Brief Title
Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Official Title
Multicenter, Prospective, Double-Blinded, Parallel Group, Randomized Phase III Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Brief Summary
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
CIDP Improvement
Secondary Outcome
CIDP Relapse
Condition
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
Intervention
Panzyga
Study Arms / Comparison Groups
Panzyga High Dose
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
30
Start Date
August 1, 2022
Completion Date
June 2025
Primary Completion Date
June 2025
Eligibility Criteria
Inclusion Criteria: 1. Age ≥2 years and ≤17 years. 2. Patients with a diagnosis of definite or probable CIDP based on European Neuromuscular Center (ENMC) criteria. 3. Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5. 4. Voluntarily given written informed consent (provided by patient's parent or legal guardian) or assent (provided by patient, if age-appropriate per Independent Ethics Committee [IEC]/Institutional Research Board [IRB] requirements). Exclusion Criteria: 1. Patients with previously diagnosed CIDP who lack any CIDP symptoms. 2. Patients with a known history of inherited neuropathy or a family history of inherited neuropathy. 3. Patients who have previously failed immunoglobulin therapy for CIDP. 4. Patients who received immunoglobulin within 8 weeks prior to the Baseline visit (washout phase). However, if a patient has clinical evidence of confirmed CIDP relapse during the washout phase (consistent with an increase in mRS of ≥1), they are eligible for trial enrolment. 5. Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever. 6. Patients on unstable (change in prescribed dose within the last 8 weeks) corticosteroids or rituximab use. 7. Patients with known or suspected hypersensitivity, anaphylaxis or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA. 8. Female patients who are breastfeeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method while on the study (acceptable methods of birth control for this study include: intrauterine device [IUD], hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap). 9. Known human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections. 10. Severe liver and/or kidney disease (alanine aminotransferase [ALT] > 3 × upper limit of normal [ULN]; aspartate aminotransferase [AST] > 3 × ULN; and/or creatinine levels >44 μmol/L for children ages 2-3 years, >62 μmol/L for children ages 4-10, and >89 μmol/L for children ages 11-17 years. 11. Known immunoglobulin (IgA) deficiency and antibodies against IgA. 12. History of alcohol or drug abuse in the previous year, as per Investigator's opin-ion. 13. Unable or unwilling to comply with the study protocol. 14. Receipt of any other investigational medicinal product (IMP) within 3 months be-fore study entry. 15. Any other condition(s) that, in the Investigator's opinion, makes it undesirable for the patient to participate in the study or may interfere with protocol compliance.
Gender
All
Ages
2 Years - 17 Years
Accepts Healthy Volunteers
No
Contacts
, 866-337-1868, [email protected]
Administrative Informations
NCT ID
NCT04929236
Organization ID
NGAM-11
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
, ,
Verification Date
January 2022