Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis (ADAPTSC+)
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| ClinicalTrials.gov Identifier: NCT04818671 |
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Recruitment Status :
Active, not recruiting
First Posted : March 26, 2021
Last Update Posted : March 31, 2023
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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.
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 |
|---|---|---|
| Generalized Myasthenia Gravis | Biological: efgartigimod PH20 SC | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| 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 : | April 26, 2021 |
| Estimated Primary Completion Date : | December 31, 2024 |
| Estimated Study Completion Date : | December 31, 2024 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: efgartigimod PH20 SC
Patients receiving efgartigimod PH20 subcutaneous (SC) treatment
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Biological: efgartigimod PH20 SC
Subcutaneous injection with efgartigimod PH20 SC |
- Incidence and severity of Adverse Events (AEs) [ Time Frame: Up to 3.5 years ]
- Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to 3.5 years ]
- Incidence of Adverse Events of Special Interest (AESI) [ Time Frame: Up to 3.5 years ]
- Myasthenia Gravis Activities of Daily Living (MG-ADL) total score changes from baseline [ Time Frame: Up to 3.5 years ]the higher the score, the more impairment
- Cycle baseline over time by cycle (for MG-ADL) [ Time Frame: Up to 3.5 years ]
- Percentage change in levels of total immunoglobulin G (IgG) from baseline [ Time Frame: Up to 3.5 years ]
- Cycle baseline over time by cycle (for total immunoglobulin G (IgG) [ Time Frame: Up to 3.5 years ]
- Percentage change of anti-acetylcholine receptor antibodies (AChR-Ab) from baseline [ Time Frame: Up to 3.5 years ]
- Cycle baseline over time by cycle in AChR-Ab seropositive participants (for acetylcholine receptor binding autoantibodies (AChR-Ab)) [ Time Frame: Up to 3.5 years ]
- Efgartigimod serum concentrations [ Time Frame: Up to 3.5 years ]
- Incidence of anti-drug antibodies (ADAs) to efgartigimod over time [ Time Frame: Up to 3.5 years ]
- Prevalence of anti-drug antibodies (ADAs) to efgartigimod over time [ Time Frame: Up to 3.5 years ]
- Incidence of neutralizing antibodies (NAbs) against efgartigimod over time [ Time Frame: Up to 3.5 years ]
- Prevalence of neutralizing antibodies (NAbs) against efgartigimod over time [ Time Frame: Up to 3.5 years ]
- Incidence of ADAs to rHuPH20 over time [ Time Frame: Up to 3.5 years ]
- Prevalence of ADAs to rHuPH20 over time [ Time Frame: Up to 3.5 years ]
- Incidence of NAbs against rHuPH20 over time [ Time Frame: Up to 3.5 years ]
- Prevalence of NAbs against rHuPH20 over time [ Time Frame: Up to 3.5 years ]
- Changes in total Myasthenia Gravis Quality of Life Questionnaire (15-item scale revised) (MG-QoL15r) from baseline [ Time Frame: Up to 3.5 years ]
- Cycle baseline by cycle (for MG-QoL15r) [ Time Frame: Up to 3.5 years ]
- Changes in EuroQoL 5 Dimensions 5-Level (EQ-5D-5L) visual analog scale (VAS) score from baseline [ Time Frame: Up to 3.5 years ]
- Cycle baseline by cycle (for EQ-5D-5L) [ Time Frame: Up to 3.5 years ]
- EQ-5D-5L responses over time by cycle [ Time Frame: Up to 3.5 years ]
- Number of participants who performed self-administration at home over time by cycle [ Time Frame: Up to 3.5 years ]
- Percentage of participants who performed self-administration at home over time by cycle [ Time Frame: Up to 3.5 years ]
- Number of caregivers who administered the injection to the participant at home over time by cycle [ Time Frame: Up to 3.5 years ]
- Percentage of caregivers who administered the injection to the participant at home over time by cycle [ Time Frame: Up to 3.5 years ]
- Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC [ Time Frame: Up to 3.5 years ]
- Number of self- or caregiver-supported study drug administration among all study treatment visits at home [ Time Frame: Up to 3.5 years ]
- Percentage of self- or caregiver-supported study drug administration among all study treatment visits at home [ Time Frame: Up to 3.5 years ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
- Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for roll over.
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Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
- 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:
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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.
a. 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.
- 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)
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Has any of the following medical conditions:
- Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at roll-over
- 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
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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)
- 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
- Received a live-attenuated vaccine within 28 days prior to study entry or plan to receive a live-attenuated vaccine during the study
- A known hypersensitivity reaction to efgartigimod, rHuPH20, or any of its excipients
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): NCT04818671
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| Responsible Party: | argenx |
| ClinicalTrials.gov Identifier: | NCT04818671 |
| Other Study ID Numbers: |
ARGX-113-2002 |
| First Posted: | March 26, 2021 Key Record Dates |
| Last Update Posted: | March 31, 2023 |
| Last Verified: | March 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Myasthenia Gravis Muscle Weakness Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms |
Neoplasms by Site Neoplasms Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases Autoimmune Diseases Immune System Diseases |