ADDRESS+: 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)

• Sponsor: argenx (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04598477
• Collaborator: (none)
• Enrollment: 150
• Locations: 114
• Arm: 1
• Anticipated Duration (Months): 25.1
• Patients Per Site: 1.3
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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

Study Design

• Study Type: Interventional
• Anticipated 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: Jul 15, 2021
• Anticipated Primary Completion Date: Aug 19, 2023
• Anticipated Study Completion Date: Aug 19, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

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

   Time to Disease Control

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

   Time to complete clinical remission

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

   Time to complete clinical remission on minimal prednisone therapy

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

   Time to complete clinical remission off prednisone therapy

7. Time to flare [Up to 60 weeks]

   Time to flare

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

   Rate of treatment failure

9. Rate of flare [Up to 60 weeks]

   Rate of flare

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

    Cumulative prednisone dose over the trial

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

    Pemphigus Disease Area Index at each visit

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

    EuroQol 5-Dimension 5-Level score

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

    Autoimmune Blister Quality of Life score

14. Efgartigimod serum concentrations [Up to 60 weeks]

    Efgartigimod serum concentrations

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

    Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels

16. Anti-Desmoglein -1 and -3 autoantibodies serum levels [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) [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) [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 [Up to 52 weeks]

    Percentage of participants who performed self-administration

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

    Frequency of self- or caregiver-supported administration at home

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, 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:

