A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II

NCT05055258 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: KVD824-201
• Study Type: interventional
• Target: 33
• Locations: 13 countries
• Sites: 33

Brief Summary

A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II.

Conditions

Angioedema, Hereditary, Types I and II

Keywords

KVD824; KOMPLETE

Outcome Measures

Primary Outcomes (1)

• The Rate of Investigator-confirmed HAE Attacks During the Treatment Period

  To examine the number of investigator-confirmed attacks whilst on treatment compared to placebo. Given the early termination of the trial, there is insufficient enrollment to satisfy powering requirements.

  12 weeks

Secondary Outcomes (5)

• Proportion of Subjects Without Investigator-confirmed HAE Attacks During the Treatment Period.

  12 weeks

• Rate of Investigator-confirmed HAE Attacks That Require Conventional Treatment During the Treatment Period.

  12 weeks

• Angioedema Quality of Life Questionnaire (AE-QoL) Total Score During the Treatment Period (Change From Baseline)

  12 weeks

• Angioedema Control Test (AECT) Score During the Treatment Period (Change From Baseline).

  12 weeks

• Proportion of Subjects With an AECT Score ≥12 at the End of the Treatment Period.

  12 weeks

Study Arms (4)

300 mg KVD824

EXPERIMENTAL

300 mg KVD824 twice a day for 12 weeks

Drug: KVD824

600 mg KVD824

EXPERIMENTAL

Two 300 mg KVD824 tablets twice a day for 12 weeks

Drug: KVD824

900 mg KVD824

EXPERIMENTAL

Three 300 mg KVD824 tablets twice a day for 12 weeks

Drug: KVD824

Placebo to KVD824

PLACEBO COMPARATOR

One, two or three placebo tablets to be taken twice a day for 12 weeks

Drug: Placebo to KVD824

Interventions

KVD824 DRUG

KVD824 300 mg Modified-Release Tablets

300 mg KVD824; 600 mg KVD824; 900 mg KVD824

Placebo to KVD824 DRUG

Placebo to KVD824 300 mg Modified-Release Tablets

Placebo to KVD824

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects 18 years of age and older.
• Confirmed diagnosis of HAE type I or II at any time in the medical history:
• Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria) AND EITHER
• 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
• Documented genetic results that confirm known mutations for HAE Type I or II.
• Subject has access to and ability to use conventional treatment for HAE attacks.
• 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.
• Subject's last dose of attenuated androgens was at least 28 days prior to first dose of IMP.
• During the Run-in Period subject meets one of the following criteria:
• Two Investigator-confirmed attacks in the first 4-week period.
• Three Investigator-confirmed attacks in ≤8 weeks.
• Subjects who are fertile and heterosexually active must adhere to contraception requirements throughout the trial as follows:
• 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).
• Subjects who are not fertile or not sexually active, as defined below, do not require contraception.
• 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.

You may not qualify if:

• 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.
• 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.
• Use of angiotensin converting enzyme (ACE) inhibitors after the Screening Visit or within 7 days prior to randomization.
• 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.
• 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.
• 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.
• Inadequate organ function, including but not limited to;
• Alanine aminotransferase (ALT) \> 2x Upper limit of Normal (ULN).
• Aspartate aminotransferase (AST) \> 2x ULN.
• Bilirubin direct \> 1.25x ULN.
• International normalized ratio (INR) \> 1.2.
• Clinically significant hepatic impairment defined as a Child-Pugh B or C.

Sponsors & Collaborators

• KalVista Pharmaceuticals, Ltd.: lead

Study Sites (33)

KalVista Investigative Site

Birmingham, Alabama, 35294, United States

Location

KalVista Investigative Site

Scottsdale, Arizona, 85251, United States

Location

KalVista Investigative Site

La Jolla, California, 92093, United States

Location

KalVista Investigative Site

Santa Monica, California, 90404, United States

Location

KalVista Investigative Site

Centennial, Colorado, 80112, United States

Location

KalVista Investigative Site

Tampa, Florida, 33620, United States

Location

KalVista Investigative Site

Chevy Chase, Maryland, 20815, United States

Location

KalVista Investigative Site

Boston, Massachusetts, 02114, United States

Location

KalVista Investigative Site

St Louis, Missouri, 63110, United States

Location

KalVista Investigative Site

Cincinnati, Ohio, 45231, United States

Location

KalVista Investigative Site

Hershey, Pennsylvania, 17033, United States

Location

KalVista Investigative Site

Dallas, Texas, 75390, United States

Location

KalVista Investigative Site

Spokane, Washington, 99204, United States

Location

KalVista Investigative Site

Campbelltown, New South Wales, Australia

Location

KalVista Investigative Site

Sofia, Bulgaria

Location

KalVista Investigative Site

North York, Ontario, Canada

Location

KalVista Investigative Site

Brno, Czechia

Location

KalVista Investigative Site

Prague, Czechia

Location

KalVista Investigative Site

Grenoble, France

Location

KalVista Investigative Site

Paris, France

Location

KalVista Investigative Site

Mainz, Rhineland-Palatinate, Germany

Location

KalVista Investigative Site

Berlin, Germany

Location

KalVista Investigative Site

Frankfurt am Main, Germany

Location

KalVista Investigative Site

Budapest, Hungary

Location

KalVista Investigative Site

Milan, Italy

Location

KalVista Investigative Site

Padova, Italy

Location

KalVista Investigative Site

Grafton, Auckland, New Zealand

Location

KalVista Investigative Site

Skopje, North Macedonia

Location

KalVista Investigative Site

San Juan, Puerto Rico

Location

KalVista Investigative Site

Birmingham, United Kingdom

Location

KalVista Investigative Site

Leeds, United Kingdom

Location

KalVista Investigative Site

London, United Kingdom

Location

KalVista Investigative Site

Newcastle upon Tyne, United Kingdom

Location

MeSH Terms

Conditions

Hereditary Angioedema Types I and II

Condition Hierarchy (Ancestors)

Angioedemas, Hereditary; Angioedema; Vascular Diseases; Cardiovascular Diseases; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Results Point of Contact

• Title: Vice President, Clinical
• Organization: KalVista Pharmaceuticals Ltd.

clinical@kalvista.com

+18579990075

Study Officials

• Study Director

  KalVista Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2021
• First Posted: September 24, 2021
• Study Start: November 18, 2021
• Primary Completion: October 27, 2022
• Study Completion: October 27, 2022
• Last Updated: May 6, 2026
• Results First Posted: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); CZ (2); DE (3); NZ (1); AU (1); FR (2); NO (1); HU (1); IT (2); PR (1); GB (4); US (13); BU (1)