Clinical trial NCT03684018: Two Dose Levels of Privigen in Pediatric CIDP
Official title: Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2018-09-25
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: 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"

Primary outcomes:
measure: Percentage (%) of subjects with CIDP relapse in the Randomized Phase by dose level
timeFrame: Approximately 24 weeks
Secondary outcomes:
measure: Percentage of subjects with treatment emergent adverse events (TEAEs) by dose level
timeFrame: Approximately 56 weeks
measure: Rate of TEAEs per infusion
timeFrame: Approximately 56 weeks
measure: Rate of mild, moderate, and severe TEAEs per infusion by dose level
timeFrame: Approximately 56 weeks
measure: Percentage of subjects with serious TEAEs
timeFrame: Approximately 56 weeks
measure: Rate of serious TEAEs per infusion
timeFrame: Approximately 56 weeks
measure: Percentage of subjects with related TEAEs
timeFrame: Approximately 56 weeks
measure: Rate of related TEAEs per infusion
timeFrame: Approximately 56 weeks
measure: Percentage of subjects with CIDP relapse in the Dose Exploration Phase by dose level assigned in the Randomized Phase
timeFrame: Approximately 24 weeks
measure: Change in modified Rankin Scale (mRS) score from baseline in the Randomized Phase
timeFrame: Baseline and Approximately 24 weeks
measure: Percentage (%) of subjects with CIDP improvement in the Randomization Phase by dose level
timeFrame: Approximately 24 weeks
measure: Percentage (%) of subjects with CIDP recovery in the Randomization Phase by dose level
timeFrame: Approximately 24 weeks
measure: Time to CIDP relapse in Randomized Phase by dose level
timeFrame: Approximately 24 weeks
measure: Percentage (%) of subjects with CIDP improvement in the Dose Exploration Phase (DEP) by dose level
timeFrame: Approximately 24 weeks
measure: Percentage (%) of subjects with CIDP recovery in the Dose Exploration Phase by dose level
timeFrame: Approximately 24 weeks
measure: Time to CIDP Relapse in the Dose Exploration Phase by dose level
timeFrame: Approximately 24 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: Le Bonheur Children's Hospital
state: Tennessee
city: Denton
country: United States
facility: Neurology Rare Disease Center
state: Texas
city: Norfolk
country: United States
facility: Children's Specialty Group
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: