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ADAPTSC+: Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis

Sponsor
argenx (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04818671
Collaborator
(none)
183
Enrollment
47
Locations
1
Arm
24.1
Anticipated Duration (Months)
3.9
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the long-term safety and tolerability of efgartigimod PH20 SC 1000 mg, and the clinical efficacy, PD, pharmacokinetics (PK), immunogenicity, impact on the quality of life (QoL) of the participants, treatment satisfaction, and administration method preference, and the feasibility of self- and caregiver-supported administration of the SC injection.

Study details include: Study duration: 2 years

Treatment duration: 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods

Health measurements: total levels of immunoglobulin G (IgG), Acetylcholine receptor binding autoantibodies (AChR-Ab) levels, Myasthenia Gravis Activities of Daly Living (MG-ADL).

Condition or Disease Intervention/Treatment Phase
  • Biological: efgartigimod PH20 SC
 Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
183 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Long-Term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia Gravis
Actual Study Start Date :
Apr 26, 2021
Anticipated Primary Completion Date :
Apr 30, 2023
Anticipated Study Completion Date :
Apr 30, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: efgartigimod PH20 SC

Patients receiving efgartigimod PH20 subcutaneous (SC) treatment

Biological: efgartigimod PH20 SC
Subcutaneous injection with efgartigimod PH20 SC

Outcome Measures

Primary Outcome Measures

  1. Incidence and severity of Adverse Events (AEs) [Up to 2 years]

  2. Incidence of Serious Adverse Events (SAEs) [Up to 2 years]

  3. Incidence of Adverse Events of Special Interest (AESI) [Up to 2 years]

Secondary Outcome Measures

  1. Myasthenia Gravis Activities of Daily Living (MG-ADL) total score changes from baseline [Up to 2 years]

    the higher the score, the more impairment

  2. Cycle baseline over time by cycle (for MG-ADL) [Up to 2 years]

  3. Percentage change in levels of total immunoglobulin G (IgG) from baseline [Up to 2 years]

  4. Cycle baseline over time by cycle (for total immunoglobulin G (IgG) [Up to 2 years]

  5. Percentage change of anti-acetylcholine receptor antibodies (AChR-Ab) from baseline [Up to 2 years]

  6. Cycle baseline over time by cycle in AChR-Ab seropositive participants (for acetylcholine receptor binding autoantibodies (AChR-Ab)) [Up to 2 years]

  7. Efgartigimod serum concentrations [Up to 2 years]

  8. Incidence of anti-drug antibodies (ADAs) to efgartigimod over time [Up to 2 years]

  9. Prevalence of anti-drug antibodies (ADAs) to efgartigimod over time [Up to 2 years]

  10. Incidence of neutralizing antibodies (NAbs) against efgartigimod over time [Up to 2 years]

  11. Prevalence of neutralizing antibodies (NAbs) against efgartigimod over time [Up to 2 years]

  12. Incidence of ADAs to rHuPH20 over time [Up to 2 years]

  13. Prevalence of ADAs to rHuPH20 over time [Up to 2 years]

  14. Incidence of NAbs against rHuPH20 over time [Up to 2 years]

  15. Prevalence of NAbs against rHuPH20 over time [Up to 2 years]

  16. Changes in total Myasthenia Gravis Quality of Life Questionnaire (15-item scale revised) (MG-QoL15r) from baseline [Up to 2 years]

  17. Cycle baseline by cycle (for MG-QoL15r) [Up to 2 years]

  18. Changes in EuroQoL 5 Dimensions 5-Level (EQ-5D-5L) visual analog scale (VAS) score from baseline [Up to 2 years]

  19. Cycle baseline by cycle (for EQ-5D-5L) [Up to 2 years]

  20. EQ-5D-5L responses over time by cycle [Up to 2 years]

  21. Number of participants who performed self-administration at home over time by cycle [Up to 2 years]

  22. Percentage of participants who performed self-administration at home over time by cycle [Up to 2 years]

  23. Number of caregivers who administered the injection to the participant at home over time by cycle [Up to 2 years]

  24. Percentage of caregivers who administered the injection to the participant at home over time by cycle [Up to 2 years]

  25. Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC [Up to 2 years]

  26. Number of self- or caregiver-supported study drug administration among all study treatment visits at home [Up to 2 years]

  27. Percentage of self- or caregiver-supported study drug administration among all study treatment visits at home [Up to 2 years]

Eligibility Criteria

Criteria

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

  1. Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

  2. Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for roll over.

  3. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:

  4. Male participants:

  • Male participants are not allowed to donate sperm from signing the informed consent (ICF) until the end of study.
  1. Female participants:
  • Women of Child bearing potential (WOCBP) must have a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.
Exclusion Criteria:
  1. The participant was discontinued early from studies ARGX-113-2001 or ARGX-113-1705, unless the reason for discontinuation from study ARGX-113-1705 was to roll over into study ARGX-113-2002.
  1. Participants who, in the investigator's judgment, are not benefiting from efgartigimod IV in study ARGX-113-1705 Part B are not eligible for roll over into ARGX-113-2002.

