Supporting weak immune system during autoimmune therapy: testing Panzyga to prevent infections

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-05-14

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Octapharma Pharmazeutika Produktionsges.m.b.H.

External identifiers

EU CT number

2025-522854-37-00

WHO UTN

U1111-1325-4876

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

Demonstrate the benefit of Panzyga versus placebo for the prevention of major infections in patients with hypogammaglobulinemia and autoimmune or rheumatic conditions receiving treatment with a BCDT.

Secondary objectives 2

1. The secondary efficacy objective of this study is to further evaluate the effectiveness of Panzyga versus placebo for extending the period without major infection in patients with hypogammaglobulinemia and autoimmune or rheumatic conditions receiving treatment with a BCDT
2. The secondary safety objective of this study is to assess safety of Panzyga versus placebo when used for the prevention of major infections in patients with hypogammaglobulinemia and autoimmune or rheumatic conditions receiving treatment with a BCDT.

Conditions and MedDRA coding

Prevention of a Major infection in patients with Hypogammaglobulinemia and Autoimmune or Rheumatic Conditions receiving treatment with B-cell depletion therapy.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Are ≥18 years of age at time of informed consent, have been diagnosed with a rheumatic or autoimmune condition, received their last BCDT dose within 3 months of Screening, and have the intention to receive BCDT during trial participation. Note: Patients with the following indications are eligible: MS, RA, vasculitis/myositis, SLE, Sjogren’s syndrome, idiopathic inflammatory myopathy, mixed connective tissue disease, undifferentiated connective tissue disease, myasthenia gravis, autoimmune encephalitis, CIDP, and neuromyelitis optica spectrum disorder. Other rheumatic and autoimmune conditions may also be acceptable with approval from the Medical Monitor.
2. 2. Have hypogammaglobulinemia (IgG levels <5 g/L as confirmed by the central laboratory).
3. 3. Are willing and able to provide voluntary written informed consent for participation in the study and to comply with all protocol requirements
4. 4. Are willing and able to comply with an acceptable effective contraception method during and for 30 days after the treatment period. Contraceptive use by men and women of child-bearing potential should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion criteria 17

1. 1. Have a history of anaphylaxis or severe systemic response to immunoglobulin, blood, or plasma-derived products, or any Panzyga component
2. 2. Have a current major infection at Screening or had >1 major infection within 6 months prior to Baseline
3. 3. Have a history of thromboembolic events such as deep vein thrombosis (DVT), pulmonary embolism, myocardial infarction, ischemic stroke, transient ischemic attack, or peripheral artery disease (Fontaine IV) within 6 months prior to Baseline
4. 4. Have a known IgA deficiency with antibodies to IgA
5. 5. Have a known blood hyperviscosity or other hypercoagulable states
6. 6. Have been diagnosed with primary immunodeficiency
7. 7. Have a severe liver disease, with signs of ascites or hepatic encephalopathy
8. 8. Have a severe kidney disease (as defined by eGFR <30 mL/min/1.73 m2)
9. 9. Have body weight >140 kg
10. 10. HIV infection at Screening (defined for the study as positive HIV NAT test or reactive HIV-1/2 antigen/antibody immunoassay followed by positive HIV-1 /HIV-2 antibody differentiation immunoassay)
11. 11. Patients found to be chronic carriers of hepatitis B virus (HBV), defined by positive surface antigen (HBsAg), positive Hepatitis B core antibodies (HBcAb) and/or low HBV titers, who will not receive targeted antiviral therapy while participating in the study, and patients with active HBV, defined as high HBV titers.
12. 12. Uncontrolled hepatitis C infection at Screening (defined for the study as positive HCV PCR).
13. 13. Have received IgG treatment within 6 months prior to Screening or plan to receive IgG therapy, other than IMP, during the study
14. 14. Are receiving or plan to receive immunosuppressive treatment (other than for underlying condition) or other forbidden medication during the entire study duration
15. 15. Are participating or plan to participate in another study that is either blinded or involves an investigational medicinal product (IMP) within 3 months prior to Baseline or during the course of this study. Participation in observational or open-label studies involving an approved product may be permitted after consultation with the Medical Monitor.
16. 16. If female, are pregnant or lactating
17. 17. Are likely to be non-compliant or uncooperative during the study, or unable to cooperate because of a language problem or poor mental development, in the opinion of the Investigator

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of at least one major infection or death in patients with or without primary infection prophylaxis with Panzyga. Major infections will be recorded throughout the study along with the type and severity of infection and time to resolution. Each patient will be counted only once for the primary endpoint calculation. Each potential infection will be assessed by an Independent Adjudication Committee (IAC) consisting of clinical experts.

Secondary endpoints 3

1. 1. Efficacy Endpoint: The time to first major infection (as assessed by the IAC) or death.
2. 2. Safety Endpoint: Incidence of AEs
3. 3. Safety Endpoint: Changes from Baseline in physical examinations, and clinical laboratory parameters.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Sponsor organisation

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Address

Oberlaaer Strasse 235, Favoriten Favoriten

City

Vienna

Postcode

1100

Country

Austria

Scientific contact point

Organisation

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Contact name

Global Clinical Project Manager

Public contact point

Organisation

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Contact name

Global Clinical Project Manager

Third parties 8

Locations

8 EU/EEA countries · 24 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 106 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications