Trial Outcomes & Findings for Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II (NCT NCT06467084)

Last Updated: 2026-08-05

Results Overview

A TEAE was defined as an adverse event (AE) that met any of the following conditions: (1) began on or after the first dose of IMP, (2) began before the first dose of IMP and increased in severity on or after first dose, (3) was completely missing a start date and the stop date, (4) was completely missing a start date and the stop date was on or after the first dose of IMP. An on-treatment TEAE was defined as any TEAE occurring within 3 days of IMP administration. A treatment-related TEAE was an AE considered related to the IMP by the investigator. A serious AE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required in-patient hospitalization or prolongation, resulted in significant disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event (based upon medical and scientific judgment).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

36 participants

Primary outcome timeframe

From first dose of IMP until the Final/Early Termination (ET) Visit, up to a maximum of 54 weeks.

Results posted on

2026-08-05

Participant Flow

Pediatric participants (aged 2 to 11 years) with hereditary angioedema (HAE) Type I or II were enrolled at trial sites in Canada, Germany, France, Israel, Italy, Japan, and the United States (US). Participants enrolled under protocol v1.0 to v3.3 received sebetralstat 300 mg equivalent dosing (ED). Following a US-specific protocol amendment, dose levels were increased; US-only participants who continued under protocol v3.4 had a Dose Increase Visit and received sebetralstat 600 mg ED thereafter.

Dose levels for both sebetralstat 300 mg ED and 600 mg ED were selected by the investigator, based on the participant's weight. The screening visit occurred up to 8 weeks before enrolment. Participants were allowed to treat HAE attacks for up to 1 year following the first attack treated with sebetralstat, or until the participant reached 12 years of age, or the trial was completed.

Participant milestones

Participant milestones
Measure	Weight Group ≥9.5 to <20 kg Participants weighing ≥9.5 to \<20 kg received a 75 mg dose of sebetralstat (matching 300 mg ED), administered as an orally disintegrating tablet (ODT). From protocol v3.4, US-only participants received a 150 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of investigational medicinal product (IMP) could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥20 to ≤45 kg Participants weighing ≥20 to ≤45 kg received a 150 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 300 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥45 kg Participants weighing ≥45 kg received a 300 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 600 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).
Overall Study STARTED	3	27	6
Overall Study Received sebetralstat 300 mg ED (protocol v1.0 - 3.3)	3	27	6
Overall Study Received sebetralstat 600 mg ED (protocol v3.4; US only)	0	6	1
Overall Study COMPLETED	3	25	3
Overall Study NOT COMPLETED	0	2	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Weight Group ≥9.5 to <20 kg Participants weighing ≥9.5 to \<20 kg received a 75 mg dose of sebetralstat (matching 300 mg ED), administered as an orally disintegrating tablet (ODT). From protocol v3.4, US-only participants received a 150 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of investigational medicinal product (IMP) could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥20 to ≤45 kg Participants weighing ≥20 to ≤45 kg received a 150 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 300 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥45 kg Participants weighing ≥45 kg received a 300 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 600 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).
Overall Study Lost to Follow-up	0	1	1
Overall Study Withdrawal by Subject	0	1	2

