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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/NCT05055258
Registration number
NCT05055258
Ethics application status
Date submitted
14/09/2021
Date registered
24/09/2021
Date last updated
10/10/2023
Titles & IDs
Public title
A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II
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Scientific title
Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of 3 Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II
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Secondary ID [1]
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KVD824-201
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Universal Trial Number (UTN)
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Trial acronym
KVD824-201
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Angioedema, Hereditary, Types I and II
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Blood
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Other blood disorders
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Cardiovascular
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Other cardiovascular diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - KVD824
Treatment: Drugs - Placebo to KVD824
Experimental: 300 mg KVD824 - 300 mg KVD824 twice a day for 12 weeks
Experimental: 600 mg KVD824 - Two 300 mg KVD824 tablets twice a day for 12 weeks
Experimental: 900 mg KVD824 - Three 300 mg KVD824 tablets twice a day for 12 weeks
Placebo Comparator: Placebo to KVD824 - One, two or three placebo tablets to be taken twice a day for 12 weeks
Treatment: Drugs: KVD824
KVD824 300 mg Modified-Release Tablets
Treatment: Drugs: Placebo to KVD824
Placebo to KVD824 300 mg Modified-Release Tablets
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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The Rate of Investigator-confirmed HAE attacks during the Treatment Period
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Assessment method [1]
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To examine the number of investigator-confirmed attacks whilst on treatment compared to placebo
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Timepoint [1]
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12 weeks
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Secondary outcome [1]
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Proportion of subjects without Investigator-confirmed HAE attacks during the Treatment Period.
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Assessment method [1]
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Timepoint [1]
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12 weeks
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Secondary outcome [2]
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Rate of Investigator-confirmed HAE attacks that require conventional treatment during the Treatment Period.
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Assessment method [2]
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Timepoint [2]
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12 weeks
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Secondary outcome [3]
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Angioedema Quality of Life Questionnaire (AE-QoL) total score and domain scores during the Treatment Period.
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Assessment method [3]
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AE-QoL is a quality of life questionnaire with a range of 0 (minimum) to 100 (maximum). A total score of 100 indicates worst possible impairment.
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Timepoint [3]
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12 weeks
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Secondary outcome [4]
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Angioedema Control Test (AECT) score and domain scores during the Treatment Period.
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Assessment method [4]
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AECT is a 4-item patient-reported outcome measure. The total score is from 0 (minimum) to 16 (maximum). A higher score indicates a higher level of angioedema control.
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Timepoint [4]
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12 weeks
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Secondary outcome [5]
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Proportion of subjects with an AECT score =12 at the end of the Treatment Period.
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Assessment method [5]
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AECT is a 4-item patient-reported outcome measure. The total score is from 0 (minimum) to 16 (maximum). A higher score indicates a higher level of angioedema control.
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Timepoint [5]
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12 weeks
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Eligibility
Key inclusion criteria
1. Male or female subjects 18 years of age and older.
2. Confirmed diagnosis of HAE type I or II at any time in the medical history:
1. Documented clinical history consistent with HAE (subcutaneous or mucosal,
nonpruritic swelling episodes without accompanying urticaria) AND EITHER
2. Diagnostic testing results obtained prior to randomization that confirm HAE Type
I or II: C1-INH functional level <40% of the normal level. Subjects with
functional C1-INH level 40-50% of the normal level may be enrolled if they also
have a C4 level below the normal range. Testing may be obtained from central or
local laboratories or obtained from documented historical testing results.
Subjects may be restested at anytime prior to randomization if results are
incongruent with clinical history or believed by the Investigator to be
confounded by recent prophylactic or therapeutic C1 INH use, OR
3. Documented genetic results that confirm known mutations for HAE Type I or II.
3. Subject has access to and ability to use conventional treatment for HAE attacks.
4. Subject is willing to cease any current medications being taken for HAE prophylaxis
and Investigator determines that doing so would not place the subject at any undue
safety risk.
5. Subject's last dose of attenuated androgens was at least 28 days prior to first dose
of IMP.
6. During the Run-in Period subject meets one of the following criteria:
1. Two Investigator-confirmed attacks in the first 4-week period.
2. Three Investigator-confirmed attacks in =8 weeks.
7. Subjects who are fertile and heterosexually active must adhere to contraception
requirements throughout the trial as follows:
a) Female subjects must agree to use at least one highly effective contraception
method from the Screening Visit until the end of the trial. Highly effective methods
of contraception include: i) Progestogen-only hormonal contraception associated with
inhibition of ovulation: oral/injectable/implantable (hormonal contraception that
contains estrogen including ethinylestradiol is excluded per
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion 4).
ii) Intrauterine device (IUD). iii) Intrauterine hormone-releasing system (IUS). iv)
Bilateral tubal occlusion. v) Vasectomized partner (provided that the partner is the
sole sexual partner of the female subject of childbearing potential and that the
vasectomized partner has received medical assessment of surgical success).
b) Male subjects with a female partner of childbearing potential must agree to use
condoms for the entire Treatment Period AND for 90 days following the final dose of
investigational medicinal product (IMP). Female partners are encouraged to use
contraception as outlined in Inclusion 7a) from the Screening Visit until the end of
the trial. Hormonal contraception that contains estrogen including ethinylestradiol is
acceptable for the female partner.
