Chronic inflammatory demyelinating polyradiculoneuropathy
Conditions
Interventions
Trade Name: Panzyga
Product Name: Panzyga
Pharmaceutical Form:
INN or Proposed INN: Panzyga
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: % percent
Concentration type:
Sponsors
Octapharma
Eligibility
Sex/Gender
All
Inclusion 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). Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 opinion. 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of 2 PANZYGA dose regimens in pediatric CIDP patients based on the percentage of patients with CIDP improvement;Secondary Objective: - To evaluate the efficacy of 2 PANZYGA dose regimens by determining the percentage of patients with CIDP relapse. - To evaluate the safety of PANZYGA (occurrence of treatment-emergent adverse events [TEAEs] and infusion related TEAES). - To further evaluate the efficacy of PANZYGA (including time to improvement and time to relapse and subgroup analyses). - To assess the effect of PANZYGA on the modified Rankin scale (mRS).;Primary end point(s): The primary efficacy endpoint of this study is the percentage (%) of patients with CIDP improvement at Week 24. CIDP improvement is defined as the following: Decrease in mRS of =1 point from the most recent score;Timepoint(s) of evaluation of this end point: Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary efficacy endpoints of this study are: - The percentage (%) of patients with CIDP relapse. CIDP relapse is defined as an increase in mRS by >1 point from most recent score. - Time to CIDP relapse or withdrawal for any other reason. - The percentage of patients with excellent response, defined by a mRS score of 0 or 1 in each arm at Week 24. The secondary safety endpoints of this study are: - Occurrence of all TEAEs. - Percentage of patients with TEAEs, serious TEAEs, and related TEAEs. - Rate of TEAEs, serious TEAEs, and related TEAEs per infusion. - Rate of mild, moderate, and severe TEAEs per infusion - Short-term tolerance variables, including vital signs. - Safety laboratory variables;Timepoint(s) of evaluation of this end point: 24 weeks | — |
Countries
United States
Contacts
Public ContactClinical Trials Information
Octapharma
Outcome results
None listed