Idiopathic Pulmonary Fibrosis (IPF)Phase 1/2STOP-IPFNCT04598919
Saracatinib in the Treatment of Idiopathic Pulmonary Fibrosis
A Phase 1/2 study testing Saracatinab in people with Idiopathic Pulmonary Fibrosis (IPF), run by National Jewish Health at 4 sites in United States.
Trial summary
Scarring of the lung, termed pulmonary fibrosis (PF), is a chronic, progressive, and usually fatal disorder. While two anti-fibrotic drugs have been approved for treating PF of unknown cause (idiopathic pulmonary fibrosis or IPF), neither drug is curative, and nearly 40% of patients stop taking the prescribed drug… Read more about this study
- Who is it for?
- Ages 40 years and older
- Conditions the study is for
- Idiopathic Pulmonary Fibrosis (IPF)
- What is being tested?
- Saracatinab
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 49 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 Nov 2020to 30 Jun 2025
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- IPF of any duration, confirmed or diagnosed by ILD center or expert according to Fleischner Guidelines (33). Subjects with a probable or indeterminant CT scan who otherwise meet the Fleischner criteria for IPF are eligible to be included in the study after a multidisciplinary evaluation. A positive Envisia genomic classifier score (34) on a lung biopsy specimen will be considered as strong evidence for a diagnosis of IPF. Subjects with a positive invisia genomic classifier score in conjunction with a probable or indeterminant CT scan are eligible to be included in the study after a multidisciplinary evaluation.
- Women or men >40 years of age at the time of screening
- FVC%>45% of predicted value (GLI-2012)
- Single breath DLCO% ≥ 30 - inclusive of predicted (without bronchodilator and uncorrected for hemoglobin GLI-2017)
- FEV1/FVC>70 (GLI-2012)
- Provision of signed/dated written informed consent prior to any study-specific procedures
- Females must be of nonchildbearing potential (defined as surgically sterilized [i.e., bilateral tubal ligation, bilateral oophorectomy or complete hysterectomy] or postmenopausal [defined as 12 months with no menses without an alternative medical cause] with a follicle-stimulating hormone [FSH] > 25.8 IU/L) or use a highly effective method of contraception (defined as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation; progestogen only hormonal contraception associated with inhibition of ovulation; intrauterine device (IUD); intrauterine hormone-releasing system (IUS) for the duration of the study (from the time they sign consent) and for 3 months after the last dose of drug/matching placebo
- Male subjects must be surgically sterile or using an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) for the duration of the study (from the time they sign consent) and for 3 months after the last dose of drug/matching placebo to prevent pregnancy in a partner. Male subjects must not donate or bank sperm for the duration of the study (from the time they sign consent) and for 3 months after the last dose of drug/matching placebo.
Who cannot join
- Requirement for supplemental oxygen > 4 L/min at rest to maintain saturation > 90%
- Active infection at screening or randomization
- Known active or latent hepatitis B or C
- Life expectancy for disease other than IPF < 2.5 years (Investigator assessment)
- Listed for lung transplantation
- Taking pirfenidone or nintedanib in the last 4 weeks
Show all 23Show fewer
- Pregnancy or lactation
- Known allergic reactions to components of saracatinib
- Treatment with another investigational drug or other intervention within 8 weeks
- Current smoker or tobacco use within 4 months
- Major surgery within the past 2 months
- Advanced hematologic, renal, hepatic, any lung disease determined by the investigator to be non-IPF related or metabolic disease that, in the opinion of the investigator, would make it unsafe for the person to receive study drug.
- Previous lung transplantation
- Inability to attend scheduled study visits
- Inability to give informed consent
- Inability to perform pulmonary function testing
- History of malignancy in the past two years, other than squamous or basal cell skin cancer
- Previous acute exacerbation of IPF requiring hospitalization and/or antibiotics within 90 days before the first dose of the investigational product
- Liver function test results ≥3× upper limit of normal (ULN) liver isoform of aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) or ≥2×ULN total bilirubin (excepting documentation of benign hereditary cause). An isolated total bilirubin elevation (ie, no significant concomitant elevation in ALT or AST) at baseline of ≤ 2xULN is permitted. If there is concomitant elevation in ALT or AST to ≤3xULN, then the threshold for total bilirubin is ≤1.5xULN.
