HIE - Perinatal Hypoxic - Ischemic EncephalopathyEarly Phase 1NCT04643821
NVD in Hypothermic HIE Neonates
A Early Phase 1 study testing N-acetylcysteine, NAC, and calcitriol in people with HIE - Perinatal Hypoxic - Ischemic Encephalopathy, run by Medical University of South Carolina.
Trial summary
Neonatal hypoxic ischemic (HI) injury is an unpredictable neurologic injury with devastating, long term consequences for parents who are expecting a normal child. Hypothermia for 72 hr within 6 hrs of birth improves the combined outcome of death or severe disability, and hypothermia is now standard of care in tertiary… Read more about this study
- Who is it for?
- Ages Up to 6 hours
- Conditions the study is for
- HIE - Perinatal Hypoxic - Ischemic Encephalopathy
- What is being tested?
- N-acetylcysteine, NAC, and calcitriol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2015to 1 Mar 2020
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 6 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Neonates > 34 weeks, > 2000 grams, within 6h of birth with moderate to severe HIE receiving therapeutic hypothermia
Who cannot join
- Evidence of a congenital CNS malformation if known prior to enrollment
- Evidence of neuromuscular disorder by family history
- More than 6 hours from birth or known insult
- Suspected genetic abnormality
About this study
Official title: N-Acetylcysteine and Vitamin D in Infants With Hypoxic Ischemic Encephalopathy Treated With Hypothermia
Neonatal hypoxic ischemic (HI) injury is an unpredictable neurologic injury with devastating, long term consequences for parents who are expecting a normal child. Hypothermia for 72 hr within 6 hrs of birth improves the combined outcome of death or severe disability, and hypothermia is now standard of care in tertiary centers throughout the world. However, approximately 50% of infants with hypoxic ischemic encephalopathy (HIE) treated with hypothermia still have adverse neurologic outcomes, due to ongoing neuroinflammation and oxidative stress in spite of hypothermia. Further, the majority of HIE infants are insufficient or deficient in a critical neurosteroid, 25(OH)vitamin D, which has been shown to adversely affect outcome after adult stroke. By adding vitamin D to N-acetylcysteine (NAC), an antioxidant, the investigators hypothesized that both drugs would increase glutathione (GSH) concentrations in critical brain areas, mitigate continuing oxidative stress after injury during hypothermia and after rewarming, and improve neurodevelopmental outcomes.
This is an open-label, non-randomized, escalating dose, pilot trial to evaluate the disposition and safety of NAC in combination with active vitamin D in neonates who present within 6 hrs of hypoxia ischemia/asphyxial event and received moderate hypothermia to 33 degrees C for 72 hours per routine protocol.
Read the detailed description
N-acetylcysteine (NAC) is an FDA-approved drug that has been used in multiple conditions to mitigate oxidative stress. The study investigators' lab and others have shown that NAC provides neuroprotection either alone or in combination with hypothermia when given within 1-6 hrs of insult in animal models of HI injury. However, in neonatal rats subjected to severe hypoxic ischemic insult, NAC + hypothermia did not neuroprotect males as well as females. The study investigators and others determined that the majority of HIE infants are insufficient or deficient in 25(OH)vitamin D, a critical neurosteroid that also augments synthesis of an important antioxidant, glutathione. By adding active, low-dose 1,25-dihydoxy-Vitamin D3 to NAC (NVD), with a 1 hour delay after starting hypothermia, and repeated daily for 14 days in neonatal rat HI model, the study investigators significantly improved severity of brain injury over hypothermia alone in both sexes. Importantly, NVD also significantly improved functional outcomes of strength, sensorimotor and memory functioning 6 weeks after HI, even in male rats with the most severe brain pathology.
NAC and active vitamin D are FDA approved and are safe even in very sick newborns. In the study investigators' trial of NAC in maternal chorioamnionitis, comprehensive physiologic monitoring in preterm and term infants exposed to intrauterine inflammation demonstrated no significant differences in cerebral blood flow, oxygenation, or left ventricular function in infants treated with NAC or saline.
