Recruiting NCT04929236
Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Panzyga.
- Who it may be relevant to
- Registry conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy. Basic parameters: 2 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Interventions
- Drug Panzyga
PANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.
Primary outcome measures
- Change in CIDP Baseline [Time frame: Up to 24 weeks]
Secondary outcome measures (3)
- CIDP Relapse [Time frame: Up to 24 weeks]
- Time to CIDP Relapse [Time frame: Up to 24 weeks]
- Percentage of Patients With Good/Excellent Response [Time frame: Up to 24 weeks]
Eligibility criteria
Inclusion criteria
- Age ≥2 years and ≤17 years.
- Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone \[1\]
- Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
- Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by the patient, if age appropriate per Independent Ethics Committee \[IEC\]/Institutional Research Board \[IRB\] requirements).
Exclusion criteria
- Patients with previously diagnosed CIDP who lack any CIDP symptoms.
- Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
- Patients who have previously failed immunoglobulin therapy for CIDP.
- Patients who received immunoglobulin or plasma exchange (PEX) within eight 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.
- Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
- Patients on unstable (change in prescribed dose within the last eight weeks) corticosteroids or rituximab use.
- Patients with known or suspected hypersensitivity, anaphylaxis, or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
- 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).
- Presence of medical history information or clinical symptoms suggestive of human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.
- 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 years, and >89 µmol/L for children ages 11-17 years.
- Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
- History of alcohol or drug abuse in the previous year, per Investigator's opinion.
- Unable or unwilling to comply with the study protocol.
- Receipt of any other investigational medicinal product (IMP) within three months before study entry or participating in another interventional clinical study. Prior participation in an observational or open-label study involving an approved product may be allowed but require prior consultation with the Medical Monitor to assess eligibilty.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- Octapharma Research Site — Birmingham
- Octapharma Research Site — Orange
- Octapharma Research Site — Louisville
- Octapharma Research Site — Philadelphia
- Octapharma Research Site — Houston
- Octapharma Research Site — Charlottesville
Identifiers
NCT: NCT04929236 · NGAM-11