Cystic Fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: SNSP113
Product Code: SNSP113
Pharmaceutical Form: Nebulisation solution
INN or Proposed INN: not assigned
CAS Number: 942196-15-8
Current Sponsor code: PAAG
Other descriptive name: PAA
Sponsors
Synspira Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject voluntarily agrees to participate in this study, is able and willing to comply with the protocol and signs a written Ethics Committee (EC) approved informed consent prior to study-specific screening procedures being performed; 2. Male and female subjects 18 to 65 years of age (inclusive), on the day of signing informed consent; 3. Documented and confirmed diagnosis of pulmonary CF as defined by CF signs and symptoms AND genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype; or sweat chloride >60 mEq/L; 4. Body mass index (BMI) of =18.0 kg/m2; 5. Spirometry at Screening demonstrating a forced expiratory volume in 1 second (FEV1) of =40% and =90% of predicted normal for age, sex, and height; 6. Stable pulmonary symptoms associated with CF for 28 days prior to Screening and between the Screening visit and Day 1 as determined by Investigator; 7. Fulfilling the following laboratory criteria at Screening or results that are considered not clinically significant by the Investigator: a) hepatic function tests (alkaline phosphatase [ALP], alanine aminotransferase [ALT], aspartate aminotransferase [AST], gamma-glutamyl transaminase [GGT], bilirubin) =3 x upper limit of normal (ULN); b) hemoglobin =10 g/dL, platelets =75,000/mm3, neutrophils =1.0 x 109/L; c) serum creatinine =2.0 mg/dL (177 µmol/L); 8. Results of ECG recording at Screening are within normal limits or deemed to be not clinically significant by the Investigator; 9. Able and willing to comply with scheduled visits, treatment plan, study restrictions, spirometry laboratory tests, contraceptive guidelines, and other study procedures as determined by the Investigator; 10. Female subjects of child-bearing potential must either be sexually inactive (abstinent) from Day -28 until 28 days after the last dose of study product (SNSP113 or placebo) or be willing to use highly effective methods of birth control from Day -14 until 28 days after the last dose of study product. For the purpose of this study highly effective contraception is defined as: a) oral, intravaginal, or transdermal combined (estrogen and progestogen containing) hormonal methods of contraception; OR b) oral, injected or implanted (progestogen only) hormonal methods of contraception (e.g., Depo-Provera®, Implanon®); OR c) placement of an intrauterine device (IUD) or intrauterine system (IUS). 11. Females of non-childbearing potential must be postmenopausal with documented amenorrhea for at least 1 year prior to dosing and follicle-stimulating hormone (FSH) serum levels consistent with postmenopausal status OR undergone sterilization procedures at least 6 months prior to dosing such as: a) hysteroscopic sterilization; b) bilateral tubal ligation or salpingectomy; c) hysterectomy; d) bilateral oophorectomy. 12. All male subjects must either be sexually inactive (abstinent) or be willing to use a condom with spermicide during the study from Day 1 until 28 days after the last dose of study product. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 56 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subjects with significant clinical/laboratory/radiological/spirometry sign(s) within 28 days prior to Screening or between the Screening visit and Day 1 that, in the opinion of the Investigator, is indicative of unstable respiratory disease; 2. Positive screening tests for hepatitis B surface antigen (HBsAG), anti-hepatitis C virus (HCV) antibodies and anti-Human Immunodeficiency Virus (HIV) 1 and 2 antibodies; positive laboratory-confirmed SARS-CoV-2 (COVID-19) infection as determined by RT-PCR, or other commercial or public health assay in any specimen 3 times per week) of a high-dose NSAID (e.g., >1.6 g ibuprofen/day) within 28 days prior to Screening or at any time between the Screening visit and Day 1; 6. Current or former regular smoker (tobacco, e-cigarette, or cannabis products) within the last 6 months; 7. Not willing to abstain from use of cannabis products for the duration of the study; 8. History of alcohol or substance abuse in the past 12 months and/or positive drug test at Screening. Presence of drug(s) administered for known conditions are permitted with concurrence from the Sponsor’s medical monitor (e.g., benzodiazepine); 9. Participation in an interventional clinical trial involving the receipt of an investigational product or device within the 28 days prior to Screening or 6 half-lives of an investigational product, whichever is longer; 10. Female subjects who are pregnant, have a positive serum pregnancy test at Screening, are lactating or who plan to become pregnant during the study and/or within 90 days after receiving the study product; 11. History of organ transplantation or listed for organ transplantation; 12. Subjects requiring supplemental oxygen; 13. Subjects with previous or current pneumothorax within 3 months or any other condition that would contraindicate the performance of spirometry in the judgement of the Investigator; 14. Hemoptysis more than 30 mL over 24 h at any time within 28 days prior to Screening or at any time between Screening visit and Day 1; 15. History of malignancy <5 years prior to signing the informed consent, except for treated basal cell or squamous cell skin cancer or in situ cervical cancer; 16. Subjects who, per Investigator's judgment, are not ideal clinical trial candidates due to personal reasons (e.g., perceived willingness or ability to comply with protocol and study procedures) that would likely impede the subject's successful study completion. 17. Sexually active male subjects with female partners of child-bearing potential not willing to use effective methods of birth control during the study from Day -14 until 28 days after the last dose of study product.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of multiple ascending doses (MAD) of inhaled SNSP113 administered once-daily for 28 days to adult Cystic Fibrosis (CF) subjects. ;Secondary Objective: No secondary objectives;Primary end point(s): • Vital signs (blood pressure, heart rate, respiratory rate) • Clinical laboratory evaluations (hematology included complete blood cell count (CBC) with differential and platelet count, serum biochemistry including blood urea nitrogen and creatinine, liver function tests, coagulation profile, and urinalysis) • ECG parameters • Pulmonary function assessed by spirometry, pulse oximetry • PE;Timepoint(s) of evaluation of this end point: Visit 1 through study completion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): not applicable;Timepoint(s) of evaluation of this end point: not applicable | — |
Countries
Hungary
Contacts
Public ContactRegulatory Unit
Accelsiors CRO and Consultancy Services Ltd
Outcome results
None listed