The primary objective of this study in human neonates after HIE birth treated with the standard hypothermia protocol, is to determine the unique pharmacokinetic (PK) parameters of NAC and vitamin D during hypothermia and after rewarming, verify the central nervous system (CNS) effect of NVD on the pharmacodynamic target, reduced glutathione, and determine the duration of CNS effect. The study investigators used low dose NAC (Acetadote, 25-40 mg/kg/dose) every 12 hours and Vitamin D3 (Calcitriol, 0.03 to 0.1microgram/kg) every 12-24 hours, infused IV for 10 days in a dose escalating study. The study investigators determined PK parameters and plasma oxidative stress markers during day 1 of life while hypothermic, and day 5 of life during normothermia (24-36 hours after rewarming). To establish effective dosing of NVD based directly on CNS effect, CNS metabolites were quantified with magnetic resonance spectroscopy (MRS) before and immediately after NVD dosing on DOL 5, infusing NVD during the routine MRI for HIE. In a subset of 10 infants the delayed effects of NVD on CNS metabolomics were determined by MRS between 2-6h after NVD dosing on DOL 5. Development was followed for >24months.
- Being tested
- N-acetylcysteine, NAC, and calcitriol
- Keywords
- oxidative stress, glutathione, pharmacokinetics
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylcysteine, NAC, and calcitriolDrug
Also called hypothermia
iv administration of antioxidant and active vitamin D
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: N-acetylcysteine, NAC, and calcitriol |
| 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 Receives: Drug: N-acetylcysteine, NAC, and calcitriol |
| 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 Receives: Drug: N-acetylcysteine, NAC, and calcitriol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in pharmacokinetic half life of NAC
PK parameters of plasma half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
Measured: first week of life
pharmacokinetic half life of calcitriol
PK parameters of serum half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
Measured: first week of life
Change in Glutathione concentration in Basal ganglia
GSH by MRS before, immediately after and up to 6h after NVD infusion on day of life 5
Measured: day of life 5
Secondary outcomes (1)
Change in plasma oxidative stress markers
isofurans measured by Liquid Chromatography-Mass Spectroscopy
Measured: day 1 and 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2020
- Study start1 Jan 2015
- Primary completion1 Apr 2017
- Study completion1 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for HIE - Perinatal Hypoxic - Ischemic EncephalopathyStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Open-label, escalating dose design
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute for Health Research, United Kingdom, National Institute of Neurological Disorders and Stroke (NINDS), Carlos III Health Institute
- Responsible party
- Principal investigator, Dorothea D. Jenkins, Medical University of South Carolina
- Investigators
- Dorothea Jenkins, MD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04643821
- Sponsor's protocol ID
- 31254
- Other IDs
- 5F31NS108623-02, FISPI14/0433, 300118, P960743, EC11-246
- Status last verified
- Dec 2020
- First submitted
- 11 Nov 2020
- Last updated
- 4 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: after publication, upon request
Publications
Jenkins DD, Wiest DB, Mulvihill DM, Hlavacek AM, Majstoravich SJ, Brown TR, Taylor JJ, Buckley JR, Turner RP, Rollins LG, Bentzley JP, Hope KE, Barbour AB, Lowe DW, Martin RH, Chang EY. Fetal and Neonatal Effects of N-Acetylcysteine When Used for Neuroprotection in Maternal Chorioamnionitis. J Pediatr. 2016 Jan;168:67-76.e6. doi: 10.1016/j.jpeds.2015.09.076. Epub 2015 Nov 3.
PubMed 26545726Nie X, Lowe DW, Rollins LG, Bentzley J, Fraser JL, Martin R, Singh I, Jenkins D. Sex-specific effects of N-acetylcysteine in neonatal rats treated with hypothermia after severe hypoxia-ischemia. Neurosci Res. 2016 Jul;108:24-33. doi: 10.1016/j.neures.2016.01.008. Epub 2016 Feb 3.
