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Idiopathic Pulmonary FibrosisPhase 2NCT03591926

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of SM04646 Inhalation Solution in Subjects With Idiopathic Pulmonary Fibrosis (IPF)

A Phase 2 study testing SM04646 in people with Idiopathic Pulmonary Fibrosis, run by Biosplice Therapeutics, Inc. at 6 sites in 2 countries.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

SM04646-IPF-03 is a Phase 2a, multi-center, open-label study evaluating the safety and efficacy of a single inhaled, nebulized dose of SM04646 solution over a 12-week treatment regimen in subjects with mild to moderate IPF. Read more about this study

Who is it for?
Ages 40 to 80 years
Conditions the study is for
Idiopathic Pulmonary Fibrosis
What is being tested?
SM04646
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
6 sitesin 2 countries
Study dates
Jan 1900to Jan 1900
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 40 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • IPF diagnosis within 5 years of study start based upon thoracic society guidelines and confirmed by Investigator at study start
  • Able to walk > 150 m in 6 Minute Walk Test without the use of supplemental oxygen at study start
  • Has a life expectancy of at least 12 months in the opinion of the Investigator
  • Full understanding of the requirements of the study and willingness and ability to comply with all study visits and procedures
  • Able to comprehend and willing to sign an informed consent form (ICF) prior to any study-related procedure being performed
  • Able to tolerate and complete placebo (vehicle) inhalation for 10 minutes without experiencing a significant cough, in the opinion of the Investigator
  • Subjects currently treated with pirfenidone or nintedanib must be willing to remain on their current treatment for the duration of the protocol, unless they experience rapid progression, or if, in the opinion of the Investigator, treatment adjustments are necessary

Who cannot join

  • Women who are pregnant or lactating
  • Women of childbearing potential who are sexually active and are not willing to use an appropriate method of birth control during the study treatment period until 90 days post study medication administration
  • Males who are sexually active and not willing to use a condom, and have a partner who is capable of becoming pregnant, if neither has had surgery to become sterilized, and/or who are not willing to use an appropriate method of birth control during the study treatment period until 90 days post study medication administration
  • Males unwilling to refrain from sperm donation during the study treatment period until 90 days post study medication administration
  • Subjects unwilling to refrain from blood and plasma donation during the study treatment period until 90 days post study medication administration
  • A history of abuse of prescription or illicit drugs within 6 months prior to study start
Show all 48Show fewer
  • Positive urine drug and alcohol screen with the exception of positive findings related to current prescription therapy at study start
  • Occurrence of serious illness requiring hospitalization within 90 days prior to study start
  • Presence of active infections at study start
  • Current smoker or past history of smoking (e.g., cigarettes, e-cigarettes, pipes, cigars) within 6 months of study start or >50 pack years
  • Use of non-inhaled tobacco- or nicotine-containing products (e.g., chewing tobacco, nicotine gum, lozenges, or patches) within 30 days prior to study start until completion of the study
  • Regular alcohol consumption exceeding 7 drinks/week for females or 14 drinks/week for males (1 drink = 5 ounces [150 mL] of wine or 12 ounces [360 mL] of beer or 1.5 ounces [45 mL] of hard liquor) within 30 days prior to study start until completion of the study
  • Lung transplantation anticipated during the duration of the trial
  • Subjects receiving treatment with pirfenidone or nintedanib that:
  • Have been on treatment for less than 12 weeks prior to study start
  • Have not been on a stable dose for at least 30 days prior to study start
  • Subjects who are not currently on but have previously received pirfenidone or nintedanib that have not been off of pirfenidone or nintedanib for at least 30 days prior to study start
  • Receipt of any of the following medication or treatment prior to study start:
  • N-acetylcysteine prescribed for the treatment of IPF within 30 days prior to study start
  • Previous therapeutic radiation treatment of the lungs, mediastinum, or chest wall
  • Participation in a clinical research trial that included the receipt of an investigational product or any experimental therapeutic procedure within 30 days or 5 half-lives of the investigational product (if known), whichever is longer, prior to study start
  • Immunosuppressive medications [e.g., methotrexate, cyclosporine, azathioprine, systemic or inhaled glucocorticosteroids with the exception of short term use of systemic glucocorticosteroids less than or equal to 10 mg of prednisone daily (or equivalent) for a non-IPF condition such as an allergic reaction or rash] within 8 weeks prior to study start
  • Use of any therapy targeted to treat IPF (including but not limited to d-penicillamine, endothelium receptor antagonist [e.g., bosentan, ambrisentan], and interferon gamma-1B) within 30 days prior to study start
  • Use of any cytokine modulator (etanercept, adalimumab, efalizumab, infliximab, golimumab, certolizumab, rituximab) within 90 days or 5 half-lives, whichever is longer, prior to study start
  • A bronchodilator used within 1 week of study start
  • SM04646
  • A "bronchodilator response" at study start, defined by an absolute increase of ≥ 12% and an increase of 0.2 L in FEV1 or FVC, or both, after bronchodilator use compared with the values before bronchodilator use
  • History of any of the following conditions:
  • Pulmonary embolism or pulmonary hypertension
  • Creatinine clearance of less than 50mL per minute
  • Active tuberculosis (TB) infection or history of incompletely treated latent TB infection
  • History of malignancy within the last 5 years; however, the following subjects are eligible:
  • Subjects with prior history of in situ cancer or basal or squamous cell skin cancer that has been completely excised
  • Subjects with other malignancies if they have been continuously disease free for at least 5 years prior to any study drug administration
  • Subjects with prostate cancer followed by surveillance.
  • Any connective tissue disease, including but not limited to scleroderma, systemic lupus erythematosus, rheumatoid arthritis, and polymyositis/dermatomyositis
  • Congenital respiratory conditions (e.g., cystic fibrosis)
  • Chronic obstructive pulmonary disease (COPD) or asthma
  • Current or recent respiratory tract infection (e.g., pneumonia, purulent bronchitis, or viral upper respiratory tract infection) within 30 days prior to study start
  • Acute exacerbation of IPF, in the opinion of the Investigator, within 30 days prior to study start
  • Human immunodeficiency virus (HIV), hepatitis C, or active hepatitis B infection
  • Clinically significant hepatic impairment (e.g., Child-Pugh A or greater severity)
  • Symptomatic coronary artery disease, congestive heart failure (CHF) New York Heart Association (NYHA) Class II-IV, myocardial infarction (MI), or unstable angina within 6 months prior to study start
  • Hypertension (systolic blood pressure (SBP) >160 millimeters of mercury (mmHg) or diastolic blood pressure (DBP) >100 mmHg)
  • Hypotension (blood pressure (BP) less than 90/60 mmHg) or mean arterial blood pressure (MAP) < 65 mmHg
  • Current use of supplemental oxygen therapy for any condition
  • Subjects who are immediate family members (spouse, parent, child, or sibling; biological or legally adopted) of personnel directly affiliated with the study at the investigative site, or are directly affiliated with the study at the investigative site
  • Subjects employed by Samumed Pacific Pty Ltd, or any of its affiliates or development partners (that is, an employee, temporary contract worker, or designee) responsible for the conduct of the study

