Two Dose Levels of Privigen in Pediatric CIDP

Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP

A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed to IVIG treatment

Study Overview

• Status: Recruiting
• Conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
• Intervention / Treatment: Biological: IgPro10

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Trial Registration Coordinator; Phone Number: 610-878-4000; Email: clinicaltrials@cslbehring.com

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Completed
      • Phoenix Children's Hospital
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital of Los Angeles
      • Contact: Use Central Contact
  • Iowa
    • Iowa City, Iowa, United States, 52242-1009
      • Recruiting
      • University of Iowa Hospitals and Clinics
      • Contact: Use Central Contact
  • Ohio
    • Akron, Ohio, United States, 44647
      • Recruiting
      • Akron Children's Hospital
      • Contact: Use Central Contact
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Contact: Use Central Contact
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Withdrawn
      • Le Bonheur Children's Hospital
  • Texas
    • Denton, Texas, United States, 76208
      • Recruiting
      • Neurology Rare Disease Center
      • Contact: Use Central Contact
  • Virginia
    • Norfolk, Virginia, United States, 23507
      • Recruiting
      • Children's Specialty Group
      • Contact: Use Central Contact

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female subjects 2 to ≤ 17 years of age with confirmed or possible CIDP.

Exclusion Criteria:

• Absence of CIDP symptoms
• History or family history of inherited neuropathy
• Diagnosed developmental delay or regression
• History of thrombotic episode
• Known or suspected hypersensitivity to Privigen
• Known allergic or other severe reactions to blood products
• Female subject of childbearing potential either not using or not willing to use a medically reliable method of contraception or not sexually abstinent during the study
• Pregnant or breastfeeding mother"

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IgPro10 (dose level 1)	Biological: IgPro10; Normal human immunoglobulin G administered intravenously; Other Names: Privigen
Experimental: IgPro10 (dose level 2)	Biological: IgPro10; Normal human immunoglobulin G administered intravenously; Other Names: Privigen

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage (%) of subjects with CIDP relapse in the Randomized Phase by dose level	CIDP relapse, defined as a clinical decline relative to the previous assessment as indicated by an increase in modified Rankin Scale (mRS) of ≥ 1 point, in the Randomized Phase	Approximately 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of subjects with treatment emergent adverse events (TEAEs) by dose level		Approximately 56 weeks
Rate of TEAEs per infusion		Approximately 56 weeks
Rate of mild, moderate, and severe TEAEs per infusion by dose level		Approximately 56 weeks
Percentage of subjects with serious TEAEs		Approximately 56 weeks
Rate of serious TEAEs per infusion		Approximately 56 weeks
Percentage of subjects with related TEAEs		Approximately 56 weeks
Rate of related TEAEs per infusion		Approximately 56 weeks
Percentage of subjects with CIDP relapse in the Dose Exploration Phase by dose level assigned in the Randomized Phase		Approximately 24 weeks
Change in modified Rankin Scale (mRS) score from baseline in the Randomized Phase	The mRS is a disability scale ranging from 0 (asymptomatic) to 6 (death)	Baseline and Approximately 24 weeks
Percentage (%) of subjects with CIDP improvement in the Randomization Phase by dose level	CIDP improvement in the Randomized Phase, defined as a decrease in mRS score ≥ 1 from previous visit	Approximately 24 weeks
Percentage (%) of subjects with CIDP recovery in the Randomization Phase by dose level	CIDP recovery in the Randomized Phase, defined as decrease in mRS score as comparedto baseline AND mRS score of 1 or 0 at end of Randomized Phase	Approximately 24 weeks
Time to CIDP relapse in Randomized Phase by dose level		Approximately 24 weeks
Percentage (%) of subjects with CIDP improvement in the Dose Exploration Phase (DEP) by dose level	CIDP improvement in the Dose Exploration Phase, defined as decrease in mRS score ≥ 1 from baseline	Approximately 24 weeks
Percentage (%) of subjects with CIDP recovery in the Dose Exploration Phase by dose level	CIDP recovery in the Dose Exploration Phase, defined as decrease in mRS score compared to baseline AND mRS score of 1 or 0 at end of DEP	Approximately 24 weeks
Time to CIDP Relapse in the Dose Exploration Phase by dose level		Approximately 24 weeks

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Director, CSL Behring

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2019
• Primary Completion (Estimated): December 20, 2029
• Study Completion (Estimated): December 20, 2029

Study Registration Dates

• First Submitted: September 24, 2018
• First Submitted That Met QC Criteria: September 24, 2018
• First Posted (Actual): September 25, 2018

Study Record Updates

• Last Update Posted (Actual): April 3, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease Attributes; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyradiculoneuropathy; Chronic Disease; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins, Intravenous
• Other Study ID Numbers: IgPro10_4002; 2018-003430-33 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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