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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 27, 2021
Sponsor:
Information provided by (Responsible Party):
argenx

Tracking Information
First Submitted Date  ICMJE October 8, 2020
First Posted Date  ICMJE October 22, 2020
Last Update Posted Date December 27, 2021
Actual Study Start Date  ICMJE July 15, 2021
Estimated Primary Completion Date August 19, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 16, 2020)
  • 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)
  • 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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 16, 2021)
  • 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.
  • 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.
  • Time to Disease Control (DC) [ Time Frame: Up to 60 weeks ]
    Time to Disease Control
  • Time to complete clinical remission (CR) [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission
  • 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
  • Time to complete clinical remission (CR) off prednisone therapy [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission off prednisone therapy
  • Time to flare [ Time Frame: Up to 60 weeks ]
    Time to flare
  • Rate of treatment failure [ Time Frame: Up to 60 weeks ]
    Rate of treatment failure
  • Rate of flare [ Time Frame: Up to 60 weeks ]
    Rate of flare
  • Cumulative prednisone dose over the trial [ Time Frame: Up to 52 weeks treatment period ]
    Cumulative prednisone dose over the trial
  • Pemphigus Disease Area Index (PDAI) at each visit [ Time Frame: Up to 52 weeks treatment period ]
    Pemphigus Disease Area Index at each visit
  • EuroQol 5-Dimension 5-Level (EQ-5D-5L) score [ Time Frame: Up to 52 weeks treatment period ]
    EuroQol 5-Dimension 5-Level score
  • Autoimmune Blister Quality of Life (ABQOL) score [ Time Frame: Up to 52 weeks treatment period ]
    Autoimmune Blister Quality of Life score
  • Efgartigimod serum concentrations [ Time Frame: Up to 60 weeks ]
    Efgartigimod serum concentrations
  • 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
  • Anti-Desmoglein -1 and -3 autoantibodies serum levels [ Time Frame: Up to 60 weeks ]
    Anti-Desmoglein -1 and -3 autoantibodies serum levels
  • 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)
  • 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
  • Percentage of participants who performed self-administration [ Time Frame: Up to 52 weeks ]
    Percentage of participants who performed self-administration
  • 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
  • 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
  • Frequency of self- or caregiver-supported administration at home [ Time Frame: Up to 52 weeks ]
    Frequency of self- or caregiver-supported administration at home
Original Secondary Outcome Measures  ICMJE
 (submitted: October 16, 2020)
  • Proportion of Pemphigus Vulgaris (PV) patients who achieve complete clinical remission (CR) on minimal prednisone therapy [ Time Frame: Up to 52 weeks treatment period ]
    Proportion of patients with Pemphigus Vulgaris who achieve complete clinical remission on minimal prednisone therapy.
  • Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) patients who achieve complete clinical remission (CR) on minimal prednisone dose therapy [ Time Frame: Up to 52 weeks treatment period ]
    Proportion of patients with Pemphigus Vulgaris and Pemphigus Foliaceus who achieve complete clinical remission on minimal prednisone therapy.
  • Time to Disease Control (DC) [ Time Frame: Up to 60 weeks ]
    Time to Disease Control
  • Time to complete clinical remission (CR) [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission
  • 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
  • Time to complete clinical remission (CR) off prednisone therapy [ Time Frame: Up to 60 weeks ]
    Time to complete clinical remission off prednisone therapy
  • Time to flare [ Time Frame: Up to 60 weeks ]
  • Rate of treatment failure [ Time Frame: Up to 60 weeks ]
  • Rate of flare [ Time Frame: Up to 60 weeks ]
  • Cumulative prednisone dose over the trial [ Time Frame: Up to 52 weeks treatment period ]
  • Pemphigus Disease Area Index (PDAI) at each visit [ Time Frame: Up to 52 weeks treatment period ]
    Pemphigus Disease Area Index at each visit
  • EuroQol 5-Dimension 5-Level (EQ-5D-5L) score [ Time Frame: Up to 52 weeks treatment period ]
  • Autoimmune Blister Quality of Life (ABQOL) score [ Time Frame: Up to 52 weeks treatment period ]
  • Efgartigimod serum concentrations [ Time Frame: Up to 60 weeks ]
  • Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels [ Time Frame: Up to 60 weeks ]
  • Anti-Desmoglein -1 and -3 autoantibodies serum levels [ Time Frame: Up to 60 weeks ]
  • Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and recombinant human hyaluronidase PH20 (plasma levels) [ Time Frame: Up to 60 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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)
Official Title  ICMJE 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
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

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Pemphigus Vulgaris
  • Pemphigus Foliaceus
Intervention  ICMJE
  • Biological: efgartigimod PH20 SC
    Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
  • Drug: prednisone
    Oral prednisone tablets
Study Arms  ICMJE Experimental: efgartigimod PH20 SC
patients receiving efgartigimod PH20 SC on top of prednisone
Interventions:
  • Biological: efgartigimod PH20 SC
  • Drug: prednisone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 16, 2020)
150
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 19, 2023
Estimated Primary Completion Date August 19, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Antonio Guglietta, MD 857-350-4834 ext +1 clinicaltrials@argenx.com
Listed Location Countries  ICMJE Australia,   Bulgaria,   China,   France,   Georgia,   Germany,   Greece,   Hungary,   India,   Israel,   Italy,   Japan,   Poland,   Romania,   Russian Federation,   Spain,   Turkey,   Ukraine,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04598477
Other Study ID Numbers  ICMJE ARGX-113-1905
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party argenx
Study Sponsor  ICMJE argenx
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account argenx
Verification Date December 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP