Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Pomalidomide Product · 1 trial · 1 indication
The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
| Arm | Type | Description |
|---|---|---|
| Pomalidomide | EXPERIMENTAL | Oral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. |
| Placebo | PLACEBO_COMPARATOR | A placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. |
| Name | Type | Description |
|---|---|---|
| Pomalidomide Oral Product | DRUG | Pomalidomide, a third generation derivative of thalidomide, given orally at a starting dose of 4 mg/day for days 1-28 of six 28-day cycles. The dose may be reduced to 3 or 2 mg/day based on specific adverse event (AE) criteria. |
| Placebo oral capsule | DRUG | Matching placebo will be given. |
Inclusion Criteria: 1. A clinical diagnosis of HHT as defined by the Curacao criteria 2. Age ≥ 18 years 3. Platelet count ≥ 100,000/µl 4. White Blood Count (WBC) ≥ 2,500/µl 5. International Normalized Ratio (INR) ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or partial thromb...