Milrinone in Congenital Diaphragmatic Hernia

Study ID: STU 042017-055

Summary

CDH is an orphan disease with high mortality and few randomized, masked clinical trials evaluating postnatal management. Since Milrinone is a commonly used medication in neonatal and pediatric intensive care units and is currently used in 17% of patients with CDH within the neonatal Research network (nRn), this will provide data and enable further studies evaluating the efficacy of pulmonary vasodilator therapy in CDH. This is a double-blind randomized controlled pilot study with a 1:1 randomization (treatment: control). Primary outcome: The primary outcome is the oxygenation response, as determined by change in oi (or oSi if no arterial blood gas is available) at 24h after initiation of study drug. Feasibility to perform a definitive trial (primary outcome x improvement in survival without eCMo) and safety (incidence of systemic hypotension, intracranial bleeding and arrhythmias)

Participant Eligibility

Eligibility criteria: Infants are eligible if they meet all of the following criteria: (i) >= 36 0/7 weeks PMA by best obstetric estimate AND birth weight of >= 2000g (ii) postnatal age <=7 days (168 hours of age, including post-operative infants following CDH repair with HRF within this postnatal age) (iii) invasive mechanical ventilation (defined as ventilation with an endotracheal tube) and (iv) one arterial blood gas with an OI >= 10 (after tracheal tube obstruction and other easily resolvable mechanical causes for increased OI are ruled out) on the most recent arterial blood gas within 12 hours prior to the time of randomization. (v) if an arterial blood gas is not available at the time of randomization, a preductal OSI of >= 5 can be used as an inclusion criterion instead of OI >= 10; (the OSI should be based on the most recent preductal pulse oximetry recording and must be within 12 hours of randomization) (vi) postnatal blood gas with PCO2 <= 80 mmHg (arterial, capillary or venous blood gas) on the most recent blood gas sample obtained within 12 hours prior to randomization Note: Criteria (iv) to (vi) must be met at the most recent analysis within 12 hours prior to randomization

Cancer Related: No
Healthy Volunteers: No
UT Southwestern Principal Investigator: Vedanta Singh Dariya

Contact:

Diana Vasil

DIANA.VASIL@UTSouthwestern.edu