Rare DiseasesPhase 1NCT04765553
A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
A Phase 1 study testing NI-0501 and Saline in people with Rare Diseases, run by Swedish Orphan Biovitrum at 1 site in Japan.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Rare Diseases
- What is being tested?
- NI-0501 and Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteJapan
- Study dates
- 14 Jan 2021to 25 Jul 2021
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
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- 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.
- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago.
- Signed informed consent.
Who cannot join
- 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.
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- 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
- Component blood donation within 2 weeks before the screening test and within 2 weeks before the first study drug administration
- Collection of 200 mL or more of blood (blood donation, etc.) from 4 weeks before the screening test until admission
- Male subject who has experience of collection of 400 mL or more of blood (blood donation, etc.) from 12weeks before the screening test until admission.
- Female subject who has experience of collection of 400 mL or more of blood (blood donation, etc.) from 16weeks before the screening test until admission.
- Usage of any prescription drugs within 2 weeks or over-the-counter medication including herbal supplements (with the exception of multi-vitamins) within 1 week before IMP administration without prior approval from the investigator
- Positive pregnancy test at screening or Day -1
- Any circumstances or conditions, which, in the opinion of the investigator, may affect the subject's full participation in the study or compliance with the protocol
- Enrollment in another concurrent clinical interventional study, or intake of another IMP, within four months or 5 half-lives (of the other IMP) prior to inclusion in this study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-blinded, Placebo-controlled, Single Center, Phase I Study to Evaluate Pharmacokinetics, Pharmacodynamics and Safety of Emapalumab After a Single Intravenous Dose in Japanese Healthy Volunteers.
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.
Read the detailed description
This is a randomized, placebo-controlled and double-blinded study to evaluate the PK, PD and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects, performed in Japan. The subjects, 8 in total, will be randomized to receive either emapalumab or matching placebo in a 3:1 ratio (emapalumab: placebo).
- Conditions
- Rare Diseases
- Being tested
- NI-0501, Saline
- Medical subject headings
- Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NI-0501Drug
Also called Gamifant
emapalumab single i.v infusion (1 mg/kg)
SalineDrug
Also called Placebo
Saline single i.v infusion
| Study group | Role | What they receive |
|---|---|---|
| emapalumab | Active comparator | emapalumab i.v infusion Receives: Drug: NI-0501 |
| Placebo | Placebo comparator | Saline i.v. infusion Receives: Drug: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Maximum Observed Concentration of Emapalumab
The maximum observed concentration of emapalumab (Cmax)
Measured: 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)
Measured: 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))
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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
Total number of reported adverse events
Measured: Continuously from start of emapalumab infusion up to 14 weeks
Change in Levels of Aspartate Aminotransferase
Change from baseline in levels of Aspartate aminotransferase (AST)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Alanine Aminotransferase
Change from baseline in levels of Alanine aminotransferase (ALT)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Direct Bilirubin
Change from baseline in levels of Direct Bilirubin
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Total Bilirubin
Change from baseline in levels of Total Bilirubin
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Uric Acid
Change from baseline in levels of Uric acid
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Alkaline Phosphatase
Change from baseline in levels of Alkaline phosphatase
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Total Protein
Change from baseline in levels of Total protein
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Albumin
Change from baseline in levels of Albumin
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Prothrombin Time/International Normalized Ratio
Change from baseline in levels of Prothrombin Time/International Normalized Ratio (PTINR)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Fibrinogen
Change from baseline in levels of Fibrinogen
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Complement C3
Change from baseline in levels of Complement C3
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Creatinine
Change from baseline in levels of Creatinine
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of C-reactive Protein
Change from baseline in levels of C-reactive protein (CRP)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Sodium
Change from baseline in levels of Sodium
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Potassium
Change from baseline in levels of Potassium
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Calcium
Change from baseline in levels of Calcium
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Glucose
Change from baseline in levels of Glucose
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of HDL
Change from baseline in levels of HDL
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of LDL
Change from baseline in levels of LDL
Measured: Baselilne, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of BUN/Urea Haematology
Change from baseline in levels of BUN/Urea haematology
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Hemoglobin
Change from baseline in levels of Hemoglobin
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Hematocrit
Change from baseline in levels of Hematocrit
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Platelet Count
Change from baseline in levels of Platelet count
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Neutrophils
Change from baseline in levels of Neutrophils
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Red Blood Cells
Change from baseline in levels of Red blood cells
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Immunoglobulin Levels
Change from baseline in levels of Immunoglobulin levels (IgG)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Coagulation Profile
Change from baseline in Activated Partial Thromboplastin Clotting Time (APTT)
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Presence of Anti-drug Antibodies and Neutralizing Antibodies
Presence of anti-drug antibodies (ADA) and neutralizing antibodies (nAb)
Measured: From Day 1 to Week 14
Change in Levels of Complement C4
Change from baseline in levels of Complement C4
Measured: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Presence of Neutralizing Antibodies
Presence of neutralizing antibodies (nAb)
Measured: From Day 1 to Week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2021
- Study start14 Jan 2021
- Primary completion25 Jun 2021
- Study completion25 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Rare DiseasesStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Care provider
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kenichi Furihata, MD, PhD, Principal investigator, P-One Clinic, 4F, View Tower Hachioji, 8-1, Yokamachi, Hachioji-shi, Tokyo 192-0071, Japan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04765553
- Sponsor's protocol ID
- Sobi.emapalumab-102
- Status last verified
- Nov 2023
- First submitted
- 18 Jan 2021
- Last updated
- 30 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blinded, Placebo-controlled, Single Center, Phase I Study to Evaluate Pharmacokinetics, Pharmacodynamics and Safety of Emapalumab After a Single Intravenous Dose in Japanese Healthy Volunteers.
