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

• Sponsor: Octapharma (Industry)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT04929236
• Collaborator: (none)
• Enrollment: 30
• Arms: 2
• Anticipated Duration (Months): 46

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy	Drug: Panzyga	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 30 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Masking Description: Double-Blinded
• Primary Purpose: Treatment
• 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
• Anticipated Study Start Date: Aug 1, 2022
• Anticipated Primary Completion Date: Dec 1, 2025
• Anticipated Study Completion Date: Jun 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Panzyga High Dose	Drug: Panzyga; Intravenous immunoglobulin formulation
Experimental: Panzyga Low Dose	Drug: Panzyga; Intravenous immunoglobulin formulation

Outcome Measures

Primary Outcome Measures

1. CIDP Improvement [Up to 24 weeks]

   Evaluate percentage of patients with CIDP improvement between 2 doses of Panzyga

Secondary Outcome Measures

1. CIDP Relapse [Up to 24 weeks]

   Evaluate percentage of patients with CIDP relapse between 2 doses of Panzyga

2. Treatment Emergent Adverse Event Occurrence [Up to 24 weeks]

   Treatment Emergent Adverse Event Occurrence

3. Time to improvement of CIDP [Up to 24 weeks]

   Time to improvement of CIDP

4. Time to relapse of CIDP [Up to 24 weeks]

   Time to relapse of CIDP

5. Modified Rankin Scale [Up to 24 weeks]

   Effect of Panzyga on modified Rankin Scale Measure of a patient's level of disability assessed on a scale of 0 (i.e., no symptoms) to 6 (death). Higher number associated with worse outcome.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

1. Known immunoglobulin (IgA) deficiency and antibodies against IgA.

2. History of alcohol or drug abuse in the previous year, as per Investigator's opin-ion.

3. Unable or unwilling to comply with the study protocol.

4. Receipt of any other investigational medicinal product (IMP) within 3 months be-fore study entry.

5. 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

Locations

No locations specified.

Sponsors and Collaborators

• Octapharma

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Octapharma
• ClinicalTrials.gov Identifier: NCT04929236
• Other Study ID Numbers: NGAM-11
• First Posted: Jun 18, 2021
• Last Update Posted: Jun 3, 2022
• Last Verified: Jun 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Polyradiculoneuropathy

Study Results

No Results Posted as of Jun 3, 2022