Trial locations and contacts

6 sites in 2 countries.

6 sites in 2 countries

Australia4 sites

  • Research Site

    Bedford Park, South Australia

  • Research Site

    Camperdown, New South Wales

  • Research Site

    Clayton, Victoria

  • Research Site

    Concord, New South Wales

Other trials in Australia

New Zealand2 sites

  • Research Site

    Christchurch

  • Research Site

    Dunedin

Other trials in New Zealand

About this study

Official title: A Phase 2a, 24-Week, Multi-Center, Open-Label Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of SM04646 Inhalation Solution in Subjects With Idiopathic Pulmonary Fibrosis (IPF)

SM04646-IPF-03 is a Phase 2a, multi-center, open-label study evaluating the safety and efficacy of a single inhaled, nebulized dose of SM04646 solution over a 12-week treatment regimen in subjects with mild to moderate IPF. A total of approximately 24 subjects will be enrolled in the study (approximately 12 subjects into the "non-bronchoalveolar lavage [BAL]" arm and approximately 12 subjects into the "BAL" arm). Subjects that currently do not require, have failed to tolerate, or have opted not to have treatment with pirfenidone or nintedanib will have the option of participation in the "BAL" arm or participation in the "non-BAL" arm. Subjects currently receiving treatment with pirfenidone or nintedanib must be on stable treatment for a minimum of 12 weeks prior to the Screening Visit. Subjects currently on treatment with pirfenidone or nintedanib may participate in the "non-BAL" arm only.

Eligible subjects will participate in a treatment period of 12 weeks and a follow-up period of 12 weeks. The treatment dosing pattern will follow a 2 weeks on, 2 weeks off regimen, wherein subjects will dose 5 consecutive days of each 7 day "on" week.

Being tested
SM04646
Keywords
SM04646, IPF, nebulized, inhalation
Medical subject headings
Idiopathic Pulmonary Fibrosis, Respiratory Aspiration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SM04646Drug

    Nebulized, inhaled solution; single dose concentration dosed once per day for 12 weeks; dosing pattern will follow a 2 weeks on, 2 weeks off regimen, wherein subjects will dose 5 consecutive days of each 7 day "on" week.

