Phase 3
N=212
Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
Hereditary Angioedema (HAE)
Bottom Line
View on ClinicalTrials.gov: NCT02741596 ↗Enrolled (actual)
212
Serious AEs
9.9%
Results posted
May 2020
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 105; 101 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- DX-2930 (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Oct 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
105; 101 | — |
| SECONDARY Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During the Treatment Period |
0.27; 0.22 | — |
| SECONDARY Rate of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During the Treatment Period |
0.20; 0.21 | — |
| SECONDARY Rate of Moderate or Severe Hereditary Angioedema (HAE) Attacks During the Treatment Period |
0.21; 0.19 | — |
| SECONDARY Rate of High-Morbidity Hereditary Angioedema (HAE) Attacks During the Treatment Period |
0.03; 0.04 | — |
| SECONDARY Time to First Investigator-Confirmed Hereditary Angioedema (HAE) Attacks in Rollover Participants |
43 | — |
Summary
This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in participants with Type I and Type II HAE.
Eligibility Criteria
Inclusion Criteria
- Male and female HAE participants who are 12 years of age or older at the time of screening
- Documented diagnosis of HAE (Type I or II) based on
- Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria).
- Diagnostic testing results obtained during screening (or a prior DX-2930 study) that confirm HAE Type I or II: C1 inhibitor (C1-INH) functional level less than ( 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's Syndrome).
- Pregnancy or breastfeeding.
- Participant has any condition that, in the opinion of the investigator or Sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (eg, history of substance abuse or dependence, a significant pre-existing illness or other major comorbidity that the investigator considers may confound the interpretation of study results).
Data sourced from ClinicalTrials.gov (NCT02741596). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.