Cystic Fibrosis Lung Disease Clinical Trial
— PRESISOfficial title:
Randomized, Double-blind, Controlled Pilot Study on Safety of Hypertonic Saline as Preventive Inhalation Therapy in Newborns and Infants With Cystic Fibrosis
Verified date | October 2017 |
Source | Heidelberg University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to assess whether 6% hypertonic saline (HS) is a safe and effective preventive therapy in newborns and infants with cystic fibrosis (CF).
Status | Completed |
Enrollment | 42 |
Est. completion date | October 2017 |
Est. primary completion date | November 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 4 Months |
Eligibility |
Inclusion Criteria: 1. Confirmed diagnosis of CF established in neonatal period either via CF newborn screening (NBS) or because of symptoms typical for CF (e.g. meconium ileus), positive family history or positive prenatal screening and fulfilling at least one of the following three criteria: - sweat chloride = 60mEq/L - two CF causing mutations of CFTR gen - alterations of transepithelial potential difference of nasal or rectal epithelia typical for CF. 2. Age at enrolment is 0 to 4 months. 3. Patient's and parent's ability to comply with medication use, study visits, and study procedures is judged by the investigator (therefore parents have to understand the character of the study and individual consequences). 4. Participation in this study is voluntary. Only patients, whose parents or legal guardians gave written consent, are included. Exclusion Criteria: 1. Born < 30 weeks gestation. 2. Prolonged mechanical ventilation in the first 3 months of life. 3. A significant medical disease or condition other than CF likely to interfere with the child's ability to complete the entire protocol. 4. Previous major surgery except for meconium ileus. 5. Other major organ dysfunction, excluding pancreatic or hepatic dysfunction or another condition due to cystic fibrosis. 6. Physical findings that would compromise the safety of the subject or the quality of the study data as determined by investigator. 7. History of adverse reaction to sedation. 8. Known hypersensitivity to study treatment. 9. Participation in other interventional studies at the same time. Criteria, which lead to a displacement of the procedures in sedation until the child has recovered: - Clinically significant upper airway obstruction as determined by investigator (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnoea). - Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset in 2 weeks preceding visit. - Oxygen saturation <95% before initial pulmonary function test or initial MRI. - Severe gastroesophageal reflux, defined as persistent frequent emesis despite anti-reflux therapy. |
Country | Name | City | State |
---|---|---|---|
Germany | University Hospital Gießen and Marburg GmbH | Gießen | |
Germany | Medizinische Hochschule Hannover | Hannover | |
Germany | University Children's Hospital Heidelberg, Cystic Fibrosis Centre | Heidelberg | Baden-Württemberg |
Germany | University Children's Hospital Schleswig-Holstein | Lübeck |
Lead Sponsor | Collaborator |
---|---|
Heidelberg University | German Center for Lung Research |
Germany,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of patients in both treatment groups with adverse events (AEs) and serious adverse events (SAEs) | Safety of inhalation with HS and IS in newborns and infants with CF assessed by proportion of adverse events (AEs) and serious adverse events (SAEs) | during the 52 week treatment period | |
Secondary | Rate of protocol-defined pulmonary exacerbations | Rate of protocol-defined pulmonary exacerbations requiring treatment with oral, inhaled or intravenous antibiotics between subjects randomized to HS and IS | during the 52 week treatment period | |
Secondary | Time to first pulmonary exacerbation in both treatment groups | during the 52 week treatment period | ||
Secondary | Proportion of children with morphological and/or functional changes due to CF lung disease at baseline and after 1 year of inhalation | Proportion of children with morphological and/or functional changes due to CF lung disease at baseline and after 1 year of inhalation according to magnetic resonance imaging (MRI) chest score and chest x-ray (CXR) Chrispin-Norman score in both groups (HS vs. IS) | during the 52 week treatment period | |
Secondary | Extent and severity of bronchial dilatation | Extent and severity of bronchial dilatation after MRI and CXR scores at baseline and after 1 year of inhalation in both groups | during the 52 week treatment period | |
Secondary | Proportion of children with impairments in lung function | Proportion of children with impairments in lung function determined via multiple breath washout at baseline, after 3, 6, 9, and 12 months of inhalation in both groups | during the 52 week treatment period | |
Secondary | Severity of impairment in lung function test | Severity of impairment in lung function test at baseline, after 3, 6, 9, and 12 months of inhalation in both groups | during the 52 week treatment period | |
Secondary | Health-related quality of life | Health-related quality of life as assessed by scores from Cystic Fibrosis Questionnaire - Revised Parent Report (CFQ-R, German version), administered quarterly | during the 52 week treatment period | |
Secondary | Change in anthropometric and basic respiratory parameters | Change in weight, height, body mass-index, weight-for-height, resting respiratory rate, and room air oxygen saturation | during the 52 week treatment period | |
Secondary | Proportion of patients with new isolation of CF pathogen | Among participants from whom Pseudomonas aeruginosa or other CF pathogens were not isolated from respiratory cultures prior to enrolment, the proportion from whom these organisms are isolated from clinically collected respiratory cultures | during the 52 week treatment period | |
Secondary | Time to first isolation of a CF pathogen | Time to acquisition of a CF pathogen is going to be compared between both treatment groups | during the 52 week treatment period |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT02270476 -
Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following Newborn Screening
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