Methotrexate for Sarcoidosis

2
Effectiveness
3
Safety
Yale-New Haven Hospital, New Haven, CT
Sarcoidosis+1 More
Methotrexate - Drug
Eligibility
18+
All Sexes
Eligible conditions
Sarcoidosis

Study Summary

Cardiac Sarcoidosis Randomized Trial

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Eligible Conditions

  • Sarcoidosis
  • Cardiac Sarcoidosis

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Methotrexate will improve 1 primary outcome and 19 secondary outcomes in patients with Sarcoidosis. Measurement will happen over the course of 6 month scan.

6 month scan
FDG-PET and myocardial perfusion
6 months
BMI
Blood pressure
CMR Endpoints
Cardiovascular hospitalizations
Complete heart block
Disease Specific Quality of Life (KSQ and SAT)
Generic Quality of Life (SF 36)
Glucocorticoid Toxicity Index
HbA1C
Highly sensitive Troponin I levels and BNP levels
LVEF and RVEF assessed on echocardiogram
Medication compliance
Medication related adverse events
Modified Cleveland Clinic Glucocorticoid Toxicity Score
Mortality
Patient reported symptoms related to medication
Summed perfusion rest score (SPRS) on FDG-PET scan
T-score on bone density scan
Ventricular arrhythmia burden

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

3 Treatment Groups

Prednisone
Methotrexate

This trial requires 194 total participants across 3 different treatment groups

This trial involves 3 different treatments. Methotrexate 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 3 and have had some early promising results.

Methotrexate[Dose everywhere except Japan] Methotrexate 15-20 mg orally, sc, or IM once a week for 6 months + Prednisone 20 mg po daily for one month then 10 mg po daily for one month then 5 mg po daily for one month and then stop. Also Folic Acid 2 mg po daily for 6 months. [Dose in Japan] Methotrexate 5-20mg mg orally, sc, or IM once a week for 6 months+ Prednisone or Prednisolone 20mg OD for 1 month then 10mg OD for 1 month then 5 mg OD one month. Also Folic Acid 2 mg po daily for 6 months.
PrednisonePrednisone 0.5 mg kg/day for 6 months (max dose 30 mg)
Prednisone (or Prednisolone)
Drug
[Dose everywhere except Japan] Prednisone 0.5 mg kg/day for 6 months (max dose 30 mg) [Dose in Japan] Prednisone or prednisolone 0.5 mg/kg po (max 30mg) for one month then reduce by 5 mg per month for five months
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisolone
FDA approved
Methotrexate
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 6 months for reporting.

Closest Location

Yale-New Haven Hospital - New Haven, CT

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
An advanced conduction system disease is a disorder of the heart which affects the conduction of electrical signals through the heart show original
significant sinus node dysfunction is when the average heart rate is less than 40 beats per minute when awake, and/or there is a sustained atrial arrhythmia. show original
There is no alternative explanation for the clinical features of the disease. show original
The text suggests that FDG-PET uptake was suggestive of active CS within two months of enrollment and this was confirmed by a PET core lab read. show original
Having a right ventricular dysfunction means that your right ventricle is not working well show original
CONDITIONS ARE MET The statement is true if one or both of the following conditions are met. show original
A ventricular arrhythmia is a problem with the heart's rhythm show original
left ventricular dysfunction (LVEF < 50%)
A positive biopsy for Sarcoidosis, either through an extended myocardial biopsy (EMB) or an extra-cardiac biopsy, confirms the presence of the disease. show original

Patient Q&A Section

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

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

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A total of 5,850 cases per year are diagnosed in the US. Half of these cases are diagnosed between the age of 30 and 60. People who are diagnosed with sarcoidosis are more likely to have more than one family member with the disease.\n

Unverified Answer

What are the signs of sarcoidosis?

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Sarcoidosis is a complex disease, often affecting multiple organ systems. As the disease presents with multisystem involvement, signs and symptoms are often less clear-cut, and thus frequently unrecognized. Patients usually present with unexplained pulmonary involvement with or without involvement of the skin, lymph nodes, liver, or kidneys. Scleroderma-like skin changes are common and may be the first presenting sign. The most common skin lesions occur on the face, and include erythema nodosum and facial palsy. Patients often have fever and tender lymph nodes. The diagnosis can only be definitively established with skin biopsy. Patients usually have positive serology. Sarcoidosis is a diagnosis of exclusion.

