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

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, Parallel Group, Open-label, 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 Status

Drug

Estimated Enrollment

30

Start Date

September 2021

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 intravenous immunoglobulin (IVIG) 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 in-crease 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]

NCT ID

NCT04929236

Organization ID

NGAM-11

Responsible Party

Sponsor

Study Sponsor

Octapharma

Study Sponsor

, , 

Verification Date

June 2021