- ClinicalTrials.gov ID
- NCT04765553
- Acronym / sponsor ID
- Sobi.emapalumab-102
- Conditions
- Rare Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 14 Jan 2021 (actual)
- Primary completion
- 25 Jun 2021 (actual)
- Study completion
- 25 Jul 2021 (actual)
- First posted
- 21 Feb 2021 (actual)
- Results
- Posted 24 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a randomized, placebo-controlled and double-blinded study to evaluate the PK, PD and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects, performed in Japan. The subjects, 8 in total, will be randomized to receive either emapalumab or matching placebo in a 3:1 ratio (emapalumab: placebo).
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| emapalumab | Active comparator | Drug: NI-0501emapalumab i.v infusion |
| Placebo | Placebo comparator | Drug: SalineSaline i.v. infusion |
Who may qualify
- Ages
- 20 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (17 listed)
- 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:
- 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).
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NI-0501Also: Gamifantemapalumab single i.v infusion (1 mg/kg) | Drug | emapalumab |
| SalineAlso: PlaceboSaline single i.v infusion | Drug | Placebo |
Primary outcome
The Maximum Observed Concentration of Emapalumab
Time frame: 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 maximum observed concentration of emapalumab (Cmax)
The Time at Which the Maximum Concentration of Emapalumab is Observed
Time frame: 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
The time at which the maximum concentration of emapalumab is observed (Tmax)
Concentration of Emapalumab at End of Infusion
Time frame: 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 (CEnd of inf))
Area Under the Plasma Concentration-time Curve
Time frame: 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 from emapalumab injection to time of last measurable concentration (AUClast)
Area Under the Concentration-time Curve Extrapolated to Infinity
Time frame: 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 from emapalumab injection extrapolated to infinity (AUCinf)
Emapalumab Elimination Half-life
Time frame: 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 (t1/2)
Apparent Total Body Clearance of Emapalumab From Plasma
Time frame: 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 (CL)
Steady State Volume of Distribution
Time frame: 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 volume of distribution at steady state (Vss)
31 secondary outcomes
Overall Summary of Adverse Events
Time frame: Continuously from start of emapalumab infusion up to 14 weeks
Change in Levels of Aspartate Aminotransferase
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Alanine Aminotransferase
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Direct Bilirubin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Total Bilirubin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Uric Acid
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Alkaline Phosphatase
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Total Protein
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Albumin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Prothrombin Time/International Normalized Ratio
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Fibrinogen
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Complement C3
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Creatinine
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of C-reactive Protein
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Sodium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Potassium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Calcium
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Glucose
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of HDL
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of LDL
Time frame: Baselilne, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of BUN/Urea Haematology
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Hemoglobin
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Hematocrit
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Platelet Count
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Neutrophils
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Red Blood Cells
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Immunoglobulin Levels
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Change in Levels of Coagulation Profile
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Presence of Anti-drug Antibodies and Neutralizing Antibodies
Time frame: From Day 1 to Week 14
Change in Levels of Complement C4
Time frame: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)
Presence of Neutralizing Antibodies
Time frame: From Day 1 to Week 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenichi Furihata, MD, PhD principal investigator
P-One Clinic, 4F, View Tower Hachioji, 8-1, Yokamachi, Hachioji-shi, Tokyo 192-0071, Japan
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NI-0501 and Saline in people with Rare Diseases, run by Swedish Orphan Biovitrum at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Nov 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Maximum Observed Concentration of Emapalumab (time frame: 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 study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2023.