- Creatinine clearance <30 mL/min calculated by Cockcroft-Gault formula
- Known pulmonary hypertension (PH) requiring PH-specific treatment
- Chronic oral corticosteroids at doses greater than prednisone 10 mg/day (or equivalent)
- Refer to 6.5 Concomitant Therapy for exclusions based on co-medications
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Baylor University Medical Center (BUMC)
Dallas, Texas, 75246
National Jewish Health
Denver, Colorado, 80206
Yale University School of Medicine
New Haven, Connecticut, 06510
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: Use of the Src Family Kinase Inhibitor Saracatinib in the Treatment of Idiopathic Pulmonary Fibrosis
Scarring of the lung, termed pulmonary fibrosis (PF), is a chronic, progressive, and usually fatal disorder. While two anti-fibrotic drugs have been approved for treating PF of unknown cause (idiopathic pulmonary fibrosis or IPF), neither drug is curative, and nearly 40% of patients stop taking the prescribed drug within a year because of side effects. The study includes the use of saracatinib, an investigational drug originally developed to treat certain types of cancers, in the treatment of IPF in a Phase 1b/2a clinical trial.
The objectives of this study are to: i) evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics, and to explore the efficacy of saracatinib in IPF; ii) identify biomarkers of Src kinase activity and fibrogenesis linked to pulmonary fibrosis; and iii) explore the application of these biomarkers to assess the anti-fibrotic effect of saracatinib in IPF patients
Read the detailed description
This is a double blind, randomized, placebo-controlled, single-dose, four-site trial. The trial is a biomarker based, integrated Phase 1b/2a clinical trial involving 100 subjects. One group (n=50) will receive placebo, while the other group (n=50) will receive 125 mg of oral saracatinib once daily.
Randomization will be stratified by center. The randomization scheme will be in random blocks of 2 and 4 within each stratum to maintain balance. In the second part of the trial, we will use a simple randomization scheme to achieve the 8:1 randomization across sites. The study is designed to have interim analysis of the drop-out rates when approximately 30% of the randomized patients have achieved the 24-week assessment. Should the drop-out rate be higher than the 20% that is anticipated, a new sample size calculation will be performed to make sure that the power of the study is maintained at 80% .
Duration of follow-up will be 28 weeks including 24 weeks of treatment with saracatinib or placebo.
- Conditions
- Idiopathic Pulmonary Fibrosis (IPF)
- Being tested
- Saracatinab, Placebo
- Keywords
- Idiopathic Pulmonary Fibrosis, Scarring of the lung, Saracatinib, randomized controlled trial
- 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.
SaracatinabDrug
125 mg once daily by mouth for 24 weeks
PlaceboDrug
once daily by mouth for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Saracatinab | Active comparator | saracatinib 125 mg once daily by mouth for 24 weeks Receives: Drug: Saracatinab |
| Placebo | Placebo comparator | matching placebo once daily by mouth for 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of saracatinib in IPF as measured by frequency of adverse events
Safety data will be listed and summarized with patient counts and percentages in each treatment arm
Measured: 24 weeks
Tolerability of saracatinib in IPF as measured by Severity of adverse events
A listing of all adverse events by patient will be presented. This listing will include patient number, adverse event (actual term and preferred term), event stand and end dates, CTCAE grade, relationship to the study drug/procedure, seriousness and outcome. A listing of SAEs will be produced using the similar format. This is not a scale. It is a data capture tool.
Measured: 24 weeks
Pharmacokinetics of saracatinib in IPF as measured by serum levels
Serum levels of saracatinib
Measured: 24 weeks
Pharmacodynamics of saracatinib in IPF as measured by change in serum β-CTX
Change in serum β-CTX as a Src kinase dependent biomarker
Measured: 24 weeks
Efficacy of saracatinib in IPF as measured by change in FVC
Change in FVC from baseline
Measured: 24 weeks
Secondary outcomes (5)
Efficacy of saracatinib in IPF (HRCT)
Change in HRCT quantitative analysis of the extent of pulmonary fibrosis. The analysis of HRCT data will involve data-driven texture analysis (DTA), a machine learning method capable of automatic detection and quantification of lung fibrosis on HRCT
Measured: 24 weeks
Efficacy of saracatinib in IPF (DLCO) as measured by change in DLCO
Change in diffusing capacity of the lung for carbon monoxide (DLCO)
Measured: 24 weeks
Efficacy of saracatinib in IPF (exacerbations) as measured in time to first acute exacerbation
Time to the first acute exacerbation
Measured: 24 weeks
Efficacy of saracatinib in quality of life in IPF (SGRQ) as measured by total score on SGRQ questionnaire
Total score on the SGRQ questionnaire. St. George's Respiratory Questionnaire (SGRQ) is a 50-item, self-administered, respiratory-specific questionnaire with items covering three domains: symptoms, activities, impacts. Each domain and a total score range from 0-100, with higher scores connoting greater impairment.