Study groups in NCT03591926
Study groupRoleWhat they receive
"BAL" ArmExperimentalSubjects in this arm will undergo a bronchoalveolar lavage (BAL) procedure at baseline and after two weeks of treatment.
Receives: Drug: SM04646
"Non-BAL" ArmExperimentalSubjects in this arm will not undergo any BAL procedures.
Receives: Drug: SM04646

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and tolerability: treatment-emergent adverse events (TEAEs)

    Evaluate the safety and tolerability of SM04646 as measured by TEAEs during the entire treatment period

    Measured: Week 24

  2. Safety and tolerability: number of subjects with a clinically significant change from baseline in clinical laboratory tests

    Evaluate the safety and tolerability of SM04646 as measured by the number of subjects with a clinically significant change from baseline in clinical laboratory tests

    Measured: Week 24

  3. Safety and tolerability: number of subjects with a clinically significant change from baseline in vital signs

    Evaluate the safety and tolerability of SM04646 as measured by the number of subjects with a clinically significant change from baseline in vital signs

    Measured: Week 24

  4. Safety and tolerability: number of subjects with a clinically significant change from baseline in oxygen saturation

    Evaluate the safety and tolerability of SM04646 as measured by the number of subjects with a clinically significant change from baseline in oxygen saturation

    Measured: Week 24

  5. Safety and tolerability: number of subjects with a clinically significant change from baseline in electrocardiogram (ECG) parameters

    Evaluate the safety and tolerability of SM04646 as measured by the number of subjects with a clinically significant change from baseline in ECG parameters

    Measured: Week 24

  6. Plasma pharmacokinetics (PK): Cmax

    Measure maximum observed concentration of SM04646 (Cmax) in blood plasma

    Measured: Baseline and Week 10

  7. Plasma PK: tmax

    Measure time to SM04646 Cmax in blood plasma

    Measured: Baseline and Week 10

  8. Plasma PK: AUC

    Measure the area under the plasma concentration-time curve (AUC) for SM04646 in blood plasma

    Measured: Baseline and Week 10

  9. Plasma PK: t 1/2

    Measure the terminal phase half-life (t 1/2) of SM04646 in blood plasma

    Measured: Baseline and Week 10

  10. Plasma PK: accumulation ratio

    Measure the accumulation ration of SM04646 in blood plasma

    Measured: Baseline and Week 10

  11. Change from baseline in concentration of SM04646 in BAL fluid ("BAL" arm only)

    Measure concentration of SM04646 in BAL fluid prior to dosing and after two weeks of dosing

    Measured: Baseline and Week 2

Secondary outcomes (11)

  1. Change from baseline of forced vital capacity (FVC) (% predicted)

    Measured: Baseline and Week 24

  2. Change from baseline of FVC (liters)

    Measured: Baseline and Week 24

  3. Categorical analysis of FVC (% predicted) change

    Categories measured as "improved", "stable", "moderate decline", or "severe decline"

    Measured: Baseline and Week 24

  4. Time to disease progression

    Disease progression as defined by death, absolute decline ≥ 10% in FVC (% predicted), or respiratory hospitalization

    Measured: Week 24

  5. Change from baseline of forced expiratory volume in 1 second (FEV1) (% predicted)

    Measured: Baseline and Week 24

  6. Change from baseline of FEV1 (liters)

    Measured: Baseline and Week 24

  7. Change from baseline of diffusion capacity of the lung for carbon monoxide (DLCO) (% predicted corrected for hemoglobin)

    Measured: Baseline and Week 24

  8. Change from baseline of quantitative high-resolution computed tomography (HRCT) (%) and mL)

    Measured: Baseline and Week 24

  9. Change from baseline of quantitative high-resolution computed tomography (HRCT) (mL)

    Measured: Baseline and Week 24

  10. Change from baseline of qualitative HRCT

    Change measured as "improved", "same" or "worse"

    Measured: Baseline and Week 24

  11. Change from baseline of biomarker concentration isolated from serum

    Measured: Baseline and Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Jul 2018
  2. Study startJan 1900 (estimated)
  3. Primary completionJan 1900 (estimated)
  4. Study completionJan 1900 (estimated)

Why it stopped: Study withdrawn for business reasons prior to screening or enrolling any subjects.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03591926
Sponsor's protocol ID
SM04646-IPF-03
Status last verified
Oct 2018
First submitted
9 Jul 2018
Last updated
9 Oct 2018
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SM04646 in people with Idiopathic Pulmonary Fibrosis, run by Biosplice Therapeutics, Inc. at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 48 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 9 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and tolerability: treatment-emergent adverse events (TEAEs) (time frame: Week 24). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03591926.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2018.

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