Birth AsphyxiaPhase 1SANE-02NCT04169191
Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia
A Phase 1 study testing Sildenafil Citrate in people with Birth Asphyxia, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
Trial summary
The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia. Read more about this study
- Who is it for?
- Ages 0 to 2 days
- Conditions the study is for
- Birth Asphyxia
- What is being tested?
- Sildenafil Citrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- 19 Sep 2019to 31 Oct 2025
- Phase
- 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 0 to 2 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female neonates meeting the criteria for induced hypothermia:
- Gestational age ≥ 36 weeks and birth weight ≥ 1800 g;
- Evidence of fetal distress, i.e., history of an acute perinatal event, cord pH ≤ 7.0 or base deficit ≤ - 16 mEq/L;
- Evidence of neonatal distress, such as an Apgar score ≤ 5 at 10 minutes, postnatal blood gas pH obtained within the first hour of life ≤ 7.0 or base deficit ≤ - 16 mEq/L, or a continued need for ventilation initiated at birth and continued for at least 10 minutes;
- Evidence of moderate to severe neonatal encephalopathy by an abnormal neurological exam and/or an amplitude-integrated electroencephalogram (aEEG).
- Evidence of brain injury on a brain magnetic resonance imaging (MRI) performed on day 2 of life.
Who cannot join
- Neonates with complex congenital heart disease
- Neonates with cerebral malformations
- Neonates with genetic syndrome
- Neonates with intraventricular and/or intraparenchymal hemorrhage on MRI performed on day 2 of life
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Treatment of Neonatal Encephalopathy With Oral Sildenafil Suspension to Repair Brain Injury Secondary to Birth Asphyxia: An Open-label Dose-finding Clinical Trial (Phase Ib Study)
The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia. They will use a 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h) in asphyxiated neonates demonstrating brain injury despite hypothermia treatment and assess whether we observe any beneficial effects of sildenafil on their brain and cardiopulmonary hemodynamics, without causing serious adverse events
- Conditions
- Birth Asphyxia
- Being tested
- Sildenafil Citrate
- Medical subject headings
- Asphyxia Neonatorum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sildenafil CitrateDrug
Cohort 1 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, and subsequent doses of 2.5mg/kg/dose q12h (= 5 mg/kg/day from dose #2) Cohort 2 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, 3rd dose of 3mg/kg/dose, and subsequent doses of 3mg/kg/dose q12h (= 6 mg/kg/day from dose #3) dose expansion phase in up to 5-15 asphyxiated neonates
| Study group | Role | What they receive |
|---|---|---|
| Sildenafil | Experimental | Not described Receives: Drug: Sildenafil Citrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Death
Number of participants with death
Measured: within first 10 days of life
Hypotension
Number of participants with hypotension
Measured: within first 10 days of life
Secondary outcomes (1)
Peak Plasma Concentration (Cmax)
Peak Plasma Concentration (Cmax)
Measured: within first 10 days of life
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2019
- Study start19 Sep 2019
- Primary completion31 Dec 2021
- Study completion31 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Birth AsphyxiaStudies of the same condition, open and under way.
- More trials run by McGill University Health Centre/Research Institute of the McGill University Health CentreThe 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h)
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pia Wintermark, McGill University Health Centre/Research Institute of the McGill University Health Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04169191
- Sponsor's protocol ID
- SANE-02
- Acronym
- SANE-02
- Status last verified
- Sep 2026
- First submitted
- 1 Oct 2019
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to make individual participant data (IPD) available to other researchers
Publications
Wintermark P, Lapointe A, Altit G, Steinhorn R, Rampakakis E, Meid AD, Burhenne J, Bajraktari-Sylejmani G, Khairy M, Adamo MT, Gilbert G, Toffoli D, Zavalkoff S, Luu TM, Hailu E, Haefeli WE. Testing Higher Doses of Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia: An Open-Label Dose-Finding Phase 1b Clinical Trial-Sildenafil Administration to Treat Neonatal Encephalopathy-Study 02. J Pediatr. 2025 Oct;285:114701. doi: 10.1016/j.jpeds.2025.114701. Epub 2025 Jun 23.
PubMed 40562301
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Neonatal Encephalopathy With Oral Sildenafil Suspension to Repair Brain Injury Secondary to Birth Asphyxia: An Open-label Dose-finding Clinical Trial (Phase Ib Study)
- ClinicalTrials.gov ID
- NCT04169191
- Acronym / sponsor ID
- SANE-02 / SANE-02
- Conditions
- Birth Asphyxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health CentreUniversity, hospital or other
- Study start
- 19 Sep 2019 (actual)
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 19 Nov 2019 (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.
The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia. They will use a 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h) in asphyxiated neonates demonstrating brain injury despite hypothermia treatment and assess whether we observe any beneficial effects of sildenafil on their brain and cardiopulmonary hemodynamics, without causing serious adverse events
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h)
| Arm | Type | Receives |
|---|---|---|
| Sildenafil | Experimental | Drug: Sildenafil Citrate |
Who may qualify
- Ages
- 0 to 2 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male and female neonates meeting the criteria for induced hypothermia:
- Evidence of brain injury on a brain magnetic resonance imaging (MRI) performed on day 2 of life.
Key exclusion criteria (4 listed)
- Neonates with complex congenital heart disease
- Neonates with cerebral malformations
- Neonates with genetic syndrome
- Neonates with intraventricular and/or intraparenchymal hemorrhage on MRI performed on day 2 of life
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 |
|---|---|---|
| Sildenafil CitrateCohort 1 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, and subsequent doses of 2.5mg/kg/dose q12h (= 5 mg/kg/day from dose #2) Cohort 2 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, 3rd dose of 3mg/kg/dose, and subsequent doses of 3mg/kg/dose q12h (= 6 mg/kg/day from dose #3) dose expansion phase in up to 5-15 asphyxiated neonates | Drug | Sildenafil |
Primary outcome
Death
Time frame: within first 10 days of life
Number of participants with death
Hypotension
Time frame: within first 10 days of life
Number of participants with hypotension
1 secondary outcome
Peak Plasma Concentration (Cmax)
Time frame: within first 10 days of life
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 15 Sep 2026.
- Derived from this study: PubMed 40562301Wintermark P, Lapointe A, Altit G, Steinhorn R, Rampakakis E, Meid AD, Burhenne J, Bajraktari-Sylejmani G, Khairy M, Adamo MT, Gilbert G, Toffoli D, Zavalkoff S, Luu TM, Hailu E, Haefeli WE. Testing Higher Doses of Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia: An Open-Label Dose-Finding Phase 1b Clinical Trial-Sildenafil Administration to Treat Neonatal Encephalopathy-Study 02. J Pediatr. 2025 Oct;285:114701. doi: 10.1016/j.jpeds.2025.114701. Epub 2025 Jun 23.
- Individual participant dataSharing planned: No. No plan to make individual participant data (IPD) available to other researchers
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sildenafil Citrate in people with Birth Asphyxia, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
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 6 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 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Death (time frame: within first 10 days of life). The study lists 2 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 NCT04169191, which also lists 1 linked publication.
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 15 Sep 2026.