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What are common treatments for sarcoidosis?

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Sarcoidosis can be divided into two subtypes: pulmonary sarcoidosis due to sarcoid crystals and extrapulmonary sarcoidosis (EPS) due to an unknown cause. The most common treatment is inhaled steroids in combination with steroids or, in cases of severe lung involvement, lung transplantation, which may be curative. Most patients have only one or two pulmonary infiltrates and thus respond to steroids alone. Sarcoid calcifications, if present, may respond to treatment with calcium blockers or surgery, which may be curative. EPS responds readily to steroids and calcification-inhibiting agents.

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What causes sarcoidosis?

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It is thought that exposure to certain fungi causes sarcoidosis, but whether this is the sole or a contributory cause is unclear. The use of inhaled corticosteroids or anticytokines (e.g. interferon) in sarcoidosis may increase the risk of chronic lung disease.

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Can sarcoidosis be cured?

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The standard of care is to treat patients with sarcoidosis until the disease becomes quiescent, and discontinuation of therapy is discouraged because of its possible risk of recurrence. Patients who have not demonstrated disease activity after 18 months of therapy are deemed to be quiescent. The success rate of spontaneous resolution in sarcoidosis is between 15-50%. Many patients remain asymptomatic and require no treatment. Patients who experience disease activity after spontaneous resolution are treated with corticosteroids to minimize or eliminate residual disease or to prevent recurrence. Treatment lasts as long as required, as the condition is often chronic and relapses may occur after a prolonged period of drug treatment.

Unverified Answer

What is sarcoidosis?

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Sarcoidosis is a chronic and systemic disease that commonly involves the lungs but also other organs such as the skin, lymph nodes, liver as well as the central nervous system. It has an important autoimmune component. The disease occurs about twice as often in women as in men but in both sexes, it causes fatigue and chronic cough and may cause painless, enlarging enlargements of the lymph nodes. It is commonly diagnosed in patients with tuberculosis who do not respond to or are resistant to anti-tuberculosis treatment. In some countries, sarcoidosis has a higher incidence in immigrants from certain Middle Eastern, South Asian and north African countries.

Unverified Answer

Have there been other clinical trials involving methotrexate?

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There has been limited research concerning the use of methotrexate. The evidence from the studies that have been carried out to date suggests that more research is needed before methotrexate can be recommended as the initial therapeutic agent.

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What is the primary cause of sarcoidosis?

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A wide range of genetic and environmental factors likely combine to produce sarcoidosis. Genetic, epigenetic interactions and environmental factors, including occupational and hormonal exposures, likely contribute to sarcoidosis predisposition. Specific risk factors for certain sarcoids may be identified by genetic linkage studies or genome-wide association studies.

Unverified Answer

Has methotrexate proven to be more effective than a placebo?

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Use of methotrexate was found to be significantly more effective than the placebo in the treatment of lung sarcoidosis, in terms of lung function improvement, radiological appearance of the lung and quality of life.

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What are the latest developments in methotrexate for therapeutic use?

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Recent findings support the results obtained with 5-azacitidine, indicating that this agent may be the first choice of methotrexate for lepromatous leprosy-associated leprosy tuberculoid leprosy syndrome and also as a salvage therapy for refractory cases.

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Does sarcoidosis run in families?

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We have not been able to demonstrate evidence of an etiological contribution of genetic factors to the incidence of sarcoidosis, which suggests that sarcoidosis is acquired, possibly through an environmental trigger.

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Does methotrexate improve quality of life for those with sarcoidosis?

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Treatment with MTX in addition to conventional corticosteroids seems to be an effective and safe treatment option for those with sarcoidosis whose disease responds to conventional therapies with corticosteroids alone. MTX-induced improvements in quality of life and respiratory function were sustained during long-term treatment, and in some cases, these improvements continued even at the completion of the long-term trial. Results from a recent paper indicate that MTX therapy should be explored as a first-line treatment for sarcoidosis. Additional trials are needed, but if confirmed in larger patient cohorts, this therapy may become a mainstay of sarcoidosis treatment.

Unverified Answer
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