Baseline Characteristics

Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Weight Group ≥9.5 to <20 kg n=3 Participants Participants weighing ≥9.5 to \<20 kg received a 75 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 150 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥20 to ≤45 kg n=27 Participants Participants weighing ≥20 to ≤45 kg received a 150 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 300 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Weight Group ≥45 kg n=6 Participants Participants weighing ≥45 kg received a 300 mg dose of sebetralstat (matching 300 mg ED), administered as an ODT. From protocol v3.4, US-only participants received a 600 mg dose of sebetralstat (matching 600 mg ED), administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack. No more than 2 doses were permitted within a 24-hour period for participants enrolled under protocol v3.4 and receiving sebetralstat 600 mg ED (US only).	Total n=36 Participants Total of all reporting groups
Age, Continuous	5.0 years n=20 Participants	8.0 years n=20 Participants	9.5 years n=40 Participants	8.0 years n=6 Participants
Age, Customized 2 - 5 years	3 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	3 Participants n=6 Participants
Age, Customized 6 - 11 years	0 Participants n=20 Participants	27 Participants n=20 Participants	6 Participants n=40 Participants	33 Participants n=6 Participants
Sex: Female, Male Female	2 Participants n=20 Participants	13 Participants n=20 Participants	3 Participants n=40 Participants	18 Participants n=6 Participants
Sex: Female, Male Male	1 Participants n=20 Participants	14 Participants n=20 Participants	3 Participants n=40 Participants	18 Participants n=6 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=20 Participants	1 Participants n=20 Participants	0 Participants n=40 Participants	1 Participants n=6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=20 Participants	21 Participants n=20 Participants	5 Participants n=40 Participants	29 Participants n=6 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants	5 Participants n=20 Participants	1 Participants n=40 Participants	6 Participants n=6 Participants
Race American Indian or Alaska Native	0 Participants n=20 Participants	0 Participants n=20 Participants	1 Participants n=40 Participants	1 Participants n=6 Participants
Race Asian	0 Participants n=20 Participants	1 Participants n=20 Participants	1 Participants n=40 Participants	2 Participants n=6 Participants
Race Black	0 Participants n=20 Participants	1 Participants n=20 Participants	0 Participants n=40 Participants	1 Participants n=6 Participants
Race Native Hawaiian or Pacific islander	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	0 Participants n=6 Participants
Race White	3 Participants n=20 Participants	20 Participants n=20 Participants	3 Participants n=40 Participants	26 Participants n=6 Participants
Race Other	0 Participants n=20 Participants	1 Participants n=20 Participants	0 Participants n=40 Participants	1 Participants n=6 Participants
Race Not Reported	0 Participants n=20 Participants	4 Participants n=20 Participants	1 Participants n=40 Participants	5 Participants n=6 Participants

PRIMARY outcome

Timeframe: From first dose of IMP until the Final/Early Termination (ET) Visit, up to a maximum of 54 weeks.

Population: The SAF included all participants who received at least 1 dose of IMP.

Outcome measures

Outcome measures
Measure	Sebetralstat 300 mg ED n=36 Participants Participants received sebetralstat 300 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 75 mg, those weighing ≥20 to ≤45 kg received 150 mg, and those weighing \>45 kg received 300 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack.	Sebetralstat 600 mg ED n=7 Participants Participants received sebetralstat 600 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 150 mg, those weighing ≥20 to ≤45 kg received 300 mg, and those weighing \>45 kg received 600 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack (with no more than 2 doses permitted within a 24-hour period).
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any TEAE	20 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any treatment-related TEAE	0 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any on-treatment TEAE	11 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any serious TEAE	0 Participants	0 Participants

SECONDARY outcome

Timeframe: At 0.5 hours (±5 min), 2 hours (±15 min), and 4 hours (±15 min) post-dose. PK assessments for sebetralstat 300 mg ED were conducted at the Enrollment Visit, and for 600 mg ED at the Dose Increase Visit (up to 12.6 months after Enrollment Visit).

Population: The PK Set included all participants who provided at least 1 PK sample. Only participants with available data were included in the analysis at each specified timepoint.

During the Enrollment Visit, participants were dosed with IMP (sebetralstat 300 mg ED) and monitored for tolerability. Pharmacokinetic (PK) samples were collected via venous access or capillary collection (eg, finger prick) at specified timepoints. For participants enrolled under protocol v3.4 who received sebetralstat 600 mg ED, a second PK assessment was performed at the Dose Increase Visit. The Dose Increase Visit occurred as soon as possible after implementation of the protocol amendment and could occur at any time during the participant's participation in the trial.

