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A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus) (ADDRESS+)

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ClinicalTrials.gov Identifier: NCT04598477
Recruitment Status : Recruiting
First Posted : October 22, 2020
Last Update Posted : December 3, 2021
Sponsor:
Information provided by (Responsible Party):
argenx

Brief Summary:

This is a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provides extension of efgartigimod PH20 SC treatment and retreatment options for participants who have been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and the first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to placebo arm in trial ARGX-113-1904. Trial ARGX-113-1905 evaluates ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CRmin, and the ability to treat flare; and assess patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial.

Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration


Condition or disease Intervention/treatment Phase
Pemphigus Vulgaris Pemphigus Foliaceus Biological: efgartigimod PH20 SC Drug: prednisone Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus
Actual Study Start Date : July 15, 2021
Estimated Primary Completion Date : August 19, 2023
Estimated Study Completion Date : August 19, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus Steroids
Drug Information available for: Prednisone

Arm Intervention/treatment
Experimental: efgartigimod PH20 SC
patients receiving efgartigimod PH20 SC on top of prednisone
Biological: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer

Drug: prednisone
Oral prednisone tablets




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) [ Time Frame: Up to 60 weeks ]
    Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)

  2. Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) [ Time Frame: Up to 60 weeks ]
    Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)


Secondary Outcome Measures :
  1. Proportion of Pemphigus Vulgaris (PV) participants who achieve complete clinical remission (CR) on minimal prednisone therapy [ Time Frame: Up to 52 weeks treatment period ]
    Proportion of participants with Pemphigus Vulgaris who achieve complete clinical remission on minimal prednisone therapy.

  2. Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone dose therapy [ Time Frame: Up to 52 weeks treatment period ]
    Proportion of participants with Pemphigus Vulgaris and Pemphigus Foliaceus who achieve complete clinical remission on minimal prednisone therapy.

  3. Time to Disease Control (DC) [ Time Frame: Up to 60 weeks ]
    Time to Disease Control

  4. Time to complete clinical remission (CR) [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission

  5. Time to complete clinical remission (CR) on minimal prednisone therapy [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission on minimal prednisone therapy

  6. Time to complete clinical remission (CR) off prednisone therapy [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission off prednisone therapy

  7. Time to flare [ Time Frame: Up to 60 weeks ]
    Time to flare

  8. Rate of treatment failure [ Time Frame: Up to 60 weeks ]
    Rate of treatment failure

  9. Rate of flare [ Time Frame: Up to 60 weeks ]
    Rate of flare

  10. Cumulative prednisone dose over the trial [ Time Frame: Up to 52 weeks treatment period ]
    Cumulative prednisone dose over the trial

  11. Pemphigus Disease Area Index (PDAI) at each visit [ Time Frame: Up to 52 weeks treatment period ]
    Pemphigus Disease Area Index at each visit

  12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score [ Time Frame: Up to 52 weeks treatment period ]
    EuroQol 5-Dimension 5-Level score

  13. Autoimmune Blister Quality of Life (ABQOL) score [ Time Frame: Up to 52 weeks treatment period ]
    Autoimmune Blister Quality of Life score

  14. Efgartigimod serum concentrations [ Time Frame: Up to 60 weeks ]
    Efgartigimod serum concentrations

  15. Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels [ Time Frame: Up to 60 weeks ]
    Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels

  16. Anti-Desmoglein -1 and -3 autoantibodies serum levels [ Time Frame: Up to 60 weeks ]
    Anti-Desmoglein -1 and -3 autoantibodies serum levels

  17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and recombinant human hyaluronidase PH20 (plasma levels) [ Time Frame: Up to 60 weeks ]
    Anti-drug antibodies to efgartigimod (serum levels) and recombinant human hyaluronidase PH20 (plasma levels)

  18. Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS) [ Time Frame: Up to 52 weeks treatment period ]
    Composite Glucocorticoid Toxicity Index comprising the Aggregate Improvement Score and the Cumulative Worsening Score

  19. Percentage of participants who performed self-administration [ Time Frame: Up to 52 weeks ]
    Percentage of participants who performed self-administration

  20. Percentage of caregivers who administered the injection to the participant [ Time Frame: Up to 52 weeks ]
    Percentage of caregivers who administered the injection to the participant

  21. Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC [ Time Frame: Up to 52 weeks ]
    Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC

  22. Frequency of self- or caregiver-supported administration at home [ Time Frame: Up to 52 weeks ]
    Frequency of self- or caregiver-supported administration at home



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits).
  2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
  3. Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating clinical trials and:

    1. Male participants: Male participants must agree to use acceptable method of contraception, and not donate sperm from signing the ICF until the end of the study
    2. Female participants Women of childbearing potential must:

      • have a negative serum pregnancy test at screening and negative urine pregnancy test at baseline before the IMP can be administered,
      • agree to use a highly effective or acceptable contraception method, which should be maintained at minimum until 90 days after the last dose of IMP

Exclusion Criteria:

  1. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP.
  2. Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk.
  3. Known hypersensitivity to any of the components of the administered treatments.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04598477


Contacts
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Contact: Antonio Guglietta, MD 857-350-4834 ext +1 clinicaltrials@argenx.com

Locations
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Sponsors and Collaborators
argenx
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT04598477    
Other Study ID Numbers: ARGX-113-1905
First Posted: October 22, 2020    Key Record Dates
Last Update Posted: December 3, 2021
Last Verified: December 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Prednisone
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents