Hereditary Hemorrhagic Telangiectasia (HHT)
Conditions
Brief summary
Frequency of AEs. Safety will also be evaluated through vital signs, ECGs, and clinical laboratory assessments.
Detailed description
Change from baseline in PLG o Plasma activity levels o Plasma protein levels, Change from baseline in epistaxis o Intensity-adjusted epistaxis duration o ESS o Epistaxis duration o Epistaxis frequency o Epistaxis intensity o Epistaxis-free days per month, • Change from baseline in hematologic parameters o HSS o Iron infusions o RBC transfusions o Hemoglobin, • Change from baseline in QoL/PRO scores o NOSE HHT o mPGI-S
Interventions
DRUGphosphate-buffered saline for SC administration
Sponsors
Alnylam Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency of AEs. Safety will also be evaluated through vital signs, ECGs, and clinical laboratory assessments. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in PLG o Plasma activity levels o Plasma protein levels, Change from baseline in epistaxis o Intensity-adjusted epistaxis duration o ESS o Epistaxis duration o Epistaxis frequency o Epistaxis intensity o Epistaxis-free days per month, • Change from baseline in hematologic parameters o HSS o Iron infusions o RBC transfusions o Hemoglobin, • Change from baseline in QoL/PRO scores o NOSE HHT o mPGI-S | — |
Countries
France, Germany, Spain
Outcome results
None listed