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SclerodermaPhase 1NCT02426229

Safety & Suitability of Dabigatran to Inhibit Thrombin in Scleroderma

A Phase 1 study testing dabigatran etexilate in people with Scleroderma and Interstitial Lung Disease, run by Medical University of South Carolina at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study evaluates if dabigatran etexilate is safe for use in patients with Scleroderma and Interstitial Lung Disease. All patients will receive 75mg of dabigatran etexilate twice a day for 6 months. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Scleroderma, Interstitial Lung Disease
What is being tested?
dabigatran etexilate
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteUnited States
Study dates
Feb 2016to 21 Jun 2018
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥ 18 and ≤ 70 years
  • All patients must fulfill the ACR/EULAR criteria for SSc. Patients may have limited (cutaneous thickening distal, but not proximal to elbows and knees, with or without facial involvement) or diffuse (cutaneous thickening proximal to elbows and knees, often involving the chest or abdomen) cutaneous SSc, or systemic sclerosis sine scleroderma
  • SSc for less than 7 years, with onset defined as the date of the first non-Raynaud phenomenon manifestation.
  • All patients must have interstitial lung disease defined by any ground glass on HRCT and >20% involvement of HRCT by pulmonary fibrosis and/or FVC <70% predicted

Who cannot join

  • Inability to sign consent
  • Currently enrolled in another clinical trial
  • FVC < 40% predicted and/or DLCO (corrected for hemoglobin) < 30% of predicted (suggesting severe probably irreparable disease)
  • Other serious concomitant medical illnesses (e.g., cancer) limiting life expectancy to <1 year at time of enrollment
  • FEV1/FVC ratio < 65% (suggesting obstructive disease)
  • Clinically significant pulmonary hypertension requiring treatment, based on the clinician's judgment.
Show all 32Show fewer
  • Smoking of cigars, pipes or cigarettes within 3 months prior to and during enrollment
  • Clinically significant abnormalities on chest x-ray other than interstitial lung disease (e.g., lung mass, evidence of active pulmonary infection, emphysema)
  • Use of prednisone (or equivalent) in doses > 10 mg daily within 3 months prior to and during enrollment
  • Use of colchicine, D-penicillamine, cyclophosphamide, mycophenolate mofetil, azathioprine, endothelin receptor antagonists, phosphodiesterase type-5 inhibitors, prostanoids, tyrosine kinase inhibitors, sirolimus, rituximab, perfinidone or other "disease modifying medications" within 3 months prior to and during enrollment
  • Pregnancy or lack of use of birth control method in women of childbearing age or lactating
  • Liver disease or increased baseline liver enzyme levels (ALT >3 x upper limit of normal)
  • Use of CYP450 inhibitors/inducers
  • Hemoglobin < 10g/L
  • If of child bearing potential, unwillingness to employ a reliable means of contraception (condom, abstinence, IUD, tubal ligation, vasectomy)
  • Active infection
  • Creatinine clearance <30 ml/min
  • Post transplantation
  • Active medical and psychiatric conditions which the investigator may consider would interfere with the subject's treatment, assessment, or compliance with the protocol
  • Anticoagulation-related exclusions include:
  • Current anticoagulation therapy with warfarin
  • Increased risk of bleeding (e.g., uncorrectable inherited or acquired bleeding disorder)
  • Platelet count <100,000/cmm or hematocrit <30% or > 55%
  • History of severe gastrointestinal bleeding within 6 months of screening
  • Known gastric antral vascular ectasia (GAVE) or gastric/intestinal arterial-venous malformations (AVMs)
  • History of CVA within 6 months of screening
  • History of risks of falls as judged by the PI
  • Surgery or major trauma within the past 30 days
  • Any condition that, in the determination of the PI, is likely to require anticoagulation therapy during the study
  • Clopidogrel, prasugrel or other anti-platelet therapy within 6 months of screening
  • Aspirin therapy >325 mg daily
  • Therapy with other thrombin inhibitors

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

Other trials in United States

About this study

This study evaluates if dabigatran etexilate is safe for use in patients with Scleroderma and Interstitial Lung Disease. All patients will receive 75mg of dabigatran etexilate twice a day for 6 months.

