Clinical trial NCT04929236: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2021-06-18
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: Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients


Eligibility criteria: Inclusion Criteria:

1. Age ≥2 years and ≤17 years.
2. Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone \[1\]
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) and assent (provided by the 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 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.
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 eight 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. 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.
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 years, and \>89 µmol/L for children ages 11-17 years.
11. Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
12. History of alcohol or drug abuse in the previous year, per Investigator's opinion.
13. Unable or unwilling to comply with the study protocol.
14. 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.
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.

Primary outcomes:
measure: Change in CIDP Baseline
timeFrame: Up to 24 weeks
Secondary outcomes:
measure: CIDP Relapse
timeFrame: Up to 24 weeks
measure: Time to CIDP Relapse
timeFrame: Up to 24 weeks
measure: Percentage of Patients With Good/Excellent Response
timeFrame: Up to 24 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Octapharma Research Site
state: Alabama
city: Orange
country: United States
facility: Octapharma Research Site
state: California
city: Louisville
country: United States
facility: Octapharma Research Site
state: Kentucky
city: Philadelphia
country: United States
facility: Octapharma Research Site
state: Pennsylvania
city: Houston
country: United States
facility: Octapharma Research Site
state: Texas
city: Charlottesville
country: United States
facility: Octapharma Research Site
state: Virginia
Study officials:
References: