SAD and MAD Study With IV and SC Doses of ARGX-117

NCT04532125 | Completed Phase 1

• 1 other identifier: ARGX-117-1901
• Study Type: interventional
• Target: 110
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase 1, first-in-human, double-blinded, randomized, placebo-controlled, escalating single and multiple dose levels trial to evaluate the safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ARGX-117 administered IV and/or SC. Up to 112 healthy, adult male and female subjects of non-childbearing potential will be enrolled in this trial.

Conditions

Healthy Volunteers

Outcome Measures

Primary Outcomes (1)

• Number of (S)AE

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

Secondary Outcomes (7)

• Area Under The Curve (AUC)

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

• Maximum serum concentrations (Cmax)

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

• Time to reach maximum serum concentrations (Tmax)

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

• Free C2 concentration

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

• Total C2 concentration

  Up to 37 weeks (arm 1) and up to 42 weeks (arm 2)

Study Arms (6)

ARGX-117 IV

EXPERIMENTAL

Subjects receiving ARGX-117 IV

Biological: ARGX-117

Placebo IV

PLACEBO COMPARATOR

Subjects receiving placebo IV

Other: Placebo

ARGX-117 PH20 SC

EXPERIMENTAL

Subjects receiving ARGX-117 PH20 SC

Biological: ARGX-117 PH20 SC

Placebo PH20 SC

PLACEBO COMPARATOR

Subjects receiving placebo PH20 SC

Other: Placebo PH20 SC

ARGX-117 + rHuPH20

EXPERIMENTAL

Subjects receiving ARGX-117 + rHuPH20

Biological: ARGX-117 + rHuPH20

Placebo + rHuPH20

PLACEBO COMPARATOR

Subjects receiving placebo + rHuPH20

Other: placebo + rHuPH20

Interventions

ARGX-117 BIOLOGICAL

Subjects treated with ARGX-117

ARGX-117 IV

Placebo OTHER

Subjects treated with placebo

Placebo IV

ARGX-117 + rHuPH20 BIOLOGICAL

Subjects treated with ARGX-117 + rHuPH20

ARGX-117 + rHuPH20

placebo + rHuPH20 OTHER

Subjects treated with placebo + rHuPH20

Placebo + rHuPH20

ARGX-117 PH20 SC BIOLOGICAL

Subjects treated with ARGX-117 PH20 SC

ARGX-117 PH20 SC

Placebo PH20 SC OTHER

Subjects treated with placebo PH20 SC

Placebo PH20 SC

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The subject is between 18 to 65 years of age, inclusive, at the time the informed consent form (ICF) is signed.
• The subject is either male, or female of nonchildbearing potential. Females in the following categories are considered a woman of nonchildbearing potential:
• Postmenopausal female: A postmenopausal state is defined as continuous amenorrhea for at least 1 year without an alternative medical cause and a follicle-stimulating hormone (FSH) measurement of \>40 IU/L. A historical pretreatment FSH measurement of\>40 IU/L is accepted as proof of a postmenopausal state for subjects on hormone replacement therapy.
• Surgically sterile female: women who have had a documented permanent sterilization procedure (ie, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy).
• Female subjects must have a negative serum pregnancy test on day -1 before IMP can be administered.
• The subject has a body mass index (BMI) within the range 18 to 30 kg/m2 and body weight 50 to 100 kg (inclusive) before IMP administration.
• The subject is able to understand the requirements of the study and provide written informed consent (including consent for the use and disclosure of research-related health information), and is willing and able to comply with the study protocol procedures (including the required study visits).
• The subject is in good physical and mental health, per the opinion of the investigator, based on medical history; physical examination findings; ECG recordings; vital sign measurements; systemic lupus erythematous (SLE) panel results; and biochemistry, hematology, INR, and urinalysis laboratory test results prior to the first dose of IMP on day 1.
• Nonsterilized male subjects who are sexually active with a female partner of childbearing potential must use effective contraception from the signing of the ICF through 260 days after the last IMP administration. A male subject practicing true sexual abstinence (as consistent with the preferred and usual lifestyle) can be included. Sterilized male subjects who have had a vasectomy and with documented absence of sperm post-procedure can be included. Male subjects are not allowed to donate sperm from signing of the ICF through 260 days after the last dose of IMP.
• The subject has abdominal skin that, in the opinion of the investigator, allows for the absorption and localized safety assessment of SC administration (applicable for dose levels with SC administration only).
• The subject agrees to discontinue and refrain from the use of all medications, including nonprescription and/or prescription medications, for at least 2 weeks before the first dose of IMP through the EOS visit on day 260 (Part A \[SAD\]), day 288 (Part B \[MAD\], cohorts 1 through 5). The occasional use of paracetamol at doses up to 2 g/day with a maximum of 10 g/2 weeks is allowed upon approval from the investigator. COVID-19 immunization recommendations are described in Section 4.3.1.1.
• The subject is a nonsmoker and does not use any nicotine-containing products. A nonsmoker is defined as an individual who has abstained from smoking for at least 3 months prior to screening.
• The subject has a negative urine drug and alcohol screen for amphetamines, barbiturates, benzodiazepines, cannabis, cocaine, opiates, methadone, tricyclic antidepressants and alcohol at screening and on day -1.
• The subject has a body temperature of 35.5 °C to 37.6 °C at screening and prior to the first dose of IMP day 1.

You may not qualify if:

• The subject has a known hypersensitivity to any of the components of the IMP, or, in the opinion of the investigator, a history of a significant allergic reaction to any drug.
• The subject has previously participated in a clinical study with efgartigimod and was administered an IMP.
• The subject has a positive serum test at screening for an active viral infection with any of the following conditions:
• Hepatitis B virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
• Hepatitis C virus (HCV) based on HCV antibody assay
• Human immunodeficiency virus
• The subject tests positively at screening for SLE as determined by the SLE test panel.
• The subject has a known family history of SLE.
• The subject has known clinically relevant immunological disorders.
• The subject has a history of severe allergic or anaphylactic reactions.
• The subject has a clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
• The subject has the presence or sequelae of gastrointestinal, liver, kidney, or other conditions known to potentially interfere with the absorption, distribution, metabolism, or excretion of IMP.
• The subject has an estimated glomerular filtration rate of \<80 mL/min/1.73 m² (calculated per the Chronic Kidney Disease Epidemiology Collaboration method) at screening.
• The subject has a history of malignancy except for:
• Adequately treated basal cell or squamous cell skin cancer

Sponsors & Collaborators

• argenx: lead

Study Sites (1)

Investigator Site 1

Groningen, Netherlands

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2020
• First Posted: August 31, 2020
• Study Start: August 3, 2020
• Primary Completion: August 26, 2022
• Study Completion: August 26, 2022
• Last Updated: September 21, 2022

Record last verified: 2022-09

Locations

NL (1)