PubMed 26851769Lowe DW, Fraser JL, Rollins LG, Bentzley J, Nie X, Martin R, Singh I, Jenkins D. Vitamin D improves functional outcomes in neonatal hypoxic ischemic male rats treated with N-acetylcysteine and hypothermia. Neuropharmacology. 2017 Sep 1;123:186-200. doi: 10.1016/j.neuropharm.2017.06.004. Epub 2017 Jun 6.
PubMed 28599922Lowe DW, Hollis BW, Wagner CL, Bass T, Kaufman DA, Horgan MJ, Givelichian LM, Sankaran K, Yager JY, Katikaneni LD, Wiest D, Jenkins D. Vitamin D insufficiency in neonatal hypoxic-ischemic encephalopathy. Pediatr Res. 2017 Jul;82(1):55-62. doi: 10.1038/pr.2017.13. Epub 2017 Jan 17.
PubMed 28099429Wiest DB, Chang E, Fanning D, Garner S, Cox T, Jenkins DD. Antenatal pharmacokinetics and placental transfer of N-acetylcysteine in chorioamnionitis for fetal neuroprotection. J Pediatr. 2014 Oct;165(4):672-7.e2. doi: 10.1016/j.jpeds.2014.06.044. Epub 2014 Jul 23.
PubMed 25064164Moss HG, Brown TR, Wiest DB, Jenkins DD. N-Acetylcysteine rapidly replenishes central nervous system glutathione measured via magnetic resonance spectroscopy in human neonates with hypoxic-ischemic encephalopathy. J Cereb Blood Flow Metab. 2018 Jun;38(6):950-958. doi: 10.1177/0271678X18765828. Epub 2018 Mar 21.
PubMed 29561203Sanchez-Illana A, Thayyil S, Montaldo P, Jenkins D, Quintas G, Oger C, Galano JM, Vigor C, Durand T, Vento M, Kuligowski J. Novel free-radical mediated lipid peroxidation biomarkers in newborn plasma. Anal Chim Acta. 2017 Dec 15;996:88-97. doi: 10.1016/j.aca.2017.09.026. Epub 2017 Sep 28.
PubMed 29137711
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- N-Acetylcysteine and Vitamin D in Infants With Hypoxic Ischemic Encephalopathy Treated With Hypothermia
- ClinicalTrials.gov ID
- NCT04643821
- Acronym / sponsor ID
- 31254
- Keywords
- oxidative stress, glutathione, pharmacokinetics
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- National Institute for Health Research, United Kingdom, National Institute of Neurological Disorders and Stroke (NINDS), Carlos III Health Institute
- Study start
- 1 Jan 2015 (actual)
- Primary completion
- 1 Apr 2017 (actual)
- Study completion
- 1 Mar 2020 (actual)
- First posted
- 25 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neonatal hypoxic ischemic (HI) injury is an unpredictable neurologic injury with devastating, long term consequences for parents who are expecting a normal child. Hypothermia for 72 hr within 6 hrs of birth improves the combined outcome of death or severe disability, and hypothermia is now standard of care in tertiary centers throughout the world. However, approximately 50% of infants with hypoxic ischemic encephalopathy (HIE) treated with hypothermia still have adverse neurologic outcomes, due to ongoing neuroinflammation and oxidative stress in spite of hypothermia. Further, the majority of HIE infants are insufficient or deficient in a critical neurosteroid, 25(OH)vitamin D, which has been shown to adversely affect outcome after adult stroke. By adding vitamin D to N-acetylcysteine (NAC), an antioxidant, the investigators hypothesized that both drugs would increase glutathione (GSH) concentrations in critical brain areas, mitigate continuing oxidative stress after injury during hypothermia and after rewarming, and improve neurodevelopmental outcomes.
This is an open-label, non-randomized, escalating dose, pilot trial to evaluate the disposition and safety of NAC in combination with active vitamin D in neonates who present within 6 hrs of hypoxia ischemia/asphyxial event and received moderate hypothermia to 33 degrees C for 72 hours per routine protocol.