  1. Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of investigational medicinal product (IMP)

  2. Has any of the following medical conditions:

  3. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at screening

  4. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk

  5. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of investigational medicinal product (IMP).

Participants with the following cancers can be included at any time:

  • adequately treated basal cell or squamous cell skin cancer

  • carcinoma in situ of the cervix

  • carcinoma in situ of the breast

  • incidental histological findings of prostate cancer (TNM classification of malignant tumors stage T1a or T1b)

  1. Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk

  2. Received a live-attenuated vaccine within 28 days prior to study entry or plan to receive a live-attenuated vaccine during the study

  3. A known hypersensitivity reaction to efgartigimod, rHuPH20, or any of its excipients

Contacts and Locations

Locations

Site City State Country Postal Code
1 Investigator site 6 - US0010032 Carlsbad California United States 92011
2 Investigator Site 47 - US0010021 Palo Alto California United States 94304
3 Investigator Site 45 - US0010108 Boca Raton Florida United States 33428
4 Investigator site 4 - US0010110 Port Charlotte Florida United States 33952
5 Investigator Site 39 - US0010006 Tampa Florida United States 41076
6 Investigator Site 41 - US0010015 Kansas City Kansas United States 66160
7 Investigator Site 46 - US0010111 Amherst New York United States 14226
8 Investigator Site 38 - US0010003 Chapel Hill North Carolina United States 27514
9 Investigator Site 44 - US0010077 Durham North Carolina United States 27710
10 Investigator Site 42 - US0010019 Cleveland Ohio United States 44195
11 Investigator site 7 - US0010008 Cordova Tennessee United States 38018
12 Investigator Site 43 - US0010066 Austin Texas United States 78756
13 Investigator Site 40 - US0010009 San Antonio Texas United States 78229
14 Investigator site 5 - BE0320007 Gent Belgium 9000
15 Investigator site 24 - CZ4200005 Brno Czechia 625 00
16 Investigator site 2 - GEO9950002 Tbilisi Georgia 0112
17 Investigator Site 1 - GEO9950001 Tbilisi Georgia 0114
18 Investigator site 3 - GEO9950003 Tbilisi Georgia 0114
19 Investigator Site 32 - GEO9950004 Tbilisi Georgia 0160
20 Investigator Site 33 - GEO9950016 Tbilisi Georgia 016
21 Investigator Site 25 - DE490006 Berlin Germany 10117
22 Investigator Site 26 - DE490009 Münster Germany 48149
23 Investigator site 10 - HU0360013 Budapest Hungary 1082
24 Investigator site 9 - HU0360012 Budapest Hungary 1204
25 Investigator site 11 - IT0390003 Milano Italy 20133
26 Investigator Site 34 - IT0390007 Napoli Italy 80138
27 Investigator Site 35 - IT0390008 Roma Italy 00189
28 Investigator site 12 - JP0810002 Chiba Chiba-Shi Japan 260-8677
29 Investigator Site 36 - JP0810055 Sapporo Hokkaido Japan 063-0005
30 Investigator site 8 - JP0810004 Hanamaki Iwate Japan 025-0082
31 Investigator Site 28- JP0810059 Ota-Ku Tokyo Japan 143-8541
32 Investigator site 14 - JP0810007 Osaka Japan 565-0871
33 Investigator Site 27 - JP0810008 Sapporo Japan 060 8542
34 Investigator site 13 - JP0810005 Sendai-shi Japan 983-8520
35 Investigator site 15 - JP0810009 Tokyo Japan 160-0023
36 Investigator site 16 - NL0310001 Leiden Netherlands 2333
37 Investigator site 17 - PL0480001 Gdańsk Poland 80-952
38 Investigator site 19 - PL0480007 Katowice Poland 40-123
39 Investigator site 22 - PL0480024 Kraków Poland 31-2002
40 Investigator site 18 - PL0480005 Kraków Poland 31-505
41 Investigator site 20 - PL0480018 Lublin Poland 20-093
42 Investigator site 21 - PL0480022 Warsaw Poland 02-097
43 Investigator Site 29- RU0070002 Novosibirsk Russian Federation 630087
44 Investigator Site 30 - RU0070014 Saint Petersburg Russian Federation 194354
45 Investigator Site 37 - ES0340021 Barcelona Spain 08035
46 Investigator Site 31 - ES0340038 Barcelona Spain 08041
47 Investigator site 23 - ES0340039 Valencia Spain 46026

Sponsors and Collaborators

  • argenx

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
argenx
ClinicalTrials.gov Identifier:
NCT04818671
Other Study ID Numbers:
  • ARGX-113-2002
First Posted:
Mar 26, 2021
Last Update Posted:
Jul 6, 2022
Last Verified:
Jul 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:
Myasthenia Gravis
Muscle Weakness

Study Results

No Results Posted as of Jul 6, 2022