Open-label Study of Inhaled RVT-1601 in Preterm Infants

Phase 1

Completed

• Conditions: Respiratory Morbidities of Prematurity (RMP)
• Interventions: Drug: RVT-1601

• Registration Number: NCT04007120
• Lead Sponsor: Respivant Sciences GmbH

Brief Summary

Preterm birth predisposes infants to greater risk for respiratory morbidities and the need for pulmonary care compared to term infants both in the short-term and long-term. In the short-term, preterm birth is a high risk factor for development of bronchopulmonary dysplasia (BPD), the second most common chronic pediatric respiratory disease after asthma. In the long-term, following discharge from the neonatal intensive care unit (NICU) and the hospital, preterm birth carries a high risk for respiratory morbidities (e.g., wheezing, cough, doctor visits, and hospitalizations for respiratory infections) and resource use, which in turn predisposes infants to the development of lung diseases in childhood and adulthood, including airway hyperresponsiveness, asthma, and chronic obstructive pulmonary disease (COPD). There is a significant unmet need for safe and efficacious approaches in the prevention and treatment of respiratory morbidities of prematurity.

The study will be conducted in the neonatal intensive care unit (NICU) in preterm infants to determine safety, tolerability and lung delivery performance of RVT-1601, a new inhalation formulation of cromolyn sodium delivered via the eFlow® Closed System (CS) nebulizer/face mask.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-06-28
• Study First Submitted QC: 2019-07-02
• Study First Posted: 2019-07-05
• Study Start: 2019-10-01
• Primary Completion: 2020-05-29
• Study Completion: 2020-05-29
• Status Verified: 2020-06
• Last Update Submitted: 2020-06-02
• Last Update Posted: 2020-06-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

• Preterm infants between 32 weeks 0 days and 34 weeks 6 days of PMA
• Born between 24 weeks 0 days and 29 weeks 6 days of estimated GA
• Requiring minimal or no respiratory support (i.e., supplemental oxygen with <2 liters per minute of nasal cannula flow acceptable)
• Body weight appropriate for gestational age
• Written informed consent obtained from at least one of the parents or legal guardians

Exclusion Criteria

• Requiring invasive or noninvasive respiratory support (e.g., mechanical ventilation, CPAP)
• Clinically unstable (i.e. unable to maintain SpO2 between 90-95 % , escalating respiratory support in the past 24 hours)
• Major congenital anomaly (chromosomal, renal, cardiac, hepatic, neurologic or pulmonary malformations)
• Significant cardiac disorder (i.e., pulmonary hypertension)
• History of major surgical procedure
• Any condition that would preclude receiving study drug or performing any study-related procedures
• Participation in any other investigational drug study
• History of hypersensitivity or intolerance to cromolyn sodium

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
RVT-1601 Low Dose	RVT-1601	Inhaled RVT-1601 administered once daily over two days via eFlow nebulizer
RVT-1601 Mid Dose	RVT-1601	Inhaled RVT-1601 administered once daily over two days via eFlow nebulizer

Primary Outcome Measures

Name	Time	Method
Change in oxygenation	Pre-dose and 15 minutes post-dose	Assessment of peripheral capillary oxygen saturation (SpO2)
Change in heart rate	Pre-dose and 15 minutes post-dose	Assessment of heart rate (beats/min)
Change in blood pressure	Pre-dose and 15 minutes post-dose	Assessment of systolic and diastolic blood pressure (mmHg)

Secondary Outcome Measures

Name	Time	Method
Total urine excretion	8 hours post-dose	Assessment of total urine content of RVT-1601
Peak plasma concentration (Cmax)	30 minutes post-dose	Assessment of peak plasma concentration of RVT-1601

Trial Locations

Locations (1)

Sharp Mary Birch Hospital for Women & Newborns; 🇺🇸 San Diego, California, United States