Detailed description
N-acetylcysteine (NAC) is an FDA-approved drug that has been used in multiple conditions to mitigate oxidative stress. The study investigators' lab and others have shown that NAC provides neuroprotection either alone or in combination with hypothermia when given within 1-6 hrs of insult in animal models of HI injury. However, in neonatal rats subjected to severe hypoxic ischemic insult, NAC + hypothermia did not neuroprotect males as well as females. The study investigators and others determined that the majority of HIE infants are insufficient or deficient in 25(OH)vitamin D, a critical neurosteroid that also augments synthesis of an important antioxidant, glutathione. By adding active, low-dose 1,25-dihydoxy-Vitamin D3 to NAC (NVD), with a 1 hour delay after starting hypothermia, and repeated daily for 14 days in neonatal rat HI model, the study investigators significantly improved severity of brain injury over hypothermia alone in both sexes. Importantly, NVD also significantly improved functional outcomes of strength, sensorimotor and memory functioning 6 weeks after HI, even in male rats with the most severe brain pathology.
NAC and active vitamin D are FDA approved and are safe even in very sick newborns. In the study investigators' trial of NAC in maternal chorioamnionitis, comprehensive physiologic monitoring in preterm and term infants exposed to intrauterine inflammation demonstrated no significant differences in cerebral blood flow, oxygenation, or left ventricular function in infants treated with NAC or saline.
The primary objective of this study in human neonates after HIE birth treated with the standard hypothermia protocol, is to determine the unique pharmacokinetic (PK) parameters of NAC and vitamin D during hypothermia and after rewarming, verify the central nervous system (CNS) effect of NVD on the pharmacodynamic target, reduced glutathione, and determine the duration of CNS effect. The study investigators used low dose NAC (Acetadote, 25-40 mg/kg/dose) every 12 hours and Vitamin D3 (Calcitriol, 0.03 to 0.1microgram/kg) every 12-24 hours, infused IV for 10 days in a dose escalating study. The study investigators determined PK parameters and plasma oxidative stress markers during day 1 of life while hypothermic, and day 5 of life during normothermia (24-36 hours after rewarming). To establish effective dosing of NVD based directly on CNS effect, CNS metabolites were quantified with magnetic resonance spectroscopy (MRS) before and immediately after NVD dosing on DOL 5, infusing NVD during the routine MRI for HIE. In a subset of 10 infants the delayed effects of NVD on CNS metabolomics were determined by MRS between 2-6h after NVD dosing on DOL 5. Development was followed for >24months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- Model description
- Open-label, escalating dose design
| Arm | Type | Receives |
|---|---|---|
| NAC 25mg/kg, calcitriol 0.05mcg/kg | Experimental | Drug: N-acetylcysteine, NAC, and calcitriolN-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 | Drug: N-acetylcysteine, NAC, and calcitriolN-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 | Drug: N-acetylcysteine, NAC, and calcitriolN-acetylcysteine 40mg/kg iv q 12h, calcitriol 0.03mcg/kg iv q 24h, for 10 days, starting within 6h of birth |
Who may qualify
- Ages
- Up to 6 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Neonates > 34 weeks, > 2000 grams, within 6h of birth with moderate to severe HIE receiving therapeutic hypothermia
Key exclusion criteria (4 listed)
- Evidence of a congenital CNS malformation if known prior to enrollment
- Evidence of neuromuscular disorder by family history
- More than 6 hours from birth or known insult
- Suspected genetic abnormality
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetylcysteine, NAC, and calcitriolAlso: hypothermiaiv administration of antioxidant and active vitamin D | Drug | NAC 25mg/kg, calcitriol 0.03mcg/kg, NAC 25mg/kg, calcitriol 0.05mcg/kg, NAC 40mg/kg, calcitriol 0.03mcg/kg |
Primary outcome
Change in pharmacokinetic half life of NAC
Time frame: first week of life
PK parameters of plasma half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
pharmacokinetic half life of calcitriol
Time frame: first week of life
PK parameters of serum half life around first dose on day of life 1 during hypothermia, and day of life 5 during normothermia
Change in Glutathione concentration in Basal ganglia
Time frame: day of life 5
GSH by MRS before, immediately after and up to 6h after NVD infusion on day of life 5
1 secondary outcome
Change in plasma oxidative stress markers
Time frame: day 1 and 5
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2020.
