A Phase 2 Study to Evaluate the Safety, Biological Activity, and PK of ND-L02-s0201 in Subjects With IPF

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Jacksonville, Florida: 18-006194
    NCT ID: NCT03538301
    Sponsor Protocol Number: ND-L02-s0201-005

About this study

This phase 2 clinical study will be a randomized, double-blind, placebo-controlled, multicenter study to evaluate the safety, tolerability, biological activity, and pharmacokinetics (PK) of ND-L02-s0201 for Injection in subjects with idiopathic pulmonary fibrosis (IPF).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

Inclusion Criteria: 

  • Males and females between 40 and 80 years of age, inclusive, at the time of consent.
  • Diagnosis of IPF within 5 years before Visit 1a, confirmed by the Principal Investigator (PI) using American Thoracic Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Thoracic Association (ALAT) guidelines (Raghu et al, 2011 or Raghu et al, 2018).
    • a. Clinical management of the patient at the site must be for IPF and the PI must be convinced that all locally available information support UIP/IPF is the most likely diagnosis. It is mandatory that the PI consider overreader interpretation of Visit 1b HRCT and, if performed, SLB in order to determine that a subject is eligible for this trial.
  • Extent of fibrosis greater than emphysema on HRCT.
  • If on pirfenidone or nintedanib, unchanged dose for at least 12 weeks before Visit 1a.  Subjects may not be on both pirfenidone and nintedanib.
  • FVC ≥ 45% of predicted.
  • DL(co) corrected for hemoglobin ≥ 30% of predicted value.
  • Pulse oximetry saturation ≥ 90%, at rest while breathing ambient air or ≤ 2L/minute supplemental oxygen by nasal prongs/cannula.
  • Ratio of forced expiratory volume in 1 second (FEV1) to FVC ≥ 0.70.
  • Adequate liver and renal function, as demonstrated by:
    • Total bilirubin ≤ 1.5 × upper limit of normal (ULN), unless participant has a documented history of Gilbert’s syndrome;
    • Aspartate transaminase (AST) and alanine transaminase (ALT) < 2.5 × ULN;
    • Creatinine < 2 × ULN;
    • Serum albumin > 3.5 g/dL.
  • Life expectancy of ≥ 12 months.
  • Serum vitamin A level at Screening ≤ ULN of the central laboratory reference range.
  • Women of childbearing potential (WCBP) must be willing to use a highly effective method of contraception throughout the study and study follow up or for at least 90 days after the last dose of study treatment.
  • Women must be willing not to breastfeed for 90 days after the last dose of the study treatment.
  • Males must agree to use a condom throughout the study and for 90 days after the last dose of study treatment to prevent seminal transmission of the investigational product.
  • Males with female partners of childbearing potential must agree to use a highly effective method of contraception throughout the study and for 90 days after the last dose of study treatment. All men with female partners of childbearing potential will be instructed to contact the Investigator immediately if their partner becomes pregnant at any time during study participation. All men must agree not to donate semen throughout the study and for 90 days after the last dose of study treatment.
  • Willing and able to provide written informed consent and comply with the study procedures and visit schedule, including follow-up visits.

Exclusion Criteria:

  • Best, acceptable FVC from separate Screening spirometry that differ by ≥ 200 mL.
  • Respiratory exacerbation(s) or hospitalization for IPF exacerbation within 3 months before Visit 1a.
  • Taking both pirfenidone and nintedanib concurrently within 12 weeks before Visit 1a.
  • Anticipated to receive a lung transplant during the subject’s participation in the study.
  • Diagnosis of any connective tissue disease with a natural history that may be associated with pulmonary disease.
  • Clinical evidence of or known history of cirrhosis.
  • History of osteomalacia, Paget’s disease of bone, jawbone necrosis, or a history of unexplained fractures, or fractures after minimal trauma, as determined by the Investigator.
  • Uncontrolled cardiac disease or cardiac surgical procedure (e.g., New York Heart Association Class III or IV, myocardial infarction, transient ischemic attack, uncontrolled atrial or ventricular cardiac arrhythmias, unstable angina, stroke, coronary angioplasty, coronary artery bypass graft) within 30 days before Visit 1a.
  • Active smoker or smoking cessation within 12 weeks before Visit 1a.
  • Malignancy within the last 5 years, with the exception of curable cancer (e.g., basal or squamous cell skin cancer, cervical cancer in situ, nonmedullary thyroid carcinoma) that has been adequately treated (e.g., excision).
  • Evidence of any unstable or untreated, clinically significant disease or condition that, in the opinion of the Investigator, might confound the interpretation of the study or place the subject at increased risk (e.g., uncontrolled hypertension, diabetes mellitus). Subjects will have to have been on stable treatment for at least 4 weeks before Visit 1a.
  • Treatment with high dose corticosteroids, cytotoxic agents (e.g., chlorambucil, azathioprine, cyclophosphamide, methotrexate), unapproved IPF-targeted therapy, and cytokine modulating agents within 8 weeks or 5 half-lives (whichever is longer) before Visit 1a.
    • A dose of ≤ 15 mg/day prednisone, or equivalent, is acceptable if unchanged for ≥ 10 weeks before Visit 1a and is expected to remain unchanged during the subject’s participation in the study.
  • Receiving an investigational treatment, whether or not approved for marketing, with the last dose of that study drug within 8 weeks or 5 half-lives (whichever is longer) before Visit 1a.  Individuals allocated to receive no treatment beyond SOC in an investigational study are not excluded from this trial.
  • History of allergic reaction to any of the study drugs to be administered.
  • Pregnant or breastfeeding.
  • Veins unsuitable for repeated venipuncture or intravenous (IV) infusion (e.g., veins that are difficult to locate, access, or puncture; veins with a tendency to rupture during or after puncture), in the opinion of study center personnel.
  • Known history of human immunodeficiency virus (HIV) infection, active chronic hepatitis B (e.g., hepatitis B surface antigen positive), and/or untreated hepatitis C antigen positive patients (with or without abnormal liver enzymes). If treated for hepatitis C viral eradication, then a viral load below the limits of quantitative detection for at least 12 weeks must be documented.
  • History of alcohol abuse and/or dependence within the last 2 years, as determined by the Investigator.
  • History within the last 2 years of significant mental illness, or dependence on any opioid or illicit drugs as defined in Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5).

 

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Tarik Haddad, M.D.

Closed for enrollment

Contact information:

Judy Olmos R.N., C.C.R.C.

127or7837898

Olmos.Judy@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20446033

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