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

• Sponsor: argenx (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04812925
• Collaborator: (none)
• Enrollment: 156
• Locations: 37
• Arm: 1
• Anticipated Duration (Months): 58.4
• Patients Per Site: 4.2
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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

Study Design

• Study Type: Interventional
• Anticipated 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: Nov 17, 2021
• Anticipated Primary Completion Date: Oct 1, 2026
• Anticipated Study Completion Date: Oct 1, 2026

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) [216 weeks]

2. Vital sign measurement: blood pressure in the overall population [216 weeks]

3. ECG: PR, QT and QRS interval in the overall population [216 weeks]

4. Laboratory safety evaluations: CRP analysis in the overall population [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 [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 [52 weeks]

3. Mean change from baseline in platelet count at each visit [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 [52 weeks]

5. The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline [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 [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 [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 [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 [52 weeks]

10. Rate of receipt of rescue therapy (rescue per patient per month) [52 weeks]

11. Incidence of the World Health Organization (WHO)-classified bleeding events [52 weeks]

12. Severity of the World Health Organization (WHO)-classified bleeding events [52 weeks]

13. Serum efgartigimod concentration observed predose (Ctrough) [52 weeks]

14. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits [52 weeks]

15. Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits [52 weeks]

16. Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits [52 weeks]

17. Pharmacodynamics markers: total IgG [52 weeks]

18. Number of patients who performed self-administration at home over time [52 weeks]

19. Percentage of patients who performed self-administration at home over time [52 weeks]

20. Number of caregivers who administered the injection to the patient at home over time [52 weeks]

21. Percentage of caregivers who administered the injection to the patient at home over time [52 weeks]

22. Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC [52 weeks]

23. Number of self- or caregiver-supported administrations at home [52 weeks]

24. Percentage of self- or caregiver-supported administrations at home [52 weeks]

25. Incidence and prevalence of antibodies to efgartigimod [216 weeks]

26. Titers of antibodies to efgartigimod [216 weeks]

27. Presence of neutralizing antibodies (NAb) against efgartigimod [216 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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:

1. 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).

2. 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site 0010116	Bentonville	Arkansas	United States	72758
2	Investigator Site 0010062	Fort Wayne	Indiana	United States	46804
3	Investigator Site 0010095	Oklahoma City	Oklahoma	United States	73142
4	Investigator Site 0540001	Buenos Aires		Argentina
5	Investigator Site 0610012	Garran		Australia
6	Investigator Site 0610003	West Perth		Australia
7	Investigator Site 3590017	Plovdiv		Bulgaria
8	Investigator Site 0860008	Bengbu		China
9	Investigator Site 0860015	Shenzhen		China
10	Investigator Site 0860001	Tianjin		China
11	Investigator Site 0860010	Wuhan		China
12	Investigator Site 0860002	Wuxi		China
13	Investigator Site 0860011	Zhengzhou		China
14	Investigator Site 9950007	Tbilisi		Georgia
15	Investigator Site 0300009	Thessaloníki		Greece
16	Investigator Site 0390032	Milan		Italy
17	Investigator Site 0390044	Napoli		Italy
18	Investigator Site 0810017	Iruma		Japan
19	Investigator Site 0810053	Kanagawa		Japan
20	Investigator Site 0810051	Kitakyushu		Japan
21	Investigator Site 0810038	Tama		Japan
22	Investigator Site 0480026	Nowy Sącz		Poland
23	Investigator Site 0480037	Skorzewo		Poland
24	Investigator Site 3510007	Lisboa		Portugal
25	Investigator Site 0400005	Bucharest		Romania
26	Investigator Site 0400007	Craiova		Romania
27	Investigator Site 0070026	Moscow		Russian Federation
28	Investigator Site 0070038	Nizhny Novgorod		Russian Federation
29	Investigator Site 0070037	Novosibirsk		Russian Federation
30	Investigator Site 0660001	Bangkok Noi		Thailand
31	Investigator Site 0660002	Bangkok		Thailand
32	Investigator Site 0660003	Bangkok		Thailand
33	Investigator Site 0900007	Adapazarı		Turkey
34	Investigator Site 0900003	Ankara		Turkey
35	Investigator Site 0900008	Ankara		Turkey
36	Investigator Site 0900015	Ankara		Turkey
37	Investigator Site 0900014	Kocaeli		Turkey

Sponsors and Collaborators

• argenx

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: argenx
• ClinicalTrials.gov Identifier: NCT04812925
• Other Study ID Numbers: ARGX-113-2005
• First Posted: Mar 24, 2021
• Last Update Posted: Jun 28, 2022
• Last Verified: Jun 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic

Study Results

No Results Posted as of Jun 28, 2022