Pediatric Chronic Inflammatory Demyelinating PolyneuropathyPhase 3NCT04929236
Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
A Phase 3 study testing Panzyga in people with Pediatric Chronic Inflammatory Demyelinating Polyneuropathy, run by Octapharma at 6 sites in United States.
Trial summary
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
- What is being tested?
- Panzyga
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 6 sitesUnited States
- Study dates
- 1 Oct 2023to 31 Dec 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥2 years and ≤17 years.
- Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone [1]
- Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
- 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).
Who cannot join
- Patients with previously diagnosed CIDP who lack any CIDP symptoms.
- Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
- Patients who have previously failed immunoglobulin therapy for CIDP.
- 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.
- Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
- Patients on unstable (change in prescribed dose within the last eight weeks) corticosteroids or rituximab use.
Show all 15Show fewer
- Patients with known or suspected hypersensitivity, anaphylaxis, or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
- 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).
- 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.
- 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.
- Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
- History of alcohol or drug abuse in the previous year, per Investigator's opinion.
- Unable or unwilling to comply with the study protocol.
- 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.
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Octapharma Research Site
Birmingham, Alabama, 35233
- Recruiting
Octapharma Research Site
Charlottesville, Virginia, 22908
- Recruiting
Octapharma Research Site
Houston, Texas, 77030
- Recruiting
Octapharma Research Site
Louisville, Kentucky, 40202
- Recruiting
Octapharma Research Site
Orange, California, 92868
- Recruiting
Octapharma Research Site
Philadelphia, Pennsylvania, 19104
About this study
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
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
- Being tested
- Panzyga
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PanzygaDrug
PANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.
| Study group | Role | What they receive |
|---|---|---|
| Panzyga High Dose | Experimental | 2.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages. Receives: Drug: Panzyga |
| Panzyga Low Dose | Experimental | 1.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages. Receives: Drug: Panzyga |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in CIDP Baseline
Evaluate the efficacy of two PANZYGA dose regimens in pediatric CIDP patients based on change in CIDP symptoms, measured by the Modified Rankin Score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
Measured: Up to 24 weeks
Secondary outcomes (3)
CIDP Relapse
Evaluate percentage of patients with CIDP relapse between 2 doses of Panzyga with relapse defined as increase in Modified Rankin score by ≥1 point from the baseline score related to CIDP. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
Measured: Up to 24 weeks
Time to CIDP Relapse
Time to CIDP relapse or withdrawal for any other reason with with relapse defined as increase in Modified Rankin Score score by ≥1 point from the baseline score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
Measured: Up to 24 weeks
Percentage of Patients With Good/Excellent Response
The percentage of patients with good/excellent response, defined by a Modified Rankin Score score of 0 or 1 in each arm at Week 24. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2021
- Study start1 Oct 2023
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Chronic Inflammatory Demyelinating PolyneuropathyStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04929236
- Sponsor's protocol ID
- NGAM-11
- Status last verified
- Aug 2026
- First submitted
- 11 Jun 2021
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04929236
- Acronym / sponsor ID
- NGAM-11
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 1 Oct 2023 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 18 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Panzyga High Dose | Experimental | Drug: Panzyga2.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages. |
| Panzyga Low Dose | Experimental | Drug: Panzyga1.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age ≥2 years and ≤17 years.
- Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone [1]
- Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
- 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).
Key exclusion criteria (15 listed)
- Patients with previously diagnosed CIDP who lack any CIDP symptoms.
- Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
- Patients who have previously failed immunoglobulin therapy for CIDP.
- 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.
- Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PanzygaPANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration. | Drug | Panzyga High Dose, Panzyga Low Dose |
Primary outcome
Change in CIDP Baseline
Time frame: Up to 24 weeks
Evaluate the efficacy of two PANZYGA dose regimens in pediatric CIDP patients based on change in CIDP symptoms, measured by the Modified Rankin Score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.
3 secondary outcomes
CIDP Relapse
Time frame: Up to 24 weeks
Time to CIDP Relapse
Time frame: Up to 24 weeks
Percentage of Patients With Good/Excellent Response
Time frame: Up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
- Individual participant dataSharing planned: No
Related trials for Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
RecruitingPhase 4
Two Dose Levels of Privigen in Pediatric CIDP
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Panzyga in people with Pediatric Chronic Inflammatory Demyelinating Polyneuropathy, run by Octapharma at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Theresa Conklin (866-337-1868, ctgov@clinicalresearchmgt.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in CIDP Baseline (time frame: Up to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2026; AzTrials last refreshed its copy on 6 Nov 2026.