- Background: PubMed 26545726Jenkins DD, Wiest DB, Mulvihill DM, Hlavacek AM, Majstoravich SJ, Brown TR, Taylor JJ, Buckley JR, Turner RP, Rollins LG, Bentzley JP, Hope KE, Barbour AB, Lowe DW, Martin RH, Chang EY. Fetal and Neonatal Effects of N-Acetylcysteine When Used for Neuroprotection in Maternal Chorioamnionitis. J Pediatr. 2016 Jan;168:67-76.e6. doi: 10.1016/j.jpeds.2015.09.076. Epub 2015 Nov 3.
- Background: PubMed 26851769Nie X, Lowe DW, Rollins LG, Bentzley J, Fraser JL, Martin R, Singh I, Jenkins D. Sex-specific effects of N-acetylcysteine in neonatal rats treated with hypothermia after severe hypoxia-ischemia. Neurosci Res. 2016 Jul;108:24-33. doi: 10.1016/j.neures.2016.01.008. Epub 2016 Feb 3.
- Background: PubMed 28599922Lowe DW, Fraser JL, Rollins LG, Bentzley J, Nie X, Martin R, Singh I, Jenkins D. Vitamin D improves functional outcomes in neonatal hypoxic ischemic male rats treated with N-acetylcysteine and hypothermia. Neuropharmacology. 2017 Sep 1;123:186-200. doi: 10.1016/j.neuropharm.2017.06.004. Epub 2017 Jun 6.
- Background: PubMed 28099429Lowe DW, Hollis BW, Wagner CL, Bass T, Kaufman DA, Horgan MJ, Givelichian LM, Sankaran K, Yager JY, Katikaneni LD, Wiest D, Jenkins D. Vitamin D insufficiency in neonatal hypoxic-ischemic encephalopathy. Pediatr Res. 2017 Jul;82(1):55-62. doi: 10.1038/pr.2017.13. Epub 2017 Jan 17.
- Background: PubMed 25064164Wiest DB, Chang E, Fanning D, Garner S, Cox T, Jenkins DD. Antenatal pharmacokinetics and placental transfer of N-acetylcysteine in chorioamnionitis for fetal neuroprotection. J Pediatr. 2014 Oct;165(4):672-7.e2. doi: 10.1016/j.jpeds.2014.06.044. Epub 2014 Jul 23.
- Results publication: PubMed 29561203Moss HG, Brown TR, Wiest DB, Jenkins DD. N-Acetylcysteine rapidly replenishes central nervous system glutathione measured via magnetic resonance spectroscopy in human neonates with hypoxic-ischemic encephalopathy. J Cereb Blood Flow Metab. 2018 Jun;38(6):950-958. doi: 10.1177/0271678X18765828. Epub 2018 Mar 21.
- Results publication: PubMed 29137711Sanchez-Illana A, Thayyil S, Montaldo P, Jenkins D, Quintas G, Oger C, Galano JM, Vigor C, Durand T, Vento M, Kuligowski J. Novel free-radical mediated lipid peroxidation biomarkers in newborn plasma. Anal Chim Acta. 2017 Dec 15;996:88-97. doi: 10.1016/j.aca.2017.09.026. Epub 2017 Sep 28.
- Individual participant dataSharing planned: Yes. after publication, upon request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dorothea Jenkins, MD principal investigator
Medical University of South Carolina
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing N-acetylcysteine, NAC, and calcitriol in people with HIE - Perinatal Hypoxic - Ischemic Encephalopathy, run by Medical University of South Carolina.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in pharmacokinetic half life of NAC (time frame: first week of life). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04643821, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2020.