A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.

NCT04765553 | Completed Phase 1 Results

• 1 other identifier: Sobi.emapalumab-102
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects.

Conditions

Rare Diseases

Outcome Measures

Primary Outcomes (8)

• The Maximum Observed Concentration of Emapalumab

  The maximum observed concentration of emapalumab (Cmax)

  Day 1 preinfusion, 1hr, 2hrs, 4hrs, 8hrs, 10hrs post dose, day 2, 3, 5, 8, week 2, 4, 6, 8, 10, 12, study completion week 14 or Withdrawal

• The Time at Which the Maximum Concentration of Emapalumab is Observed

  The time at which the maximum concentration of emapalumab is observed (Tmax)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Concentration of Emapalumab at End of Infusion

  Concentration of emapalumab at end of infusion (CEnd of inf))

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Area Under the Plasma Concentration-time Curve

  Area under the plasma concentration-time curve from emapalumab injection to time of last measurable concentration (AUClast)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Area Under the Concentration-time Curve Extrapolated to Infinity

  Area under the plasma concentration-time curve from emapalumab injection extrapolated to infinity (AUCinf)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Emapalumab Elimination Half-life

  Emapalumab elimination half-life (t1/2)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Apparent Total Body Clearance of Emapalumab From Plasma

  Apparent total body clearance of emapalumab from plasma (CL)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

• Steady State Volume of Distribution

  Apparent volume of distribution at steady state (Vss)

  Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Secondary Outcomes (31)

• Overall Summary of Adverse Events

  Continuously from start of emapalumab infusion up to 14 weeks

• Change in Levels of Aspartate Aminotransferase

  Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

• Change in Levels of Alanine Aminotransferase

  Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

• Change in Levels of Direct Bilirubin

  Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

• Change in Levels of Total Bilirubin

  Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Study Arms (2)

emapalumab

ACTIVE COMPARATOR

emapalumab i.v infusion

Drug: NI-0501

Placebo

PLACEBO COMPARATOR

Saline i.v. infusion

Drug: Saline

Interventions

NI-0501 DRUG

emapalumab single i.v infusion (1 mg/kg)

Also known as: Gamifant

emapalumab

Saline DRUG

Saline single i.v infusion

Also known as: Placebo

Placebo

Eligibility Criteria

• Age: 20 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy Japanese (male and female) subjects between 20 and 50 years (inclusive).
• Body weight greater than 45 kg (female) or 50 kg (male) and a body mass index (BMI) \>18 kg/m2 and \< 30 kg/m2 (BMI= weight (kg) / height (m)²)
• Vital signs in the following range:
• Axillary body temperature: 35.2 - 37.5℃
• Heart rate (after at least 3 minutes of rest, measured in the supine position): 40-100 bpm
• BP \< 140/80, mean of 3 readings after 15 minutes rest
• Haemoglobin level equal or above 11 g/dL in females and 13 g/dL in males.
• Subject having C-reactive protein (CRP) levels within the normal range (local laboratory range).
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant having agreed to use highly effective methods of contraception during dosing and for 6 months after receiving IMP.
• Highly effective contraception methods include:
• Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
• Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
• Male sterilization (at least 6 months prior to screening). For female patients on the study, the vasectomized male partner should be the sole partner for that patient, otherwise highly effective methods to be applied.
• Use of oral (estrogen and progesterone) hormonal method of contraception, or placement of an intrauterine device (IUD) or intrauterine system (IUS)
• In case of use of oral contraception women should have been stable on the same brand (or generic equivalent) for a minimum of 3 months before taking study treatment.

You may not qualify if:

• Any clinically significant abnormality in the results of the safety laboratory tests. Subjects presenting a minor deviation from laboratory ranges could be enrolled if the investigator judge it to be non-clinically significant
• Any clinically significant abnormality on the screening electrocardiogram (ECG), as judged by the investigator
• History or clinical evidence of any disease and/or existence of any surgical or medical condition that might interfere with the absorption, distribution, metabolism or excretion of the study drugs
• Actual presence or occurrence of any bacterial, viral, parasitic or fungal infection within the 4 weeks preceding IMP infusion
• Positive results from serology examination for Hepatitis B surface antigen (HBsAg), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), syphilis (TP-antigen and RPR) or pregnancy
• Positive stool test for Shigella or salmonella infection.
• Positive results from Sars-CoV-2 screening within 96 hours prior to randomization
• History or clinical evidence suggestive of active or latent tuberculosis at screening. (i.e. test positive to the interferon gamma (IFNγ)-release assay)
• History or presence of any severe allergic reactions
• History of hypersensitivity or allergy to any component of emapalumab and/or valaciclovir hydrochloride
• History or presence of any malignancy
• History or presence of drug or alcohol abuse
• Subject with a smoking history within the last 6 months prior to the time of screening
• Immunization with a live vaccine within 6 weeks prior to receiving IMP and 12 weeks after IMP infusion
• Experience of collected blood corresponding to any of the following

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (1)

P-One Clinic

Tokyo, Japan

Location

MeSH Terms

Conditions

Rare Diseases

Interventions

Emapalumab; Sodium Chloride

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Veronica Asnaghi
• Organization: Swedish Orphan Biovitrum AB

Veronica.Asnaghi@Sobi.com

+41 61 508 72 13

Study Officials

• Kenichi Furihata, MD, PhD

  P-One Clinic, 4F, View Tower Hachioji, 8-1, Yokamachi, Hachioji-shi, Tokyo 192-0071, Japan

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: CARE PROVIDER
• Masking Details: Double blind
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2021
• First Posted: February 21, 2021
• Study Start: January 14, 2021
• Primary Completion: June 25, 2021
• Study Completion: July 25, 2021
• Last Updated: November 30, 2023
• Results First Posted: November 24, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)