A Phase 1 first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of KP104.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)Blood - Haematological diseases
- Registration Number
- ACTRN12621000041853
- Lead Sponsor
- Kira Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 66
1. Is male or female, age 18 to 55 years, inclusive, at Screening
2. Weight of > 40 kg and < 120 kg at Screening
3. In good general health, determined by no clinically significant findings in the opinion of the Investigator from medical history, physical examination, 12-lead electrocardiogram (ECG), clinical laboratory findings, and vital signs at Screening and Check-in
4. Hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and platelet count results within the normal range at the Screening Visit; subjects 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.
5. Creatinine clearance based on the Cockcroft-Gault equation of equals to 80 ml/min
6. Females of childbearing potential and males must practice effective contraception from Screening until 28 days after the EOS visit.
7. 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.
8. Males must agree to not donate sperm for 90 days following the last dose of IP
9. Must provide evidence of prior vaccination against Neisseria meningitidis, Streptococcus pneumoniae, and Hemophilus influenzae at least 2 weeks prior to their initial dose of IP (subjects receiving vaccination during Screening and less than 2 weeks prior to their initial dose of IP are required to receive treatment with appropriate antibiotic prophylaxis until 2 weeks after vaccination); if unvaccinated and they decline vaccination, must agree to self-administer oral antibiotic prophylaxis for a total of 6 weeks following their last dose of IP
10. Able to provide Informed Consent
11. Willing and able to comply with this protocol and be available for the entire duration of the study
1. Any clinically significant underlying illness in the opinion of the Investigator
2. 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
3. Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibacterials, antivirals, or antifungals
4. History of clinically significant hematologic or bone marrow disease or blood dyscrasias
5. History of meningococcal infection
6. History of tuberculosis
7. History of asplenia (functional or anatomical)
8. Prior exposure to KP104
9. Known allergy to penicillin antibiotics or history of allergy or contraindication to required prophylactic antibiotic therapy to be used during the study
10. Known or suspected complement deficiency during screening
11. Positive serology for HBV, HCV or HIV at Screening
12. 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
13. Received any type of live attenuated vaccine < 1 month prior to Screening or is planning to receive any such live attenuated vaccine over the course of the study
14. Use of any prescription or OTC medications including food supplements and herbal medications (e.g. St. John’s wort), with the exception of contraceptive medications and as needed (prn) paracetamol (not exceeding 2 grams/day) within 7 days prior to IP administration
15. History of malignancy, except adequately treated basal cell carcinoma or in situ carcinoma of the uterine cervix
16. History of drug allergy or drug hypersensitivity, or intolerance of IV or SC injections
17. Smoking greater than 10 cigarettes per week in the 3 months prior to IP administration
18. History of excessive bleeding following trauma or medical/dental procedures
19. Any female who is pregnant or breastfeeding, or any female who is planning to become pregnant during the study and follow-up period
20. Any condition that, in the investigator’s opinion, may compromise study participation, present a safety risk to the subject, or may confound the interpretation of the study results
21. A QT duration corrected for heart rate by Fridericia's formula (QTcF) > 450 millisecond (msec) for males or > 470 msec for females based on either single or averaged QTcF values of triplicate ECGs obtained over a 3-minute interval (at Screening).
22. Currently enrolled in another investigational device or drug study, or less than 30 days have passed since ending another investigational device or drug study
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method