RECRUITING

Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

Conditions

Pediatric Chronic Inflammatory Demyelinating Polyneuropathy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2023-10-01

Study Completion:2026-06

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04929236

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
1. Age ≥2 years and ≤17 years. 2. Patients with a diagnosis of definite or possible 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) and assent (provided by the patient, if age appropriate per Independent Ethics Committee \[IEC\]/Institutional Research Board \[IRB\] requirements).	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. 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.

Inclusion Criteria

Exclusion Criteria

1. Age ≥2 years and ≤17 years.
2. Patients with a diagnosis of definite or possible 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) and assent (provided by the patient, if age appropriate per Independent Ethics Committee \[IEC\]/Institutional Research Board \[IRB\] requirements).

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.
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.

Contacts and Locations

Study Contact

Patrick Murphy

CONTACT

866-337-1868

ctgov@clinicalresearchmgt.com

Study Locations (Sites)

Octapharma Research Site

Birmingham, Alabama, 35233

United States

Octapharma Research Site

Orange, California, 92868

United States

Octapharma Research Site

Louisville, Kentucky, 40202

United States

Octapharma Research Site

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Octapharma

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-01

Study Completion Date2026-06

Study Record Updates

Study Start Date2023-10-01

Study Completion Date2026-06

Terms related to this study

Additional Relevant MeSH Terms

Pediatric Chronic Inflammatory Demyelinating Polyneuropathy