Ursodeoxycholic Acid for Sarcoidosis

1
Effectiveness
2
Safety
University of Pennsylvania, Philadelphia, PA
Sarcoidosis+1 More
Ursodeoxycholic Acid - Drug
Eligibility
18+
All Sexes
Eligible conditions
Sarcoidosis

Study Summary

This study is evaluating whether a drug may help improve liver function and quality of life for individuals with liver disease.

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Eligible Conditions

  • Sarcoidosis
  • Hepatic Sarcoidosis, Elevated Alkaline Phosphatase

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Ursodeoxycholic Acid will improve 1 primary outcome in patients with Sarcoidosis. Measurement will happen over the course of from baseline.

from baseline
Reduction in alkaline phosphatase and/or gamma glutamyl transferase

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Side Effects for

LUM001 5 mg + UDCA
Abdominal Pain
100%
Diarrhoea
100%
Vomiting
100%
Dizziness
100%
Nausea
100%
Mouth Ulceration
0%
Flatulence
0%
Toothache
0%
Faeces Discoloured
0%
Excoriation
0%
Myalgia
0%
Cough
0%
Upper Respiratory Tract Infection
0%
Headache
0%
Dry Eye
0%
Gingival Pain
0%
Musculoskeletal Chest Pain
0%
Pain
0%
Muscular Weakness
0%
Constipation
0%
Anal Injury
0%
Bronchitis
0%
Chills
0%
Gastrooesophageal Reflux Disease
0%
Urinary Tract Infection
0%
Rash
0%
Muscle Spasms
0%
Pyrexia
0%
Paraesthesia
0%
Abdominal Distension
0%
Asthenia
0%
Sinusitis
0%
Vaginal Discharge
0%
Pharyngeal Erythema
0%
Dry Mouth
0%
Fatigue
0%
Pain In Extremity
0%
Pruritus
0%
Myocardial Infarction
0%
Pancytopenia
0%
Nasal Congestion
0%
Laryngeal Inflammation
0%
Sinus Operation
0%
Back Pain
0%
Oropharyngeal Pain
0%
Gastrointestinal Haemorrhage
0%
Influenza Like Illness
0%
Abdominal Pain Upper
0%
Dyspepsia
0%
This histogram enumerates side effects from a completed 2015 Phase 2 trial (NCT01904058) in the LUM001 5 mg + UDCA ARM group. Side effects include: Abdominal Pain with 100%, Diarrhoea with 100%, Vomiting with 100%, Dizziness with 100%, Nausea with 100%.

Trial Design

2 Treatment Groups

Control
UDCA at Month 6

This trial requires 10 total participants across 2 different treatment groups

This trial involves 2 different treatments. Ursodeoxycholic Acid is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

UDCA at Month 6
Drug
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ursodeoxycholic acid
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from baseline
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly from baseline for reporting.

Closest Location

University of Pennsylvania - Philadelphia, PA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Granulomas on liver biopsy
Hepatomegaly on imaging
Elevated liver-specific alkaline phosphatase
Portal Hypertension (via imaging or endoscopy)
Stable dose of immunosuppressant, if taking (no dose variation for 6 months)
If cirrhotic, absence of hepatocellular carcinoma as indicated by imaging within 6 months of screening

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of sarcoidosis?

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Arthralgias, fevers, enlarged lymph nodes, cough or shortness of breath will be seen in patients with sarcoidosis. A history of the disease with onset of lung function deficit and cough suggestive of granulomatous inflammation may implicate the diagnosis.

Unverified Answer

What causes sarcoidosis?

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Many hypotheses to explain how sarcoidosis arises have been proposed and none have been substantiated. As a result, it is difficult to ascertain the basic mechanisms underlying sarcoidosis. Some researchers suggest aberrant regulation of the immune response is an important mechanism in sarcoidosis. Alternatively, the role of genetics is emerging, suggesting at least part of the variability in sarcoidosis may be due to genetic factors. The mechanism by which genetic factors may produce sarcoidosis involves both genes and environmental factors. Many genes are found to be mutated in sarcoidosis, as well as in other immune diseases. Additionally, many environmental factors seem to have some effect on sarcoidosis, as in many other diseases.

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What are common treatments for sarcoidosis?

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Sarcoidosis is commonly treated with the medications cyclophosphamide or pentostatin along with corticosteroids. A Cochrane Review has shown that all therapies used to treat sarcoidosis have modest effect on outcomes such as disability and mortality, although these drugs can have significant side effects. Surgical treatment of affected organs may also be available in select cases. Vaccination may be given to individuals with pulmonary sarcoidosis to try to prevent the condition or future development. Appropriate diagnosis and early treatment are essential to optimise outcomes, and this is best achieved by a multidisciplinary team in a specialist centre. Evidence-based recommendations have been published by the British Society for Pulmonary Medicine.

Unverified Answer

What is sarcoidosis?

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Sarcoidosis is a chronic multi-systemic autoimmune disease with a wide variety of clinical and pathological manifestations. It is characterised by granulomas in multiple organs and involves several lymph nodes. Sarcoidosis should be considered in patients presenting with pulmonary hypertension and nodular, diffuse or other non-specific abnormalities on radiology.

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Can sarcoidosis be cured?

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A new treatment approach is suggested which is based on a personalized approach, a comprehensive assessment of prognostic factors, and a strong motivation to overcome disease and minimize adverse events.

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How many people get sarcoidosis a year in the United States?

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People with sarcoidosis in the United States are far more likely to be young rather than elderly or middle-aged. Although a peak in the incidence occurs in teenagers, cases begin to decline in adulthood. These trends vary by region rather than by race or ethnicity.

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How does ursodeoxycholic acid work?

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Because of its strong lipid-lowering properties, UDCA is effective in a broad spectrum of different diseases. The exact pharmacologic mechanisms by which this drug acts are not entirely understood, however, it appears UDCA, via the inhibition of cholesterol biosynthesis, directly stimulates cellular cholesterol breakdown, resulting in the depletion of intracellular cholesterol and the reversal of many of the disease manifestations. We believe these data justify early clinical assessment of ursodeoxycholic acid as a potential treatment for sarcoidosis.

Unverified Answer

Does ursodeoxycholic acid improve quality of life for those with sarcoidosis?

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UDC therapy did not appear to affect health-related quality of life for patients with sarcoidosis. It is possible however that a more homogeneous patient group is needed in order to determine the true impact of UDC on HRQOL.

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Has ursodeoxycholic acid proven to be more effective than a placebo?

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UDCA was no more effective than a placebo in the treatment of patients with active and untreated primary biliary cirrhosis. This finding has implications for treatment of advanced liver disease.

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Have there been any new discoveries for treating sarcoidosis?

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Until now, a few effective drugs have been found; these have been used in combination with or separate from standard of care medications for sarcoidosis. These are, most notably: corticosteroids, methotrexate, hydroxychloroquine, and other cyclooxygenase-2 (COX-2) inhibitors. The most effective combination of therapeutic agents for sarcoidosis is not certain. There are no new drugs that are yet in development to be more effective than existing drugs like azathioprine and biologics, and the only medications to have shown convincing efficacy are the same ones already known.

Unverified Answer

What are the common side effects of ursodeoxycholic acid?

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UDCA has some side effects. Most often these consist of abdominal pain, bloating, itchiness, nausea, diarrhea and headache because some patients are allergic to UDCA. If a patient is allergic to any drug it is better to have the specific name and dosage that he/she uses. I used to get many of our patients to start on the drug UDC, because there was such a shortage of physicians who could help them...

Unverified Answer
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