Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
N-acetylcysteine, NAC, and calcitriol · 1 trial · 1 indication
PK parameters of plasma half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
PK parameters of serum half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
GSH by MRS before, immediately after and up to 6h after NVD infusion on day of life 5
| Arm | Type | Description |
|---|---|---|
| NAC 25mg/kg, calcitriol 0.05mcg/kg | EXPERIMENTAL | N-acetylcysteine 25mg/kg iv q 12h, calcitriol 0.05mcg/kg iv q 12h, for 10 days, starting within 6h of birth |
| NAC 25mg/kg, calcitriol 0.03mcg/kg | EXPERIMENTAL | N-acetylcysteine 25mg/kg iv q 12h, calcitriol 0.03mcg/kg iv q 24h, for 10 days, starting within 6h of birth |
| NAC 40mg/kg, calcitriol 0.03mcg/kg | EXPERIMENTAL | N-acetylcysteine 40mg/kg iv q 12h, calcitriol 0.03mcg/kg iv q 24h, for 10 days, starting within 6h of birth |
| Name | Type | Description |
|---|---|---|
| N-acetylcysteine, NAC, and calcitriol | DRUG | iv administration of antioxidant and active vitamin D |
Inclusion Criteria: * Neonates \> 34 weeks, \> 2000 grams, within 6h of birth with moderate to severe HIE receiving therapeutic hypothermia Exclusion Criteria: * Evidence of a congenital CNS malformation if known prior to enrollment * Evidence of neuromuscular disorder by family history * More th...
N-acetylcysteine and calcitriol is an investigational combination being studied for perinatal hypoxic-ischemic encephalopathy (HIE) in neonates. It is a small molecule therapy developed by United Therapeutics Corporation. The drug is in early clinical development, with one completed Phase 1 trial evaluating its use in hypothermic HIE neonates.
The specific molecular target of the N-acetylcysteine and calcitriol combination is not disclosed. The drug is being investigated for its potential effects in perinatal hypoxic-ischemic encephalopathy, a condition caused by reduced blood flow and oxygen to the brain around the time of birth.
United Therapeutics Corporation (NASDAQ: UTHR) is developing the combination of N-acetylcysteine and calcitriol. The company is conducting clinical research on this therapy for perinatal hypoxic-ischemic encephalopathy in neonates.
N-acetylcysteine and calcitriol is in Phase 1 clinical development. A single early Phase 1 trial has been completed, with 30 participants enrolled. The drug remains investigational and has not been approved by regulatory authorities.
N-acetylcysteine and calcitriol has one completed clinical trial, NCT04643821, titled "NVD in Hypothermic HIE Neonates." This early Phase 1 study enrolled 30 participants with perinatal hypoxic-ischemic encephalopathy. The trial was controlled but not randomized or double-blinded.
N-acetylcysteine and calcitriol is a combination therapy that includes N-acetylcysteine, also known as NAC, along with calcitriol. The drug name refers to the combination of these two components, not NAC alone. United Therapeutics is developing this combination for perinatal hypoxic-ischemic encephalopathy.