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A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE SC+)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04812925
Recruitment Status : Recruiting
First Posted : March 24, 2021
Last Update Posted : June 28, 2022
Sponsor:
Information provided by (Responsible Party):
argenx

Brief Summary:
A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia Biological: efgartigimod PH20 SC Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 156 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Open-Label, Long-Term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
Actual Study Start Date : November 17, 2021
Estimated Primary Completion Date : October 1, 2026
Estimated Study Completion Date : October 1, 2026


Arm Intervention/treatment
Experimental: efgartigimod PH20 SC
Patients receiving efgartigimod PH20 SC treatment
Biological: efgartigimod PH20 SC
Subcutaneous injection with efgartigimod PH20 SC
Other Name: ARGX-113 PH20 SC




Primary Outcome Measures :
  1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) [ Time Frame: 216 weeks ]
  2. Vital sign measurement: blood pressure in the overall population [ Time Frame: 216 weeks ]
  3. ECG: PR, QT and QRS interval in the overall population [ Time Frame: 216 weeks ]
  4. Laboratory safety evaluations: CRP analysis in the overall population [ Time Frame: 216 weeks ]

Secondary Outcome Measures :
  1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L [ Time Frame: 52 weeks ]
  2. Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period [ Time Frame: 52 weeks ]
  3. Mean change from baseline in platelet count at each visit [ Time Frame: 52 weeks ]
  4. For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L [ Time Frame: 52 weeks ]
  5. The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline [ Time Frame: 52 weeks ]
  6. In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline [ Time Frame: 52 weeks ]
  7. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24 [ Time Frame: 5 weeks (week 19-24) ]
  8. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24 [ Time Frame: 7 weeks (week 17-24) ]
  9. Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline [ Time Frame: 52 weeks ]
  10. Rate of receipt of rescue therapy (rescue per patient per month) [ Time Frame: 52 weeks ]
  11. Incidence of the World Health Organization (WHO)-classified bleeding events [ Time Frame: 52 weeks ]
  12. Severity of the World Health Organization (WHO)-classified bleeding events [ Time Frame: 52 weeks ]
  13. Serum efgartigimod concentration observed predose (Ctrough) [ Time Frame: 52 weeks ]
  14. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits [ Time Frame: 52 weeks ]
  15. Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits [ Time Frame: 52 weeks ]
  16. Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits [ Time Frame: 52 weeks ]
  17. Pharmacodynamics markers: total IgG [ Time Frame: 52 weeks ]
  18. Number of patients who performed self-administration at home over time [ Time Frame: 52 weeks ]
  19. Percentage of patients who performed self-administration at home over time [ Time Frame: 52 weeks ]
  20. Number of caregivers who administered the injection to the patient at home over time [ Time Frame: 52 weeks ]
  21. Percentage of caregivers who administered the injection to the patient at home over time [ Time Frame: 52 weeks ]
  22. Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC [ Time Frame: 52 weeks ]
  23. Number of self- or caregiver-supported administrations at home [ Time Frame: 52 weeks ]
  24. Percentage of self- or caregiver-supported administrations at home [ Time Frame: 52 weeks ]
  25. Incidence and prevalence of antibodies to efgartigimod [ Time Frame: 216 weeks ]
  26. Titers of antibodies to efgartigimod [ Time Frame: 216 weeks ]
  27. Presence of neutralizing antibodies (NAb) against efgartigimod [ Time Frame: 216 weeks ]


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 and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits).
  2. Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period.

Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case.

3a. Agree to use contraceptives consistent with local regulations regarding the methods of contraception for those participating in clinical trials and the following:

  • Male participants.
  • Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP.

In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP or available through another patient program for patients with primary ITP), the following criteria apply:

4. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).

5. Participant has completed a 52-week treatment period.

Exclusion criteria:

  1. Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines)
  2. Use of any other investigational drug or participation in any other investigational trial
  3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients
  4. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC

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): NCT04812925


Contacts
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Contact: Sabine Coppieters, 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: NCT04812925    
Other Study ID Numbers: ARGX-113-2005
First Posted: March 24, 2021    Key Record Dates
Last Update Posted: June 28, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations