A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects

A Phase I, First-in-human, Two-part, Randomized, Placebo-controlled, Double-blind, Single and Repeated Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Subcutaneous Doses of SAR442501 in Healthy Adult Subjects

The purpose of the first-in-human (FIH) study is to obtain safety, tolerability, and pharmacokinetic information on SAR442501 in a healthy adult volunteer population using an integrated single ascending dose (SAD)-multiple ascending dose (MAD) parallel cohort study design.

Study Overview

• Status: Completed
• Conditions: Osteochondrodysplasia
• Intervention / Treatment: Drug: Placebo; Drug: SAR442501

• Study Type: Interventional
• Enrollment (Actual): 76
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Newark, New Jersey, United States, 07103
      • Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Male and female participants, between 18 and 45 years of age, inclusive.
• Body weight between 50.0 and 85.0 kg, inclusive, body mass index between 18.0 and 32.0 kg/m2, inclusive.
• Certified as healthy by a comprehensive clinical assessment.
• Having given written informed consent prior to undertaking any study-related procedure.

Exclusion Criteria:

• Any history or presence of clinically relevant medical status as per the protocol.
• Any participant who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development.

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Subcutaneous injection
Experimental: SAR442501	Drug: SAR442501; Subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of participants with adverse events/treatment-emergent adverse events/adverse events of special interest	Baseline up to end of study (EOS) (Day 89)

Secondary Outcome Measures

Outcome Measure	Time Frame
Assessment of PK parameter: Maximum observed concentration (Cmax)	Baseline up to EOS (Day 89)
Assessment of PK parameter: First time to reach Cmax (tmax)	Baseline up to EOS (Day 89)
Assessment of PK parameter: Partial area under the serum concentration time curve (AUC)	Baseline up to EOS (Day 89)
Immunogenicity: evaluate the presence of anti-SAR442501 antibodies	Baseline up to EOS (Day 89)

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2020
• Primary Completion (Actual): January 12, 2022
• Study Completion (Actual): January 12, 2022

Study Registration Dates

• First Submitted: April 26, 2023
• First Submitted That Met QC Criteria: April 26, 2023
• First Posted (Actual): May 6, 2023

Study Record Updates

• Last Update Posted (Actual): January 9, 2024
• Last Update Submitted That Met QC Criteria: January 5, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Connective Tissue Diseases; Bone Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Bone Diseases, Developmental; Mucopolysaccharidoses; Osteochondrodysplasias; Mucopolysaccharidosis IV
• Other Study ID Numbers: TDU16639/TDR16640

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No