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A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

NCT04638153 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2024-02-15

Study results available
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Summary

Approximately 63 participants will be randomized to one of three doses to receive Recifercept either

* Low Dose
* Medium Dose
* High Dose

Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2, 3 6, 9 \& 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires

Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study.

A PK cohort will include 12 participants who will randomly receive a single dose of 3 mg/kg of Phase 2 study (process 1c) formulation and a single dose of 3 mg/kg of the proposed Phase 3 (process 2) study formulation in a cross over study. Dose of the cohort could be changed due to emerging safety and efficacy data in the study.

Conditions

Interventions

BIOLOGICAL

Recifercept

Recifercept

Sponsors & Collaborators

Principal Investigators

  • Pfizer CT.gov Call Center · Pfizer

Study Design

Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
3 Months
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-02
Primary Completion
2023-01-16
Completion
2023-03-27
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Denmark
  • Italy
  • Japan
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04638153 on ClinicalTrials.gov