Measured: 24 weeks
Efficacy of saracatinib in quality of life in IPF (L-IPF) as measured by total score on L-IPF questionnaire
Total score on the L-IPF questionnaire. Living with IPF (L-IPF) is an IPF-specific questionnaire whose 43 items cover two modules: symptoms and impacts. A model-based scoring algorithm has been developed via psychometric methods.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2020
- Study start12 Nov 2020
- Primary completion15 Sep 2024
- Study completion30 Jun 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Pulmonary Fibrosis (IPF)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This will be a randomized (8:1 ratio), double blind, parallel design, placebo controlled trial.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 49 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Jewish Health
- Sponsor type
- University, hospital or other
- Collaborators
- Yale University, Icahn School of Medicine at Mount Sinai, AstraZeneca, National Center for Advancing Translational Sciences (NCATS), Baylor University, International Center for Health Outcomes and Innovation Research
- Responsible party
- Sponsor
- Investigators
- Gregory Downey, MD, Principal investigator, National Jewish HealthMaria Padilla, MD, Principal investigator, Icahn School of Medicine at Mount SinaiDanielle Antin-Ozerkis, MD, Principal investigator, Yale UniversitySusan Mathai, MD, Principal investigator, Baylor University Medical Center (BUMC)Annetine Gelijns, PhD, Principal investigator, Data and Clinical Coordinating Center- InCHOIR
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04598919
- Sponsor's protocol ID
- 5UH3TR002445
- Other IDs
- UG3TR002445
- Acronym
- STOP-IPF
- Status last verified
- Nov 2024
- First submitted
- 15 Sep 2020
- Last updated
- 14 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after deidentification.
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of the Src Family Kinase Inhibitor Saracatinib in the Treatment of Idiopathic Pulmonary Fibrosis
- ClinicalTrials.gov ID
- NCT04598919
- Acronym / sponsor ID
- STOP-IPF / 5UH3TR002445
- Conditions
- Idiopathic Pulmonary Fibrosis (IPF)
- Keywords
- Idiopathic Pulmonary Fibrosis, Scarring of the lung, Saracatinib, randomized controlled trial
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 49 participants (estimated)
- Lead sponsor
- National Jewish HealthUniversity, hospital or other
- Collaborators
- Yale University, Icahn School of Medicine at Mount Sinai, AstraZeneca, National Center for Advancing Translational Sciences (NCATS), Baylor University, International Center for Health Outcomes and Innovation Research
- Study start
- 12 Nov 2020 (actual)
- Primary completion
- 15 Sep 2024 (actual)
- Study completion
- 30 Jun 2025 (estimated)
- First posted
- 22 Oct 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.
Scarring of the lung, termed pulmonary fibrosis (PF), is a chronic, progressive, and usually fatal disorder. While two anti-fibrotic drugs have been approved for treating PF of unknown cause (idiopathic pulmonary fibrosis or IPF), neither drug is curative, and nearly 40% of patients stop taking the prescribed drug within a year because of side effects. The study includes the use of saracatinib, an investigational drug originally developed to treat certain types of cancers, in the treatment of IPF in a Phase 1b/2a clinical trial.
The objectives of this study are to: i) evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics, and to explore the efficacy of saracatinib in IPF; ii) identify biomarkers of Src kinase activity and fibrogenesis linked to pulmonary fibrosis; and iii) explore the application of these biomarkers to assess the anti-fibrotic effect of saracatinib in IPF patients
Detailed description
This is a double blind, randomized, placebo-controlled, single-dose, four-site trial. The trial is a biomarker based, integrated Phase 1b/2a clinical trial involving 100 subjects. One group (n=50) will receive placebo, while the other group (n=50) will receive 125 mg of oral saracatinib once daily.
Randomization will be stratified by center. The randomization scheme will be in random blocks of 2 and 4 within each stratum to maintain balance. In the second part of the trial, we will use a simple randomization scheme to achieve the 8:1 randomization across sites. The study is designed to have interim analysis of the drop-out rates when approximately 30% of the randomized patients have achieved the 24-week assessment. Should the drop-out rate be higher than the 20% that is anticipated, a new sample size calculation will be performed to make sure that the power of the study is maintained at 80% .
