Idiopathic Pulmonary FibrosisPhase 1NCT04233814
Safety, Tolerability and Pharmacokinetic Study of LTI-03 in Healthy Adult Subjects
A Phase 1 study testing Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in people with Idiopathic Pulmonary Fibrosis, run by Rein Therapeutics at 1 site in United Kingdom.
Trial summary
The current study will investigate the initial safety, tolerability, and PK profile of inhaled LTI-03 in healthy volunteers. In order to minimize exposure, the study will first test single ascending doses (SAD) of LTI-03 followed by multiple ascending dose (MAD) cohorts. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Idiopathic Pulmonary Fibrosis
- What is being tested?
- Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 71 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 20 Jan 2020to 23 Dec 2021
- Phase
- Phase 1
- 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 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Non-smoker (no use of tobacco products within 6 months prior to dosing) with a negative urine cotinine test at Screening or Day -1
- Age of 18-55 years (inclusive)
- Body mass index (BMI) of 18 - 30.5 kg/m2 (inclusive)
- Body weight > 50 kg
- Willing and able to provide written informed consent
Who cannot join
- History of asthma
- Presence of active or recurring allergies, asthma, chronic obstructive pulmonary disease (COPD), chronic sinus drainage, chronic or acute cough or other respiratory condition deemed exclusionary by the Investigator or designee
- Pulmonary infiltrate or pneumonia within 6 months prior to dosing or acute infection within 14 days prior to dosing
- History of significant allergy or anaphylaxis
- Any clinically significant hematologic, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic disease (excluding hay fever), as determined by the Investigator or designee
- Any current clinically relevant abnormalities identified by a detailed medical history, complete physical examination including blood pressure and heart rate measurement, and clinical laboratory tests (hematology, coagulation, urinalysis, clinical chemistries) at Screening or Day -1, as determined by the Investigator or designee
Show all 30Show fewer
- Any clinically significant illness and/or surgery within 28 days prior to dosing
- Febrile illness within 7 days prior to dosing
- Weight loss > 5 kg within 28 days prior to dosing
- Clinically significant 12-lead electrocardiogram (ECG) abnormalities or vital sign abnormalities (systolic blood pressure < 90 mmHg or > 140 mmHg, diastolic blood pressure < 50 mmHg or > 90 mmHg, or heart rate < 45 beats per minute [bpm] or > 100 bpm) at Screening or Day -1, as determined by the Investigator or designee
- History of, or existing severe, acute, chronic, and/or psychiatric medical condition(s), laboratory abnormality, or other medical concerns that may increase the risk associated with study participation or IMP administration which, in the judgment of the Investigator, would make the subject inappropriate for entry into the study
- History of cancer with the exception of adequately treated basal cell or squamous cell carcinoma of the skin
- Hemoglobin < lower limit of normal (LLN)
- Abnormal liver function- alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 times the upper limit of the normal range (ULN)
- total bilirubin > 1.5 times ULN
- Abnormal renal function: estimated glomerular filtration rate (eGFR) (modification of diet and renal disease [MDRD]) < 55 mL/min/1.73 m2
- Pulmonary function outside the normal range, including forced expiratory volume 1 (FEV1), forced vital capacity (FVC) each < 80% of predicted or FEV1/FVC ratio of ≤ 0.7 at Screening
- Inability to use study inhaler device appropriately.
- Positive test results for human immunodeficiency virus (HIV) HIV-1/HIV-2 antibodies, hepatitis B surface antigen (HBsAG) or hepatitis C virus antibody (HCV-AB) Concurrent Intake of Other Substances
- History of alcohol abuse within one year prior to Screening or regular use of alcohol of ≥ 14 units of alcohol per week for females and ≥ 21 units of alcohol for males (1 unit = 150 mL wine, 360 mL beer or 45 mL of 40% alcohol) within 6 months prior to dosing or a positive urine alcohol test at Screening or Day -1
- History of drug abuse or misuse within 5 years prior to dosing or a positive urine drug test at Screening or Day -1
- Inability or unwillingness to abstain from alcohol or any drug of abuse for 48 hours prior to the first dose until completion of the Day 8 visit for the SAD and Day 21 visit for the MAD
- Exposure to any live vaccines within 28 days prior to dosing
- Treatment with an investigational product within 30 days or 5 half-lives (whichever is longer) prior to dosing
- Use of prescription or non-prescription medications and dietary supplements within 14 days or 5 half-lives (whichever is longer) prior to the first dose of study medication. Herbal supplements must be discontinued 28 days prior to dosing. Approved medications taken for contraception are permitted.