Outcome measures

Outcome measures
Measure	Sebetralstat 300 mg ED n=34 Participants Participants received sebetralstat 300 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 75 mg, those weighing ≥20 to ≤45 kg received 150 mg, and those weighing \>45 kg received 300 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack.	Sebetralstat 600 mg ED n=7 Participants Participants received sebetralstat 600 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 150 mg, those weighing ≥20 to ≤45 kg received 300 mg, and those weighing \>45 kg received 600 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack (with no more than 2 doses permitted within a 24-hour period).
Plasma Concentrations of Sebetralstat 0.5 hours post-dose	1786.00 ng/mL Standard Deviation 1263.690	4042.86 ng/mL Standard Deviation 1843.056
Plasma Concentrations of Sebetralstat 2 hours post-dose	1145.59 ng/mL Standard Deviation 775.594	2761.33 ng/mL Standard Deviation 1090.667
Plasma Concentrations of Sebetralstat 4 hours post-dose	601.05 ng/mL Standard Deviation 1103.278	953.57 ng/mL Standard Deviation 563.929

POST_HOC outcome

Population: The PK Set included all participants who provided at least 1 PK sample.

During the Enrollment Visit, participants were dosed with IMP (sebetralstat 300 mg ED) and monitored for tolerability. PK samples were collected via venous access or capillary collection (eg, finger prick) at specified timepoints. For participants enrolled under protocol v3.4 who received sebetralstat 600 mg ED, a second PK assessment was performed at the Dose Increase Visit. The Dose Increase Visit occurred as soon as possible after implementation of the protocol amendment and could occur at any time during the participant's participation in the trial. A final population-based PK model of sebetralstat was utilized to obtain individual post hoc estimates of PK parameters. For participants with measurable plasma concentrations of sebetralstat, Cmax was predicted using individual post hoc PK parameter estimates.

Outcome measures

Outcome measures
Measure	Sebetralstat 300 mg ED n=34 Participants Participants received sebetralstat 300 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 75 mg, those weighing ≥20 to ≤45 kg received 150 mg, and those weighing \>45 kg received 300 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack.	Sebetralstat 600 mg ED n=7 Participants Participants received sebetralstat 600 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 150 mg, those weighing ≥20 to ≤45 kg received 300 mg, and those weighing \>45 kg received 600 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack (with no more than 2 doses permitted within a 24-hour period).
Maximum Concentration (Cmax) of Sebetralstat	2010 ng/mL Geometric Coefficient of Variation 42.9	4690 ng/mL Geometric Coefficient of Variation 30.5

POST_HOC outcome

Population: The PK Set included all participants who provided at least 1 PK sample.

During the Enrollment Visit, participants were dosed with IMP (sebetralstat 300 mg ED) and monitored for tolerability. PK samples were collected via venous access or capillary collection (eg, finger prick) at specified timepoints. For participants enrolled under protocol v3.4 who received sebetralstat 600 mg ED, a second PK assessment was performed at the Dose Increase Visit. The Dose Increase Visit occurred as soon as possible after implementation of the protocol amendment and could occur at any time during the participant's participation in the trial. A final population-based PK model of sebetralstat was utilized to obtain individual post hoc estimates of PK parameters. For participants with measurable plasma concentrations of sebetralstat, AUC0-inf was predicted using individual post hoc PK parameter estimates.

Outcome measures

Outcome measures
Measure	Sebetralstat 300 mg ED n=34 Participants Participants received sebetralstat 300 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 75 mg, those weighing ≥20 to ≤45 kg received 150 mg, and those weighing \>45 kg received 300 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack.	Sebetralstat 600 mg ED n=7 Participants Participants received sebetralstat 600 mg ED using weight-based dosing as follows: participants weighing ≥9.5 to \<20 kg received 150 mg, those weighing ≥20 to ≤45 kg received 300 mg, and those weighing \>45 kg received 600 mg sebetralstat, administered as an ODT. During the treatment period, each HAE attack was initially treated with a single dose of sebetralstat. If needed (as determined by the participant and/or caregiver), up to 3 total doses of IMP could be administered for each attack (with no more than 2 doses permitted within a 24-hour period).
Area Under the Plasma Concentration-time Curve From Time 0 to Infinity (AUC0-inf) of Sebetralstat	5350 ng*h/mL Geometric Coefficient of Variation 53.1	11660 ng*h/mL Geometric Coefficient of Variation 29.8

Adverse Events

Sebetralstat 300 mg ED

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Sebetralstat 600 mg ED

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Vice President Clinical

KalVista Pharmaceuticals Ltd

Phone: 1 (857) 999-0075

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place