Duration of follow-up will be 28 weeks including 24 weeks of treatment with saracatinib or placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This will be a randomized (8:1 ratio), double blind, parallel design, placebo controlled trial.
| Arm | Type | Receives |
|---|---|---|
| Saracatinab | Active comparator | Drug: Saracatinabsaracatinib 125 mg once daily by mouth for 24 weeks |
| Placebo | Placebo comparator | Drug: Placebomatching placebo once daily by mouth for 24 weeks |
Who may qualify
- Ages
- 40 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- IPF of any duration, confirmed or diagnosed by ILD center or expert according to Fleischner Guidelines (33). Subjects with a probable or indeterminant CT scan who otherwise meet the Fleischner criteria for IPF are eligible to be included in the study after a multidisciplinary evaluation. A positive Envisia genomic classifier score (34) on a lung biopsy specimen will be considered as strong evidence for a diagnosis of IPF. Subjects with a positive invisia genomic classifier score in conjunction with a probable or indeterminant CT scan are eligible to be included in the study after a multidisciplinary evaluation.
- Women or men >40 years of age at the time of screening
- FVC%>45% of predicted value (GLI-2012)
- Single breath DLCO% ≥ 30 - inclusive of predicted (without bronchodilator and uncorrected for hemoglobin GLI-2017)
- FEV1/FVC>70 (GLI-2012)
Key exclusion criteria (23 listed)
- Requirement for supplemental oxygen > 4 L/min at rest to maintain saturation > 90%
- Active infection at screening or randomization
- Known active or latent hepatitis B or C
- Life expectancy for disease other than IPF < 2.5 years (Investigator assessment)
- Listed for lung transplantation
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 |
|---|---|---|
| Saracatinab125 mg once daily by mouth for 24 weeks | Drug | Saracatinab |
| Placeboonce daily by mouth for 24 weeks | Drug | Placebo |
Primary outcome
Safety of saracatinib in IPF as measured by frequency of adverse events
Time frame: 24 weeks
Safety data will be listed and summarized with patient counts and percentages in each treatment arm
Tolerability of saracatinib in IPF as measured by Severity of adverse events
Time frame: 24 weeks
A listing of all adverse events by patient will be presented. This listing will include patient number, adverse event (actual term and preferred term), event stand and end dates, CTCAE grade, relationship to the study drug/procedure, seriousness and outcome. A listing of SAEs will be produced using the similar format. This is not a scale. It is a data capture tool.
Pharmacokinetics of saracatinib in IPF as measured by serum levels
Time frame: 24 weeks
Serum levels of saracatinib
Pharmacodynamics of saracatinib in IPF as measured by change in serum β-CTX
Time frame: 24 weeks
Change in serum β-CTX as a Src kinase dependent biomarker
Efficacy of saracatinib in IPF as measured by change in FVC
Time frame: 24 weeks
Change in FVC from baseline
5 secondary outcomes
Efficacy of saracatinib in IPF (HRCT)
Time frame: 24 weeks
Efficacy of saracatinib in IPF (DLCO) as measured by change in DLCO
Time frame: 24 weeks
Efficacy of saracatinib in IPF (exacerbations) as measured in time to first acute exacerbation
Time frame: 24 weeks
Efficacy of saracatinib in quality of life in IPF (SGRQ) as measured by total score on SGRQ questionnaire
Time frame: 24 weeks
Efficacy of saracatinib in quality of life in IPF (L-IPF) as measured by total score on L-IPF questionnaire
Time frame: 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2024.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after deidentification.
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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.
Gregory Downey, MD principal investigator
National Jewish Health
Maria Padilla, MD principal investigator
Icahn School of Medicine at Mount Sinai
Danielle Antin-Ozerkis, MD principal investigator
Yale University
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Saracatinab in people with Idiopathic Pulmonary Fibrosis (IPF), run by National Jewish Health at 4 sites in United States.
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 8 inclusion and 23 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: under way, not looking for participants, as of the sponsor's last update on 14 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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 combined 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.
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?
This study is still under way. Results are not available in the source record yet.
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 of saracatinib in IPF as measured by frequency of adverse events (time frame: 24 weeks). The study lists 5 primary outcomes in all. 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 14 Nov 2024.