] 0 0
Baseline and Month 12
Secondary outcome [20] 0 0
Change From Baseline in Polysomnography Other Parameters at Month 12
Timepoint [20] 0 0
Baseline and Month 12
Secondary outcome [21] 0 0
Change From Baseline in SaO2 Nadir at Month 12
Timepoint [21] 0 0
Baseline and Month 12

Eligibility
Key inclusion criteria
* Main cohort: Aged =2 years to <11 years (up to the day before 11th birthday inclusive) at time of enrollment; or exploratory cohort: aged =3 months to <2 years (up to the day before 2nd birthday inclusive) at time of enrollment
* Documented, confirmed genetic diagnosis of achondroplasia from historical medical records prior to entry into this trial (test must have been performed at a laboratory fully accredited for genetic testing under local regulations).
* Completed the C4181001 natural history study with at least 2 valid height/length measurements (at least 3 months apart) prior to enrollment in this study. One of these measurement timepoints must be within the 3 months prior to enrollment in C4181005.
* Tanner stage 1 based on investigator assessment during physical examination (must include assessment of breast development for females, testicular stage for males).
* Able to stand independently for height measurements (if =2 years of age at enrollment).
* If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.
Minimum age
3 Months
Maximum age
10 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 (BMI >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].14
* Known closure of long bone growth plates (cessation of height growth).
* Body weight <7 kg or >30 kg.
* Moderate or severe renal impairment CrCL GFR <60 mL/min/1.73m2 (Calculated GFR based on updated "bedside" Schwartz formula for pediatric patients (CrCL (mL/min/1.73 m2) = 0.413 * Height (cms)/ Serum cr (mg/dL) or hepatic impairment (AST/ALT >1.5 ULN).
* 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 beclametasone 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 investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
* History of receipt of an investigational product (not for achondroplasia/growth affecting) within the last 30 days or 5 half-lives (whichever is longer).

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Melbourne
Recruitment hospital [2] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment postcode(s) [2] 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
Edegem
Country [5] 0 0
Belgium
State/province [5] 0 0
Leuven
Country [6] 0 0
Denmark
State/province [6] 0 0
Copenhagen NV
Country [7] 0 0
Italy
State/province [7] 0 0
Roma
Country [8] 0 0
Japan
State/province [8] 0 0
Osaka
Country [9] 0 0
Japan
State/province [9] 0 0
Okayama
Country [10] 0 0
Portugal
State/province [10] 0 0
Coimbra
Country [11] 0 0
Spain
State/province [11] 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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.