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An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness (ADAPT)

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ClinicalTrials.gov Identifier: NCT03669588
Recruitment Status : Completed
First Posted : September 13, 2018
Results First Posted : February 8, 2022
Last Update Posted : February 8, 2022
Sponsor:
Information provided by (Responsible Party):
argenx

Tracking Information
First Submitted Date  ICMJE September 6, 2018
First Posted Date  ICMJE September 13, 2018
Results First Submitted Date  ICMJE January 14, 2022
Results First Posted Date  ICMJE February 8, 2022
Last Update Posted Date February 8, 2022
Actual Study Start Date  ICMJE August 22, 2018
Actual Primary Completion Date April 6, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 14, 2022)
Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population [ Time Frame: Baseline up to Day 63 (end of TC1) ]
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 [C1B]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Original Primary Outcome Measures  ICMJE
 (submitted: September 11, 2018)
Efficacy of ARGX-113 as assessed by the percentage of "Myasthenia Gravis Activities of Daily Living (MG-ADL) responders" in the acetylcholine receptor (AChR)-antibody (Ab) seropositive population [ Time Frame: Week 8 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 14, 2022)
  • Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population [ Time Frame: Baseline up to Day 63 (end of TC1) ]
    The QMG scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity. A patient was considered a QMG responder during C1 if there was a reduction of ≥3-points on the QMG total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
  • Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population [ Time Frame: Baseline up to Day 63 (end of TC1) ]
    The percentage of MG-ADL responders during C1 in the overall population is reported for this secondary end point; percentage of MG-ADL responders during C1 in the AChR-Ab seropositive population is reported previously as a primary end point.
  • Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population [ Time Frame: Baseline up to Day 126 ]
    An MG-ADL CMI was defined as a reduction of ≥2 points on the total MG-ADL score compared to study entry baseline (SEB).
  • Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population [ Time Frame: Week 4 up to Day 182 (end of study [EoS]) ]
    Time to qualify for retreatment was defined as time from the Week 4 assessment until the first visit with a <2-point reduction compared to SEB in the MG-ADL total score and MG-ADL total score ≥5 points with >50% of the total score attributable to nonocular symptoms.
  • Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population [ Time Frame: Baseline up to Day 63 (end of TC1) ]
    A patient was considered an early MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than Week 2 (ie, after 1 or maximum 2 infusions of IMP in C1).
Original Secondary Outcome Measures  ICMJE
 (submitted: September 11, 2018)
  • Efficacy of ARGX-113 as assessed by the percentage of "Quantitative Myasthenia Gravis (QMG) responders" in the AChR-Ab seropositive population. [ Time Frame: Week 8 ]
  • Efficacy of ARGX-113 as assessed by the percentage of "MG-ADL responders" in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients). [ Time Frame: Week 8 ]
  • Efficacy of ARGX-113 as assessed by the percentage of time that patients show a "clinically meaningful improvement" in total MG-ADL score during the trial in the AChR-Ab seropositive population [ Time Frame: Through study completion, an average of 26 weeks ]
  • Duration of response [ Time Frame: Through study completion, an average of 26 weeks ]
  • Onset of efficacy of ARGX-113 as assessed by the percentage of "early MG-ADL responders" in the AChR-Ab seropositive population. [ Time Frame: Up to Week 8 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness
Brief Summary A randomized, double-blind, placebo controlled, multicenter Phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of ARGX-113 in patients with gMG.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Generalized Myasthenia Gravis
Intervention  ICMJE
  • Biological: ARGX-113
    Intravenous administration of ARGX-113
    Other Name: efgartigimod
  • Biological: Placebo
    Intravenous administration of placebo
Study Arms  ICMJE
  • Experimental: ARGX-113
    Intervention: Biological: ARGX-113
  • Placebo Comparator: Placebo
    Intervention: Biological: Placebo
Publications * Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. Erratum In: Lancet Neurol. 2021 Aug;20(8):e5.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 23, 2020)
167
Original Estimated Enrollment  ICMJE
 (submitted: September 11, 2018)
150
Actual Study Completion Date  ICMJE April 6, 2020
Actual Primary Completion Date April 6, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
  2. Male or female patients aged ≥ 18 years.
  3. Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa and IVb.

Other, more specific inclusion criteria are defined in the protocol

Exclusion Criteria:

  1. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
  2. Male patients who are sexually active and do not intend to use effective methods of contraception during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
  3. MGFA Class I and V patients.
  4. Patients with worsening muscle weakness secondary to concurrent infections or medications.
  5. Patients with known seropositivity or who test positive for an active viral infection at Screening with:

    • Hepatitis B Virus (HBV) (except patients who are seropositive because of HBV vaccination)
    • Hepatitis C Virus (HCV)
    • Human Immunodeficiency Virus (HIV)

Other, more specific exclusion criteria are further defined in the protocol.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   Czechia,   Denmark,   France,   Georgia,   Germany,   Hungary,   Italy,   Japan,   Netherlands,   Poland,   Russian Federation,   Serbia,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03669588
Other Study ID Numbers  ICMJE ARGX-113-1704
2018-002132-25 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party argenx
Original Responsible Party Same as current
Current Study Sponsor  ICMJE argenx
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Antonio Guglietta, MD argenx
PRS Account argenx
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP