Two Dose Levels of Privigen in Pediatric CIDP

Study Overview
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Brief Summary
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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

Official Title
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Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP

Conditions
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Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Intervention / Treatment
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* Biological: IgPro10


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IgPro10 (dose level 1)<br> | Biological: IgPro10<br>* Normal human immunoglobulin G administered intravenously<br>* Other names: Privigen;|
| Experimental: IgPro10 (dose level 2)<br> | Biological: IgPro10<br>* Normal human immunoglobulin G administered intravenously<br>* Other names: Privigen;|

What is the study measuring?
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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 |