A First-in-Human, Randomized, Double-Blinded, Placebo-Controlled Trial in Healthy Subjects to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Single and Multiple Ascending Intravenous Dose Levels of ARGX-117 and Subcutaneous Dose Levels of ARGX-117 Co-Mixed with Recombinant Human Hyaluronidase PH20

• Register: OMON
• Last refreshed on: 15 April 2024
• Main ID: NL-OMON52853
• Date of registration: 2020-02-20
• Prospective Registration: Yes
• Primary sponsor: argenx BV
• Public title: A First-in-Human, Randomized, Double-Blinded, Placebo-Controlled Trial in Healthy Subjects to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Single and Multiple Ascending Intravenous Dose Levels of ARGX-117 and Subcutaneous Dose Levels of ARGX-117 Co-Mixed with Recombinant Human Hyaluronidase PH20
• Scientific title: A First-in-Human, Randomized, Double-Blinded, Placebo-Controlled Trial in Healthy Subjects to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Single and Multiple Ascending Intravenous Dose Levels of ARGX-117 and Subcutaneous Dose Levels of ARGX-117 Co-Mixed with Recombinant Human Hyaluronidase PH20 - SAD and MAD study with IV and SC doses of ARGX-117
• Date of first enrolment: 2020-03-10
• Target sample size: 112
• Recruitment status: Completed
• URL: https://onderzoekmetmensen.nl/en/trial/52853
• Study type: Interventional
• Study design: Randomized controlled trial, Double blinded (masking used), Placebo, Parallel, Treatment

Countries of recruitment

Netherlands

Contacts

• Affiliation: argenx BV

• Affiliation: argenx BV

Key inclusion & exclusion criteria

Inclusion criteria:

1. The subject is between 18-65 years of age, inclusive, at the time the
informed consent form is signed.
2. The subject is either male or female of non-childbearing potential. Females
in the following categories are considered a woman of non-childbearing
potential:
a. 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.
b. Surgically sterile female: women who have had a documented permanent
sterilization procedure (ie, hysterectomy, bilateral salpingectomy, or
bilateral oophorectomy).
3. Female subjects must have a negative serum pregnancy test on day -1 before
IMP can be administered.
4. The subject has a body mass index (BMI) within the range 18-30 kg/m2 and
body weight 50-100 kg (inclusive) before IMP administration.
5. The subject is able to understand the requirements of the trial 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
trial protocol procedures (including the required trial visits).
Further criteria apply.

Exclusion criteria:

1. The subject has a known hypersensitivity to one of the components of the
IMP, or, in the opinion of the investigator, a history of a significant
allergic reaction to any drug.
2. The subject has previously participated in a clinical trial with
efgartigimod and was administered an IMP.
3. The subject has a positive serum test at screening for an active viral
infection with any of the following conditions:
a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
b. Hepatitis C virus (HCV) based on HCV antibody assay
c. Human immunodeficiency virus (HIV)
4. The subject tests positively at screening for SLE as determined by the SLE
test panel.
5. The subject has a known family history of SLE.
Further criteria apply.

Age minimum: 18
Age maximum: 64
Gender:

Health Condition(s) or Problem(s) studied

autoimmune diseases

10003816

Intervention(s)

Part A:
In Groups 1 to 8, ARGX-117 or placebo will be given as a 2-hour intravenous
infusion or ARGX-117 or placebo will be given in combination with rHuPH20 will
as an injection under the skin (subcutaneous) or infusion under the skin over
approximately 1 hour. Group 9 is an optional group. Based on results in the
preceding groups, it will be determined whether ARGX-117 or placebo is
administered intravenously or subcutaneously in Group 9.

Group Study compound Dose# Route of administration
1 ARGX-117 or placebo 0.1 mg/kg Intravenous
2 ARGX-117 or placebo 0.5 mg/kg Intravenous
3 ARGX-117 or placebo 2.5 mg/kg Intravenous
4 ARGX-117 or placebo 10 mg/kg Intravenous
5 ARGX-117 or placebo 30 mg/kg Intravenous
6 ARGX-117 or placebo 60 mg/kg Intravenous
7 ARGX-117 or placebo 60 mg/kg Subcutaneous
8 ARGX-117 + rHuPH20 or
Placebo + rHuPH20 80 mg/kg Intravenous
9 ARGX-117 (+ rHuPH20) or
Placebo (+ rHuPH20) 15mg/kg subcutaneous

Part B:
In Group 1: the volunteer will receive ARGX-117 or placebo 4 times, with at
least 1 week between each administration.
In Group 2 and 3: the volunteer will receive ARGX-117 or placebo 3 times, with
at least 1 week between each administration.
In Group 4 and 5: the volunteer will receive ARGX 117 or placebo 4 times in
combination with rHuPH20 as an injection under the skin

Group Day Study compound Dose# Route of administration
1 1 ARGX-117 or placebo 10 mg/kg Intravenous
8 10 mg/kg
15 10 mg/kg
22 10 mg/kg.

2 1 ARGX-117 or placebo 60 mg/kg Intravenous
8 10 mg/kg
22 10 mg/kg.

3 1 ARGX-117 or placebo 10 mg/kg Intravenous.
8 ARGX-117 or placebo 50 mg/kg Intravenous.
22 ARGX-117 or Placebo 20 mg/kg Int

Primary Outcome(s)

Part A (single ascending dose [SAD]): To evaluate the safety and tolerability

of single ascending intravenous (IV) doses of ARGX-117 and subcutaneous (SC)

doses of ARGX-117 co-mixed with recombinant human hyaluronidase PH20 (rHuPH20)

in healthy adult subjects, compared to placebo.



Part B (multiple ascending dose [MAD]): To evaluate the safety and tolerability

of multiple ascending IV doses of ARGX-117 and SC doses of ARGX-117 co-mixed

with rHuPH20 in healthy adult subjects, compared to placebo.

Secondary Outcome(s)

Part A (SAD): To investigate the pharmacokinetics (PK), pharmacodynamic (PD),

and immunogenicity effects of single ascending IV doses of ARGX-117 and SC

doses of ARGX-117 co-mixed with rHuPH20 in healthy adult subjects.



Part B (MAD): To investigate the PK, PD, and immunogenicity effects of multiple

ascending IV doses of ARGX-117 and SC doses of ARGX-117 co-mixed with rHuPH20

in healthy adult subjects.

Secondary ID(s)

NCT04532125

EUCTR2019-004986-42-NL

NL72813.056.20

Source(s) of Monetary Support

Pharmaceutical Industry

Ethics review

Status: Approved
Approval date: 20/02/2020
Contact:

Status: Approved
Approval date: 02/03/2020
Contact:

Status: Approved
Approval date: 16/06/2020
Contact:

Status: Approved
Approval date: 16/07/2020
Contact:

Status: Approved
Approval date: 07/08/2020
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Status: Approved
Approval date: 13/08/2020
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Status: Approved
Approval date: 03/12/2020
Contact:

Status: Approved
Approval date: 07/12/2020
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Status: Approved
Approval date: 12/01/2021
Contact:

Status: Approved
Approval date: 28/01/2021
Contact:

Status: Approved
Approval date: 12/03/2021
Contact:

Status: Approved
Approval date: 26/03/2021
Contact:

Status: Approved
Approval date: 26/06/2021
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Status: Approved
Approval date: 30/06/2021
Contact:

Status: Approved
Approval date: 14/10/2021
Contact:

Status: Approved
Approval date: 22/10/2021
Contact:

Status: Approved
Approval date: 03/12/2022
Contact:

Status: Approved
Approval date: 06/12/2022
Contact:

Results

• Date Posted: 08/06/2023