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

Open-label Study to Assess the Safety, Tolerability, Feasibility, and Pharmacokinetics of Inhaled RVT-1601 in Preterm Infants at High-risk for Long-term Respiratory Morbidities

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.

Study Overview

• Status: Completed
• Conditions: Respiratory Morbidities of Prematurity (RMP)
• Intervention / Treatment: Drug: RVT-1601

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • San Diego, California, United States, 92123
      • Sharp Mary Birch Hospital for Women & Newborns

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 2 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RVT-1601 Low Dose; Inhaled RVT-1601 administered once daily over two days via eFlow nebulizer	Drug: RVT-1601; Inhaled RVT-1601 administered once daily over two days
Experimental: RVT-1601 Mid Dose; Inhaled RVT-1601 administered once daily over two days via eFlow nebulizer	Drug: RVT-1601; Inhaled RVT-1601 administered once daily over two days

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Respivant Sciences GmbH
• Collaborators: Respivant Sciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2019
• Primary Completion (Actual): May 29, 2020
• Study Completion (Actual): May 29, 2020

Study Registration Dates

• First Submitted: June 28, 2019
• First Submitted That Met QC Criteria: July 2, 2019
• First Posted (Actual): July 5, 2019

Study Record Updates

• Last Update Posted (Actual): June 4, 2020
• Last Update Submitted That Met QC Criteria: June 2, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Premature Birth
• Other Study ID Numbers: RVT1601-RMP-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No