4. 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

5. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator site 89 - US0010091	Scottsdale	Arizona	United States	85259
2	Investigator site 1 - US0010087	Boca Raton	Florida	United States	33428
3	Investigator site 90 - US0010117	Miami	Florida	United States	33173
4	Investigator site 91 - US0010109	Orlando	Florida	United States	32827
5	Investigator site 111 - US0010098	Saint Louis	Missouri	United States	63110
6	Investigator site 10 - US0010088	Buffalo	New York	United States	14203-1070
7	Investigator site 76 - US0010096	Durham	North Carolina	United States	27710
8	Investigator site 30 - US0010094	Cleveland	Ohio	United States	44106-1716
9	Investigator site 84 - US0010089	Philadelphia	Pennsylvania	United States	19104
10	Investigator site 103 - US0010097	Philadelphia	Pennsylvania	United States	19140
11	Investigator site 87 - US0010084	Dripping Springs	Texas	United States	78620
12	Investigator site 88 - US0010114	Houston	Texas	United States	77008
13	Investigator site 44 - US0010106	Norfolk	Virginia	United States	23502
14	Investigator site 15 - AU0610006	Sydney	New South Wales	Australia	2217
15	Investigator site 11 - AU0610007	Parkville	Victoria	Australia	3050
16	Investigator site 92 - AU0610013	Melbourne		Australia	3065
17	Investigator site 17 - BG3590012	Pleven		Bulgaria	5800
18	Investigator site 18 - BG3590013	Plovdiv		Bulgaria	4000
19	Investigator site 2 - BG3590010	Sofia		Bulgaria	1431
20	Investigator site 16 - BG3590009	Sofia		Bulgaria	1510
21	Investigator site 3 - BG3590011	Sofia		Bulgaria	1606
22	Investigator site 101 - CN0860017	Beijing		China	100034
23	Investigator site 107 - CN0860018	Chengdu		China	610000
24	Investigator site 100 - CN0860021	Guanzhou		China	510000
25	Investigator site 113 - CN0860024	Nanjing		China	210042
26	Investigator site 102 - CN0860020	Shanghai		China	200025
27	Investigator site 99 - CN0860016	Shanghai		China	200040
28	Investigator site 112 - CN0860019	Wuhan		China	430022
29	Investigator site 77 - FR0330028	Bobigny		France	93000
30	Investigator site 60 - FR0330027	La Tronche		France	38700
31	Investigator site 108 - FR0330029	Rouen		France	76031
32	Investigator site 51 - FR0330026	Saint-Étienne		France	42055
33	Investigator site 78 - GE9950014	Tbilisi		Georgia	0159
34	Investigator site 32 - GE9950013	Tbilisi		Georgia	0162
35	Investigator site 31 - GE9950015	Tbilisi		Georgia	0179
36	Investigator site 45 - DE0490029	Berlin		Germany	10117
37	Investigator site 34 - DE0490030	Dresden		Germany	01307
38	Investigator site 33 - DE0490024	Frankfurt am main		Germany	60590
39	Investigator site 53 - DE0490023	Freiburg		Germany	79104
40	Investigator site 35 - DE0490028	Kiel		Germany	24105
41	Investigator site 20 - DE0490002	Lübeck		Germany	23538
42	Investigator site 52 - DE0490001	Marburg		Germany	35043
43	Investigator site 19 - DE0490025	Tübingen		Germany	72076
44	Investigator site 93 - DE0490027	Ulm		Germany	89081
45	Investigator site 4 - DE0490026	Würzburg		Germany	97080
46	Investigator site 21 - GR030004	Athens		Greece	11525
47	Investigator site 37 - GR030006	Athens		Greece	16121
48	Investigator site 54 - GR0300001	Athens		Greece	16121
49	Investigator site 22 - GR030003	Chaïdári		Greece	12462
50	Investigator site 36 - GR0300002	Thessaloníki		Greece	54643
51	Investigator site 23 - GR030005	Thessaloníki		Greece	56429
52	Investigator site 6 - HU0360003	Debrecen		Hungary	4032
53	Investigator site 5 - HU0360001	Pécs		Hungary	7632
54	Investigator site 24 - HU0360002	Szeged		Hungary	6720
55	Investigator site 65 - IN0910002	Ahmedabad		India	380016
56	Investigator site 94 - IN0910001	Chandigarh		India	160012
57	Investigator site 79 - IN0910004	Lucknow		India	226005
58	Investigator site 80 - IN0910003	Nagpur		India	440003
59	Investigator site 85 - IL9720002	Tel Aviv		Israel	64239
60	Investigator site 95 - IT0390039	Catania		Italy	95123
61	Investigator site 38 - IT0390031	Firenze		Italy	50125
62	Investigator site 81 - IT0390030	Genova		Italy	16132
63	Investigator site 55 - IT0390038	Perugia		Italy	06129
64	Investigator site 12 - IT0390006	Roma		Italy	00167
65	Investigator site 25 - IT-0390005	Roma		Italy	00168
66	Investigator site 61 - IT0390040	Siena		Italy	53100
67	Investigator site 82 - JP0810046	Aichi		Japan	480-1195
68	Investigator site 68 - JP0810040	Hiroshima		Japan	734-8551
69	Investigator site 69 - JP0810050	Kurume		Japan	830-001
70	Investigator site 66 - JP0810042	Kōfu		Japan	400-8506
71	Investigator site 73 - JP0810047	Okayama		Japan	700-8558
72	Investigator site 70 - JP0810041	Okayama		Japan	701-0192
73	Investigator site 71 - JP0810049	Osaka		Japan	545-8586
74	Investigator site 72 - JP0810045	Sapporo		Japan	060-8648
75	Investigator site 114 - JP0810067	Sendai		Japan	980-8574
76	Investigator site 67 - JP0810043	Tokyo		Japan	113-8431
77	Investigator site 27 - PL0480027	Katowice		Poland	40-081
78	Investigator site 86 - PL0480036	Poznań		Poland	60-369
79	Investigator site 28 - PL0480025	Rzeszów		Poland	35-055
80	Investigator site 26 - PL0480028	Wrocław		Poland	50-566
81	Investigator site 56 - PL0480032	Łódź		Poland	90-647
82	Investigator site 97 - RO0400013	Bucharest		Romania	011216
83	Investigator 96 - RO0400014	Cluj-Napoca		Romania	400006
84	Investigator site 98 - RO0400015	Iaşi		Romania	700111
85	Investigator site 40 - RU0070035	Chelyabinsk		Russian Federation	454092
86	Investigator site 41 - RU0070033	Ekaterinburg		Russian Federation	620076
87	Investigator site 48 - RU0070029	Kazan		Russian Federation	420111
88	Investigator site 49 - RU0070030	Krasnodar		Russian Federation	350020
89	Investigator site 39 - RU0070032	Rostov-on-Don		Russian Federation	344002
90	Investigator site 46 - RU0070031	Saint Petersburg		Russian Federation	191123
91	Investigator site 50 - RU0070034	Saint Petersburg		Russian Federation	197022
92	Investigator site 47 - RU0070028	Saratov		Russian Federation	410012/410028
93	Investigator site 109 - RS3810011	Belgrade		Serbia	11000
94	Investigator site 110 - RS3810010	Belgrade		Serbia	11000
95	Investigator site 105 - RS3810012	Niš		Serbia	18000
96	Investigator site 104 - RS3810009	Novi Sad		Serbia	21000
97	Investigator site 62 - ES0340026	Barcelona		Spain	08907
98	Investigator site 13 - ES0340032	Barcelona		Spain	8036
99	Investigator site 59 - ES0340034	Madrid		Spain	28007
100	Investigator site 42 - ES0340025	Madrid		Spain	28034
101	Investigator site 7 - ES0340029	Madrid		Spain	28041
102	Investigator site 57 - ES0340027	Madrid		Spain	28046
103	Investigator site 58 - ES0340028	Sevilla		Spain	41013
104	Investigator site 64 - TR0900020	Gaziantep		Turkey	27310
105	Investigator site 63 - TR0900012	Istanbul		Turkey	34098
106	Investigator site 74 - TR0900011	Istanbul		Turkey	34722
107	Investigator site 75 - UA3800017	Dnipro		Ukraine	49074
108	Investigator site 29 - UA3800023	Ivano-Frankivs'k		Ukraine	76018
109	Investigator site 14 - UA3800020	Kyiv		Ukraine	04050
110	Investigator site 8 - UA3800019	Kyiv		Ukraine	4209
111	Investigator site 43 - UA3800021	Lviv		Ukraine	79013
112	Investigator site 9 - UA3800018	Zaporizhzhia		Ukraine	69063
113	Investigator site 106 - UK0440021	Birmingham		United Kingdom	B15 2GW
114	Investigator site 83 - UK0440022	Bristol		United Kingdom	BS2 8HW

Sponsors and Collaborators

• argenx

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: argenx
• ClinicalTrials.gov Identifier: NCT04598477
• Other Study ID Numbers: ARGX-113-1905
• First Posted: Oct 22, 2020
• Last Update Posted: Jul 29, 2022
• Last Verified: Jul 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Pemphigus; Prednisone

Study Results

No Results Posted as of Jul 29, 2022