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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06067425




Registration number
NCT06067425
Ethics application status
Date submitted
20/09/2023
Date registered
4/10/2023

Titles & IDs
Public title
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
Scientific title
A Phase 2, Open-label, Multi-center, 2-stage Sequential Cohort, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Subcutaneous SAR442501 in Pediatric Participants With Achondroplasia
Secondary ID [1] 0 0
U1111-1280-5374
Secondary ID [2] 0 0
DRI16646
Universal Trial Number (UTN)
Trial acronym
upreACH-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Osteochondrodysplasia 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
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SAR442501

Experimental: Cohort 1 -

Experimental: Cohort 2 -

Experimental: Cohort 3 -


Treatment: Drugs: SAR442501
Solution for injection; Subcutaneous injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent period
Timepoint [1] 0 0
Baseline to Week 52
Secondary outcome [1] 0 0
Change in annualized growth velocity (AGV) Zscore
Timepoint [1] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [2] 0 0
Change in AGV (cm/year)
Timepoint [2] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [3] 0 0
Change in height Z score
Timepoint [3] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [4] 0 0
Change in upper-to-lower body segment ratio
Timepoint [4] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [5] 0 0
Change in upper to lower extremity ratio
Timepoint [5] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [6] 0 0
Change in sitting to standing height ratio (crown-to-rump length to total length for infants)
Timepoint [6] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [7] 0 0
Change in arm span to height ratio
Timepoint [7] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [8] 0 0
Change in upper arm to forearm length ratio
Timepoint [8] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [9] 0 0
Change in upper leg to lower leg ratio
Timepoint [9] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [10] 0 0
Change in head circumference to height ratio
Timepoint [10] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [11] 0 0
Change in brainstem parameter
Timepoint [11] 0 0
Baseline to Week 52
Secondary outcome [12] 0 0
Change in skull parameter
Timepoint [12] 0 0
Baseline to Week 52
Secondary outcome [13] 0 0
Change in spine morphometric parameter
Timepoint [13] 0 0
Baseline to Week 52
Secondary outcome [14] 0 0
Change in volumetric parameter
Timepoint [14] 0 0
Baseline to Week 52
Secondary outcome [15] 0 0
Change in overall health-related quality of life score in the PedsQL Inventory Generic Core Scale
Timepoint [15] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [16] 0 0
Change in fatigue score in the PedsQL Multidimensional Fatigue Scale
Timepoint [16] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [17] 0 0
Change in present pain and worst pain rating (PPQ) score
Timepoint [17] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [18] 0 0
Change in mobility and symptom rating (STEMS) score
Timepoint [18] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [19] 0 0
Change in developmental score in the Achondroplasia Developmental Recording Form
Timepoint [19] 0 0
Baseline to Week 52
Secondary outcome [20] 0 0
Assessment of pharmacokinetic (PK) parameter: plasma concentration of SAR442501
Timepoint [20] 0 0
Baseline to Week 26 and 52
Secondary outcome [21] 0 0
Assessment of PK parameter: maximum plasma concentration observed (Cmax)
Timepoint [21] 0 0
Baseline to Week 26 and 52
Secondary outcome [22] 0 0
Assessment of PK parameter: time to reach Cmax (Tmax)
Timepoint [22] 0 0
Baseline to Week 26 and 52
Secondary outcome [23] 0 0
Assessment of PK parameter: Area under the plasma concentration versus time curve calculated using the trapezoidal method during a dose interval (AUC0-t)
Timepoint [23] 0 0
Baseline to Week 26 and 52
Secondary outcome [24] 0 0
Assessment of PK parameter: concentration observed before treatment administration during repeated dosing (Ctrough)
Timepoint [24] 0 0
Baseline to Week 26 and 52
Secondary outcome [25] 0 0
Assessment of pharmacodynamics (PD) parameter: change in collagen X biomarker (CXM) levels
Timepoint [25] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [26] 0 0
Assessment of PD parameter: change in osteocalcin levels
Timepoint [26] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [27] 0 0
Assessment of PD parameter: change in bone-specific alkaline phosphatase
Timepoint [27] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [28] 0 0
Assessment of PD parameter: change in procollagen type 1 N-terminal propeptide (P1NP) levels
Timepoint [28] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [29] 0 0
Assessment of PD parameter: change in collagen-type 1 C-Telopeptide (CTX) levels
Timepoint [29] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [30] 0 0
Number of participants with treatment-emergent anti-drug antibodies (ADA)
Timepoint [30] 0 0
Baseline to Week 26 and Week 52
Secondary outcome [31] 0 0
Changes in neurological examination
Timepoint [31] 0 0
Baseline through Week 26 and Week 52

Eligibility
Key inclusion criteria
* Participants must have ACH with a confirmed mutation in the FGFR3 gene
* Participants and/or parent(s) or legal representative(s) must be willing and able to perform all the study procedures to the best of their physical ability.
* Parent(s) or legal representative(s) capable of giving signed informed consent and participants capable of giving assent when applicable.
Minimum age
0 Days
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Have hypochondroplasia (or the N540K mutation) or short stature condition other than ACH (eg, trisomy 21, pseudochondroplasia)
* Participants have received any dose of medications or investigational product, including human growth hormone, IGF-1, intended to affect participants' stature or body proportions between the completion of OBS16647 and enrollment (Week 0/Day 1/Visit 2).
* Have a history of growth plate closure.
* Long bone fracture within 3 months of enrollment (Week 0/Day 1/Visit 2)
* Current evidence of corneal or retinal disorder/keratopathy.
* Participants have had a previous surgical intervention involving the foramen magnum (Stage 2 only).
* Hyperphosphatemia.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Study design
Purpose of the study
Treatment
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
Open (masking not used)
Who is / are masked / blinded?



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

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
2/12/2027
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Investigational Site Number : 0360001 - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
China
State/province [1] 0 0
Shanghai
Country [2] 0 0
China
State/province [2] 0 0
Wuhan
Country [3] 0 0
Italy
State/province [3] 0 0
Lombardia
Country [4] 0 0
Italy
State/province [4] 0 0
Roma
Country [5] 0 0
Korea, Republic of
State/province [5] 0 0
Seoul-teukbyeolsi
Country [6] 0 0
Spain
State/province [6] 0 0
Catalunya [Cataluña]
Country [7] 0 0
Spain
State/province [7] 0 0
Pais Vasco

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
Contact-US@sanofi.com
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?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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?


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.


Results not provided in https://clinicaltrials.gov/study/NCT06067425