Inhaled Ciclesonide Study in Preterm Infants

NCT06589245 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: STUDY00003195R21HD116421
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

Our overall objective is to conduct a safety study with inhaled ciclesonide to evaluate known glucocorticoids (sGC)-related acute and intermediate toxic effects while measuring for the first time in neonates its systemic absorption and potential bioactivity (i.e. activation of primary target, the GR, in blood cells).

Conditions

Bronchopulmonary Dysplasia

Outcome Measures

Primary Outcomes (8)

• Number of hyperglycemia events (blood glucose >150mg/dL) during treatment.

  Blood Glucose (BG) levels will be collected 24 hours prior to initiation of study drug and then every 12 hours after initiation of study drug for 2 weeks. BG will be monitored daily for a week after cessation of drug treatment and as per NICU protocol thereafter.

  Through study completion, an average of 4 months.

• Average change in z-scores for weight, length, and head circumference.

  Baseline weight, head circumference, and length measurements will be measured as per routine NICU standards, which is daily weights and weekly length and head circumference. Weights, lengths and weights/lengths are routinely plotted in the growth chart for each baby in the NICU, and z-scores trajectories deviations (positive or negative) derived against published reference standards for this population. We will compare the average z-score deviations for growth parameters among infants exposed to the study drug and controls unexposed to study drug.

  Through the time of discharge, an average of 4 months.

• Number of high systolic blood pressure events defined as >95% centile for gestational age and day of life.

  Blood Pressure will be collected 48 hours prior to initiation of study drug as per NICU protocol (at least twice a day) and then every 8 hours after initiation of study drug for 2 weeks. Blood pressure will also be monitored at least daily for first two weeks after cessation of drug treatment, and as per NICU protocol till discharge (at least once a day). Blood pressure is usually monitored using appropriate BP cuff size for weights. If indwelling arterial catheters are present, then blood pressures will be measured that way.

  Through the time of discharge, an average of 4 months.

• Number of infants who do not pass the Adrenocorticotrophic Hormone (ACTH) stimulation test.

  Measured via Adrenocorticotropic Hormone (ACTH) Stimulation Test, which quantifies the increase in cortisol after ACTH administration.

  6 weeks or later after last postnatal steroid course, prior to discharge

• Number of episodes of bronchospasm related to inhaled ciclesonide administration.

  The investigators will quantify any episodes of bronchospasm as evaluated by changes in oxygenation/ventilation status, auscultation for wheezes and changes in ventilator loops. This is evaluated by experienced respiratory therapists and bedside nurses.

  The entire duration of the study drug treatment (14 days).

• Number of infants who develop oral thrush that require treatment.

  Bedside nurses routinely evaluate for this common, mostly benign complication of prematurity. We will collect data on % of infants who develop oral thrush and % of infants that require topical treatment among infants exposed to inhaled ciclesonide and the control group.

  The duration of study drug treatment (14 days).

• Compare rates of BPD and severe BPD between cases and controls.

  Investigators will collect and report data on BPD and severity on each infant in the study irrespective of whether they were treated with inhaled ciclesonide or served as a control. We will also compare rates of BPD and severe BPD among the two groups.

  BPD and severe BPD are ascertained at 36 weeks post-menstrual age.

• Systemic Absorption of inhaled Ciclesonide

  Investigators will measure serum levels of ciclesonide and its active metabolite, des-ciclesonide 3 hour after inhaled drug administration (based on T1/2 in prior studies in children) in peripheral blood by mass spectrometry. This will provide us information on CIC to des-CIC conversion in preterm neonates, as well correlation with doses administered. Measured in picomol/ml (pmol/mL).

  Day 1, 3, 7, 15 of study (during drug administration).

Secondary Outcomes (2)

• Compare rates of extubation success between infants exposed to study drug and controls receiving standard NICU clinical care

  Extubation rates will be calculated from the day of study drug initiation to 7 days after cessation of study drug, i.e., a 21 day window.

• Assess changes in lung injury and need in mechanical ventilation support.

  Baseline, and daily from study drug initiation to 1 week after study drug cessation.

Study Arms (2)

Ciclesonide

EXPERIMENTAL

Eligible infants will be approached by study team. Parents whose infants consent to the study drug will enter the dose-escalation part of the study. Inhaled Alvesco will be administered daily for 14 days at escalating doses 80mcg and 160mcg.

Drug: Alvesco Inhalant Product

Control

NO INTERVENTION

Eligible infants will be approached by study team. Parents who refuse consent to the study drug will have standard of care. These parents can accept for their infant's de-identified clinical data to be used as part of the control group.

Interventions

Alvesco Inhalant Product DRUG

Inhaled Alvesco will be administered daily for 14 days at escalating doses of 80mcg and 160mcg.

Also known as: ciclesonide

Ciclesonide

Eligibility Criteria

• Age: 8 Days - 35 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Viable Infants born between 23 0/7 - 29 6/7 gestation
• Requiring invasive (through an endotracheal tube) mechanical ventilation
• Between day of life 8 to 35.
• Infants have not received dexamethasone for 120 hours
• If receiving hydrocortisone, then receiving ≤ 1mg/kg/day

You may not qualify if:

• Infants with major congenital lung or other organ anomalies, life-threatening illness, active sepsis or NEC, and grade IV hemorrhage will be excluded.
• Infants receiving DEX therapy will be excluded.
• We will exclude infants who have had ≥ 1 glucose level \> 150mg in the 24 hours prior to study entry or those on insulin therapy to treat hyperglycemia. We will exclude infants who have hypertension (\>95% centile for gestational age) in the 48 hours prior to study entry.
• For small for gestational age, we will exclude infants with a birthweight \< 5% centile for gestational age.
• Infants with history of recent pulmonary hemorrhage (within 72 hours of study entry) will also be excluded.
• Infants receiving or have received any dexamethasone in the prior 120 hours.
• Infants receiving \>1mg/kg/day of hydrocortisone.
• Infants receiving any other inhaled or systemic steroid.

Sponsors & Collaborators

• University of Pittsburgh: collaborator
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator
• Venkatesh Sampath: lead

Study Sites (1)

Children's Mercy Kansas City

Kansas City, Missouri, 64108, United States

RECRUITING

MeSH Terms

Conditions

Bronchopulmonary Dysplasia

Interventions

ciclesonide

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Venkatesh Sampath, MD

  Children's Mercy Hospital Kansas City

  PRINCIPAL INVESTIGATOR

• Venkatesh Sampath, MD

  Physician-Scientist

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Venkatesh Sampath, MD

CONTACT

816-234-3596; vsampath@cmh.edu

Miah R Ruffin, BA, CCRC

CONTACT

816-731-7443; mrruffin@cmh.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Physician-Scientist

Model Details: An open label dose escalation

Study Record Dates

• First Submitted: August 20, 2024
• First Posted: September 19, 2024
• Study Start: September 16, 2025
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)