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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05490017




Registration number
NCT05490017
Ethics application status
Date submitted
4/08/2022
Date registered
5/08/2022
Date last updated
1/03/2023

Titles & IDs
Public title
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
Scientific title
SYNERGY-1: A Phase 1 First-in-human, Randomized, Double Blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 in Healthy Subjects
Secondary ID [1] 0 0
KP104-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KP104
Treatment: Drugs - Placebo

Experimental: Part 1: Single Ascending Dose Cohort 1 -

Experimental: Part 1: Single Ascending Dose Cohort 2 -

Experimental: Part 1: Single Ascending Dose Cohort 3 -

Experimental: Part 1: Single Ascending Dose Cohort 4 -

Experimental: Part 1: Single Ascending Dose Cohort 5 -

Experimental: Part 1: Single Ascending Dose Cohort 6 -

Experimental: Part 1: Single Ascending Dose Cohort 7 -

Experimental: Part 2: Multiple Ascending Dose Cohort 1 -

Experimental: Part 2: Multiple Ascending Dose Cohort 2 -

Experimental: Part 2: Multiple Ascending Dose Cohort 3 -

Placebo Comparator: Part 1: Placebo -

Placebo Comparator: Part 2: Placebo -


Treatment: Drugs: KP104
Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Treatment: Drugs: Placebo
Participants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Up to Day 85
Primary outcome [2] 0 0
Number of participants reporting Treatment Emergent Serious Adverse Events (TESAEs)
Timepoint [2] 0 0
Up to Day 85
Primary outcome [3] 0 0
Number of participants with Dose-limiting toxicities (DLT)
Timepoint [3] 0 0
Up to Day 85
Primary outcome [4] 0 0
Number of participants reporting AEs of Special interests (AESIs)
Timepoint [4] 0 0
Up to Day 85
Secondary outcome [1] 0 0
Maximum concentration (Cmax) of KP104
Timepoint [1] 0 0
Up to Day 29
Secondary outcome [2] 0 0
Area under the concentration-time profile (AUC) of KP104
Timepoint [2] 0 0
Up to Day 29
Secondary outcome [3] 0 0
Change from baseline in total and free serum C5 levels
Timepoint [3] 0 0
Baseline and up to Day 29
Secondary outcome [4] 0 0
Change from baseline in rabbit red blood cell (RBC) assay
Timepoint [4] 0 0
Baseline and up to Day 29

Eligibility
Key inclusion criteria
- Weight of > 40 kilograms (kg) and < 120 kg at Screening.

- In good general health, determined by no clinically significant findings in the
opinion of the Investigator from medical history, physical examination, 12-lead ECG,
clinical laboratory findings, and vital signs at Screening and Check-in.

- Hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and
platelet count results within the normal range at the Screening Visit; participants
with Gilbert's disease with associated abnormalities of liver function tests are
eligible for enrollment. Tests may be repeated at the discretion of the Investigator
to confirm abnormalities.

- Creatinine clearance based on the Cockcroft-Gault equation of >= 80 milliliters per
minute (ml/min).

- Females of childbearing potential and males must practice effective contraception from
Screening until 28 days after the end of study (EOS) visit.

- Females of childbearing potential must have a negative pregnancy test at Screening and
within 24 hours prior to dosing of study drug; for post-menopausal subjects, a blood
sample will also be tested for follicle stimulating hormone to confirm post-menopausal
status.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Any clinically significant underlying illness in the opinion of the Investigator.

- Any history or sign of significant chronic active or recurrent infection, or screening
laboratory evidence consistent with a significant chronic active or recurrent
infection requiring treatment with antibacterials, antivirals, or antifungals.

- Treatment of any infection with IV (within 30 days of Screening) or oral (within 14
days of Screening) antibacterials, antivirals, or antifungals.

- History of clinically significant hematologic or bone marrow disease or blood
dyscrasias.

- History of meningococcal infection.

- History of tuberculosis.

- History of asplenia (functional or anatomical).

- Prior exposure to KP104.

- Known allergy to penicillin antibiotics or history of allergy or contraindication to
required prophylactic antibiotic therapy to be used during the study.

- Known or suspected complement deficiency during screening.

- Positive serology for Hepatitis B virus (HBV), hepatitis C virus (HCV) or human
immunodeficiency virus (HIV) at Screening.

- History of drug or alcohol abuse within 1 year of Screening in the opinion of the
investigator, or a positive test for drugs of abuse or alcohol at Screening or
Check-in.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/09/2022
Sample size
Target
Accrual to date
Final
80
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
CMAX Clinical Research - Adelaide
Recruitment postcode(s) [1] 0 0
- Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Kira Pharmacenticals (US), LLC.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate safety, tolerability, immunogenicity,
pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study
will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human
(FIH) study of KP104 and Part 2, multiple ascending dose (MAD).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05490017
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries