A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema.
- Conditions
- Hereditary Angioedema Type I or IITherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2018-004489-32-HU
- Lead Sponsor
- KalVista Pharmaceuticals Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
1. Male or female adult subjects 18 years of age and older.
2. Confirmed diagnosis of HAE type I or II at anytime in the medical history
3. At least 3 documented HAE attacks in the past 93 days, as supported by medical history.
4. Access to and ability to use conventional attack treatment for attacks of HAE.
5. Adequate organ functions as defined below:
a. Hemoglobin within normal range;
b. International normalized ratio (INR)< 1.2;
c. Activated partial thromboplastin time (aPTT) = upper limit of normal (ULN);
d. Creatinine < 1.0 g;
e. Creatinine clearance (CrCl) = 60 mL/min;
f. Alanine aminotransferase (ALT) = 2x ULN;
g. Aspartate aminotransferase (AST) = 2x ULN;
h. Total bilirubin = 1.5x ULN;
i. Leucocytes = 1.5x ULN;
j. Thrombocytes = 1.5x ULN.
6. Females of childbearing potential must agree to use highly effective birth control from the last menstrual cycle prior to the start of the study drug until the end of the trial follow-up procedures.
7. Females of non-childbearing potential, defined as surgically sterile or post-menopausal for at least 12 months, do not require contraception during the study.
8. Males with female partners of childbearing potential must agree to be abstinent or else use a medically acceptable form of contraception from the Screening visit through until the end of the trial follow-up procedures. Female partners of males who are surgically sterile, must agree to use one additional form of medically acceptable contraception.
9. Provide signed informed consent and are willing and capable of complying with study requirements and procedures.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 55
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
1. Any concomitant diagnosis of another form of chronic angioedema.
2. Current use of C1INH, androgens, or tranexamic acid for HAE prophylaxis.
3. Use of angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption within 90 days prior to initial study treatment.
4. Use of androgens or antifibrinolytics within 90 days prior to initial study treatment.
5. Use of strong CYP3A4/CYP2C9 inhibitors and inducers during participation in the trial.
6. Clinically significant abnormal electrocardiogram (ECG) at Visit 1 and pre-dose at Visit 2.
7. Any clinically significant history of angina, myocardial infarction, syncope, clinically significant cardiac arrhythmias, left ventricular hypertrophy, cardiomyopathy, or any other cardiovascular abnormality.
8. Any other systemic dysfunction (e.g., gastrointestinal, renal, respiratory, cardiovascular) or significant disease or disorder which, in the opinion of the Investigator, would jeopardize the safety of the subject by taking part in the trial.
9. History of substance abuse or dependence that would interfere with the completion of the study, as determined by the Investigator.
10. Known lactose allergy or intolerance.
11. Known hypersensitivity to KVD900 or placebo or to any of the excipients.
12. Participation in an interventional investigational clinical study within 3 months or within 5 half-lives of the last dosing of investigational drug (whichever is longer) prior to initial study treatment.
13. Any pregnant or breast-feeding subject
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To investigate the efficacy of KVD900 compared to placebo in halting the progression of attacks of HAE.<br>;Secondary Objective: To investigate the safety and tolerability of KVD900<br>To investigate the pharmacokinetic (PK) profile of KVD900<br>To investigate the pharmacodynamic (PD) profile of KVD900;Primary end point(s): Primary Efficacy Endpoints:<br>• Time to use of conventional attack treatment.<br>;Timepoint(s) of evaluation of this end point: 12 hours
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary Efficacy Endpoints:<br>• Proportion of HAE attacks that progress by one level or more on the 5LS or that require conventional attack treatment within 12h of study drug.<br>• Time between treatment and (1) progression of global attack severity on the 5LS by one level or more, or (2) use of conventional attack treatment, whichever comes first within 12h;Timepoint(s) of evaluation of this end point: 12 hours