Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Clinical Trial
Official title:
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
| Status | Recruiting |
| Enrollment | 30 |
| Est. completion date | December 20, 2029 |
| Est. primary completion date | December 20, 2029 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years to 17 Years |
| Eligibility | 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" |
| Country | Name | City | State |
|---|---|---|---|
| United States | Akron Children's Hospital | Akron | Ohio |
| United States | Neurology Rare Disease Center | Denton | Texas |
| United States | University of Iowa Hospitals and Clinics | Iowa City | Iowa |
| United States | Children's Hospital of Los Angeles | Los Angeles | California |
| United States | Le Bonheur Children's Hospital | Memphis | Tennessee |
| United States | Children's Specialty Group | Norfolk | Virginia |
| United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Phoenix Children's Hospital | Phoenix | Arizona |
| Lead Sponsor | Collaborator |
|---|---|
| CSL Behring |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | Percentage of subjects with treatment emergent adverse events (TEAEs) by dose level | Approximately 56 weeks | ||
| Secondary | Rate of TEAEs per infusion | Approximately 56 weeks | ||
| Secondary | Rate of mild, moderate, and severe TEAEs per infusion by dose level | Approximately 56 weeks | ||
| Secondary | Percentage of subjects with serious TEAEs | Approximately 56 weeks | ||
| Secondary | Rate of serious TEAEs per infusion | Approximately 56 weeks | ||
| Secondary | Percentage of subjects with related TEAEs | Approximately 56 weeks | ||
| Secondary | Rate of related TEAEs per infusion | Approximately 56 weeks | ||
| Secondary | Percentage of subjects with CIDP relapse in the Dose Exploration Phase by dose level assigned in the Randomized Phase | Approximately 24 weeks | ||
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | Time to CIDP relapse in Randomized Phase by dose level | Approximately 24 weeks | ||
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | Time to CIDP Relapse in the Dose Exploration Phase by dose level | Approximately 24 weeks |