Read the detailed description

Skin and pulmonary fibrosis result in substantial morbidity in scleroderma (SSc). Furthermore, interstitial lung disease (ILD) culminating in pulmonary fibrosis is a major cause of death among scleroderma patients. Studies implicate the coagulation system, most notably the serine protease thrombin, in the pathogenesis of SSc-ILD. Thrombin can transform normal lung fibroblasts to a scleroderma fibroblast phenotype. Dabigatran etexilate is a selective thrombin inhibitor which is FDA-approved for the prevention of thromboembolic complications in patients with atrial fibrillation. Dabigatran etexilate needs to be studied as a potential anti-fibrotic agent for the treatment of SSc-ILD. This study is designed to see if dabigatran etexilate is safe for use in patients with scleroderma. If so, the long term goal of this study is to determine whether or not the fundamental results will translate to a potential clinical intervention for SSc-ILD which can be tested in a future randomized control trial.

Being tested
dabigatran etexilate
Keywords
SSc, systemic sclerosis, cutaneous systemic sclerosis
Medical subject headings
Scleroderma, Diffuse, Lung Diseases, Interstitial, Scleroderma, Systemic

What participants receive

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

  • dabigatran etexilateDrug

    Also called Dabigatran, Pradaxa

    dabigatran etexilate 75mg orally twice a day for 6 months

Study groups in NCT02426229
Study groupRoleWhat they receive
dabigatran 75mgExperimentaldabigatran etexilate 75mg orally twice daily for 6 months
Receives: Drug: dabigatran etexilate

What is being measured

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

  1. Composite: Safety of dabigatran patients with scleroderma interstitial lung disease. (complete blood counts, comprehensive metabolic profile, and coagulation studies).

    Subjects taking dabigatran will undergo monthly complete blood counts (white blood cell count, hemoglobin, hematocrit, platelet), comprehensive metabolic profile (sodium, potassium, chloride, bicarbonate, BUN, creatinine, glucose, total bilirubin, AST, ALT, alkaline phosphatase, protein and albumin), and coagulation studies (prothrombin time, partial thromboplastin time and thrombin time). Women of child-bearing age will be required to have a urine pregnancy test monthly while receiving dabigatran.

    Measured: Up to 6 months

Secondary outcomes (1)

  1. Composite: Preliminary estimate of efficacy of dabigatran in scleroderma. (skin score and dermal fibroblast biology)

    We will also include investigations of scleroderma skin (skin score and dermal fibroblast biology) together with studies of scleroderma lung fibroblasts, to obtain preliminary estimates of the effectiveness of dabigatran as a potential disease modifying drug for patients with SSc-ILD.

    Measured: Up to 6 months

Timeline

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

  1. First posted24 Apr 2015 (estimated)
  2. Study startFeb 2016
  3. Primary completionJun 2018
  4. Study completion21 Jun 2018

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02426229
Sponsor's protocol ID
1R21AR065089-01A1
Other IDs
1R21AR065089-01A1
Status last verified
Jul 2018
First submitted
4 Mar 2015
Last updated
13 Jul 2018
Results posted
No
Data monitoring committee
Yes

Publications

  • Bogatkevich GS, Ludwicka-Bradley A, Silver RM. Dabigatran, a direct thrombin inhibitor, demonstrates antifibrotic effects on lung fibroblasts. Arthritis Rheum. 2009 Nov;60(11):3455-64. doi: 10.1002/art.24935.

    PubMed 19877031

  • Bogatkevich GS, Huggins TJ, Ismail AA, Atanelishvili I, Silver RM. Anti-fibrotic effects of thrombin inhibition in systemic sclerosis-associated interstitial lung disease: Proof of concept. J Scleroderma Relat Disord. 2025 Feb 24;10(3):359-367. doi: 10.1177/23971983241311625. eCollection 2025 Oct.

    PubMed 40013233

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing dabigatran etexilate in people with Scleroderma and Interstitial Lung Disease, run by Medical University of South Carolina at 1 site 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 4 inclusion and 32 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 13 Jul 2018. 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 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. 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?

Composite: Safety of dabigatran patients with scleroderma interstitial lung disease. (complete blood counts, comprehensive metabolic profile, and coagulation studies). (time frame: Up to 6 months). 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 NCT02426229, which also lists 2 linked publications.

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 13 Jul 2018.

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