- Positive serum pregnancy test in female subjects
- Female subjects who are lactating
- Female subjects of childbearing potential (FOCBP) and men with partners of childbearing potential who do not agree to use an acceptable form of contraception for the duration of study treatment and for at least 90 days after the last dose of study medication. Male subjects who do not agree to refrain from donating sperm during this same period.
- Not eligible to receive study medication within 2 weeks of receiving a COVID-19 vaccination, including an initial, second, or booster injection.
- NOTE: Female who is surgically sterile or post-menopausal for at least 12 months with follicle stimulating hormone (FSH) > 30 mIU/ml, are not considered to be of childbearing potential.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Ph 1a, First-in-Man, Single Ascending Dose & Multiple Ascending Dose Safety, Tolerability and PK Study of a Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in Healthy Adult Subjects
The current study will investigate the initial safety, tolerability, and PK profile of inhaled LTI-03 in healthy volunteers. In order to minimize exposure, the study will first test single ascending doses (SAD) of LTI-03 followed by multiple ascending dose (MAD) cohorts.
Findings from this study will direct the clinical development of LTI-03 for the treatment of IPF
Read more
The study subject population will include normal healthy male and female volunteers between 18 and 55 years of age (inclusive).
Consistent with other trials involving inhaled medication, subjects must have normal pulmonary function at Screening and will be excluded if they have a history of active or recurring allergies, asthma, chronic obstructive pulmonary disease (COPD), chronic sinus drainage, chronic or acute cough or other respiratory condition deemed exclusionary by the Investigator. History of liver dysfunction or elevated bilirubin, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values at Screening will also be grounds for exclusion.
- Conditions
- Idiopathic Pulmonary Fibrosis
- Being tested
- Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03), Placebo
- Medical subject headings
- Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)Drug
LTI-03, a Caveolin-1 scaffold protein-derived 7-amino acid peptide to be administered as a dry powder by inhalation through a dry powder inhaler.
PlaceboDrug
Matching placebo is micronized lactose powder administered by inhalation through a dry powder inhaler.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Sham comparator | Matching placebo is a micronized lactose powder administered by inhalation through a dry powder inhaler (DPI) Receives: Drug: Placebo |
| SAD Cohort 1 | Experimental | LTI-03 20 mg delivered qd x 1 day via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| SAD Cohort 2 | Experimental | LTI-03 40 mg delivered qd x 1 day via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| SAD Cohort 3 | Experimental | LTI-03 80 mg delivered qd x 1 day via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| MAD Cohort 1 | Experimental | LTI-03 dose at 20mg once daily x 14 days via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| MAD Cohort 2 | Experimental | LTI-03 dose at 40mg once daily x 14 days via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| MAD Cohort 3 | Experimental | LTI-03 dose at 2.5 mg once daily x 14 days via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| MAD Cohort 4 | Experimental | LTI-03 dose at 5 mg once daily x 14 days via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
| MAD Cohort 5 | Experimental | LTI-03 dose at 5 mg twice daily x 14 days via DPI Receives: Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (TEAE)
Incidence of TEAE by system-organ class and dose group as assessed by the Toxicity Grading Scale for Healthy Adult Volunteers
Measured: up to 49 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2020
- Study start20 Jan 2020
- Primary completion23 Dec 2021
- Study completion23 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Pulmonary FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- The first part of the study will follow a Single Ascending Dose design. Subjects receive a single dose of double-blind study medication (investigational medicinal product LTI-03 or placebo) on Day 1. For each dose level cohort, two eligible sentinel subjects will be randomized in a 1:1 ratio to receive active:placebo study medication. If no tolerability issues are observed by the Investigator within 48 hours post-dose, dosing will commence for the remaining six subjects in the cohort, who will be randomized in a 5:1 ratio to receive active:placebo study medication. The second part of the study will follow a Multiple Ascending Dose design. Subjects receive double-blind study medication (LTI-03 or placebo) once-daily from Day 1 to Day 14. For each dose level cohort, eight eligible subjects will be randomized in a 6:2 ratio to receive active:placebo study medication.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rein Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Devinda Weeraratne, MD, Principal investigator, Celerion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04233814
- Sponsor's protocol ID
- LTI-03-1001
- Status last verified
- Mar 2022
- First submitted
- 12 Jan 2020
- Last updated
- 7 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Ph 1a, First-in-Man, Single Ascending Dose & Multiple Ascending Dose Safety, Tolerability and PK Study of a Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT04233814
- Acronym / sponsor ID
- LTI-03-1001
- Conditions
- Idiopathic Pulmonary Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Rein TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Jan 2020 (actual)
- Primary completion
- 23 Dec 2021 (actual)
- Study completion
- 23 Dec 2021 (actual)
- First posted
- 18 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The current study will investigate the initial safety, tolerability, and PK profile of inhaled LTI-03 in healthy volunteers. In order to minimize exposure, the study will first test single ascending doses (SAD) of LTI-03 followed by multiple ascending dose (MAD) cohorts.
