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

Launched by KALVISTA PHARMACEUTICALS, LTD. · Jun 19, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called Sebetralstat (KVD900) for children aged 2 to 11 who have a condition called Hereditary Angioedema (HAE) Type I or II. HAE is a genetic disorder that causes sudden swelling in various parts of the body, which can be painful and scary. The goal of this trial is to see how safe and effective Sebetralstat is for these young patients compared to existing treatments. The trial is currently looking for participants, and to be eligible, children must have had at least one HAE attack in the past year and have a confirmed diagnosis of HAE Type I or II.

If your child is chosen to participate, they will receive Sebetralstat and will be closely monitored by medical professionals throughout the study. Caregivers will need to help with storing and giving the medication and completing a daily diary to track any changes or reactions. It’s important to know that certain other medical conditions or medications may make your child ineligible for the trial. Overall, this study aims to help improve treatment options for children with HAE, and participating families will contribute to valuable research that could benefit many others in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female patients 2 to 11 years of age.
• • 2. Confirmed diagnosis of HAE Type I or II.
• • 3. For patients ≥20 kg at screening, patient has had at least 1 documented HAE attack in the last year prior to screening.
• • 4. Caregiver, as assessed by the Investigator, must be able to appropriately store and administer IMP and be able to read, understand, and complete the diary.
• • 5. Investigator believes that the patient and caregiver are willing and able to adhere to all protocol requirements.
• • 6. Parent or Legally Authorized Representative (LAR) provides signed informed consent and patient provides assent (when applicable).
• Exclusion Criteria:
• • 1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH, idiopathic angioedema, or angioedema associated with urticaria.
• • 2. A clinically significant history of poor response to bradykinin receptor 2 blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
• • 3. Patient weighs \<9.5 kg.
• • 4. Use of angiotensin-converting enzyme inhibitors after the Screening Visit.
• • 5. Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
• • 6. Patients who require sustained use of strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers or moderate CYP3A4 inducers.
• • 7. Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial.
• • 8. Known hypersensitivity to sebetralstat or to any of the excipients.
• • 9. Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.