8. Subjects who are not fertile or not sexually active, as defined below, do not require
contraception.
1. Subjects who refrain from heterosexual intercourse during the trial if the
reliability of the heterosexual abstinence has been evaluated in relation to the
duration of the clinical trial and is the preferred and usual lifestyle of the
subject.
2. Male subjects who are surgically sterile (e.g. vasectomized with medical
assessment of surgical success).
3. Female subjects who are surgically sterile (e.g. status post hysterectomy,
bilateral oophorectomy, or bilateral tubal ligation) or post menopausal for at
least 12 months.
9. Subjects must be able to swallow trial tablets whole.
10. Subjects assessed by the Investigator must be able to appropriately receive and store
IMP, and be able to read, understand, and complete the eDiary.
11. Investigator believes that the subject is willing and able to adhere to all protocol
requirements.
12. Subject provides signed informed consent and is willing and capable of complying with
trial requirements and procedures.
Exclusion Criteria
1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1
inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III),
idiopathic angioedema, or angioedema associated with urticaria.
2. A clinically significant history of poor response to C1-INH therapy or plasma
kallikrein inhibitor therapy for the management of HAE, in the opinion of the
Investigator.
3. Use of angiotensin converting enzyme (ACE) inhibitors after the Screening Visit or
within 7 days prior to randomization.
4. Any estrogen containing medications with systemic absorption (such as oral
contraceptives including ethinylestradiol or hormonal replacement therapy) after the
Screening Visit or within 7 days prior to randomization.
5. Use of narrow therapeutic index drugs metabolized by CYP3A4 or CYP2C9 or transported
by OAT1, OCT2, and OATP1B1, starting at screening, as determined by the Investigator.
6. Use of strong CYP3A4 inhibitors and inducers during participation in the trial,
starting at the Screening Visit.
Note: These medications include but are not limited to the following:
Inhibitors: boceprevir, clarithromycin, cobicistat, dasabuvir, denoprevir,
elvitegravir, idelalisib, indinavir, itraconazole, ketoconazole, lopinavir,
nefazodone, nelfinavir ombitasvir, paritaprevir, posaconazole, ritonavir, saquinavir,
telaprevir, telithromycin, tipranavir, troleandomycin, and voriconazole.
Inducers: apalutamide, carbamazepine, enzalutamide, mitotane, phenytoin, rifampin, St.
John's Wort.
7. Inadequate organ function, including but not limited to;
1. Alanine aminotransferase (ALT) > 2x Upper limit of Normal (ULN).
2. Aspartate aminotransferase (AST) > 2x ULN.
3. Bilirubin direct > 1.25x ULN.
4. International normalized ratio (INR) > 1.2.
5. Clinically significant hepatic impairment defined as a Child-Pugh B or C.
6. Estimated glomerular filtration rate (eGFR) <60 mL/min.
8. Any clinically significant comorbidity or systemic dysfunction that in the opinion of
the Investigator would jeopardize the safety of the subject by participating in the
trial.
9. History of substance abuse or dependence that would interfere with the completion of
the trial, as determined by the Investigator.
10. Known hypersensitivity to KVD824 or placebo or to any of the excipients.
11. Any prior use of any gene therapy treatment for HAE.
12. Participation in any interventional investigational clinical trial, including an
investigational COVID-19 vaccine trial, within 4 weeks of the last dosing of
investigational drug prior to screening.
13. Any pregnant or breastfeeding subject.
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Study design
Purpose of the study
Prevention
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/09/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
27/10/2022
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Sample size
Target
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Accrual to date
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Final
33
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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KalVista Investigative Site - Campbelltown
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Recruitment postcode(s) [1]
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- Campbelltown
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Recruitment outside Australia
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United States of America
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Alabama
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United States of America
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Arizona
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California
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Colorado
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Florida
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Maryland
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Massachusetts
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Missouri
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Ohio
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Pennsylvania
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Texas
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Washington
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Bulgaria
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Sofia
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Canada
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Ontario
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Czechia
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Brno
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Czechia
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Praha
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France
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Grenoble
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France
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Paris
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Germany
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Rheinland-Pfalz
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Germany
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Berlin
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Germany
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Frankfurt am main
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Hungary
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Budapest
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Italy
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Milan
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Italy
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Padova
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New Zealand
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Auckland
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North Macedonia
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Skopje
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Puerto Rico
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San Juan
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United Kingdom
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Birmingham
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United Kingdom
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Leeds
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United Kingdom
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London
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United Kingdom
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Newcastle Upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
KalVista Pharmaceuticals, Ltd.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
A study to assess whether different doses of KVD824 are effective in preventing attacks of
Hereditary Angiodedema Type I or Type II.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05055258
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Study Director
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Address
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KalVista Pharmaceuticals
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05055258
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