Findings from this study will direct the clinical development of LTI-03 for the treatment of IPF
The study subject population will include normal healthy male and female volunteers between 18 and 55 years of age (inclusive).
Consistent with other trials involving inhaled medication, subjects must have normal pulmonary function at Screening and will be excluded if they have a history of active or recurring allergies, asthma, chronic obstructive pulmonary disease (COPD), chronic sinus drainage, chronic or acute cough or other respiratory condition deemed exclusionary by the Investigator. History of liver dysfunction or elevated bilirubin, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values at Screening will also be grounds for exclusion.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 9
- Model description
- The first part of the study will follow a Single Ascending Dose design. Subjects receive a single dose of double-blind study medication (investigational medicinal product LTI-03 or placebo) on Day 1. For each dose level cohort, two eligible sentinel subjects will be randomized in a 1:1 ratio to receive active:placebo study medication. If no tolerability issues are observed by the Investigator within 48 hours post-dose, dosing will commence for the remaining six subjects in the cohort, who will be randomized in a 5:1 ratio to receive active:placebo study medication. The second part of the study will follow a Multiple Ascending Dose design. Subjects receive double-blind study medication (LTI-03 or placebo) once-daily from Day 1 to Day 14. For each dose level cohort, eight eligible subjects will be randomized in a 6:2 ratio to receive active:placebo study medication.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Sham comparator | Drug: PlaceboMatching placebo is a micronized lactose powder administered by inhalation through a dry powder inhaler (DPI) |
| SAD Cohort 1 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 20 mg delivered qd x 1 day via DPI |
| SAD Cohort 2 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 40 mg delivered qd x 1 day via DPI |
| SAD Cohort 3 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 80 mg delivered qd x 1 day via DPI |
| MAD Cohort 1 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 dose at 20mg once daily x 14 days via DPI |
| MAD Cohort 2 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 dose at 40mg once daily x 14 days via DPI |
| MAD Cohort 3 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 dose at 2.5 mg once daily x 14 days via DPI |
| MAD Cohort 4 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 dose at 5 mg once daily x 14 days via DPI |
| MAD Cohort 5 | Experimental | Drug: Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03 dose at 5 mg twice daily x 14 days via DPI |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Non-smoker (no use of tobacco products within 6 months prior to dosing) with a negative urine cotinine test at Screening or Day -1
- Age of 18-55 years (inclusive)
- Body mass index (BMI) of 18 - 30.5 kg/m2 (inclusive)
- Body weight > 50 kg
- Willing and able to provide written informed consent
Key exclusion criteria (30 listed)
- History of asthma
- Presence of active or recurring allergies, asthma, chronic obstructive pulmonary disease (COPD), chronic sinus drainage, chronic or acute cough or other respiratory condition deemed exclusionary by the Investigator or designee
- Pulmonary infiltrate or pneumonia within 6 months prior to dosing or acute infection within 14 days prior to dosing
- History of significant allergy or anaphylaxis
- Any clinically significant hematologic, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic disease (excluding hay fever), as determined by the Investigator or designee
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03)LTI-03, a Caveolin-1 scaffold protein-derived 7-amino acid peptide to be administered as a dry powder by inhalation through a dry powder inhaler. | Drug | MAD Cohort 1, MAD Cohort 2, MAD Cohort 3, MAD Cohort 4, MAD Cohort 5, SAD Cohort 1, SAD Cohort 2, SAD Cohort 3 |
| PlaceboMatching placebo is micronized lactose powder administered by inhalation through a dry powder inhaler. | Drug | Placebo |
Primary outcome
Incidence of Treatment-Emergent Adverse Events (TEAE)
Time frame: up to 49 days
Incidence of TEAE by system-organ class and dose group as assessed by the Toxicity Grading Scale for Healthy Adult Volunteers
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Devinda Weeraratne, MD principal investigator
Celerion
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in people with Idiopathic Pulmonary Fibrosis, run by Rein Therapeutics at 1 site in United Kingdom.
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 5 inclusion and 30 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: finished, as of the sponsor's last update on 7 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Treatment-Emergent Adverse Events (TEAE) (time frame: up to 49 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Mar 2022.