FatiguePhase 1PBCNCT00943176
Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis (PBC)
A Phase 1 study testing Modafinil in people with Fatigue and Primary Biliary Cirrhosis, run by Mayo Clinic at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the use of modafinil in the treatment of fatigue in patients with Primary Biliary Cirrhosis. The general aim of the study is to identify a safe and effective therapy for fatigue in patients with primary biliary cirrhosis. Read more about this study
- Who is it for?
- Ages 21 to 75 years
- Conditions the study is for
- Fatigue, Primary Biliary Cirrhosis
- What is being tested?
- Modafinil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to Apr 2011
- 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 21 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Primary biliary cirrhosis will be defined as present when 2 of 3 of the following criteria are met:
- Chronic cholestatic liver disease for greater than 6 months with alkaline phosphatase levels greater than 1.5 times the upper limit of normal prior to UDCA treatment.
- Positive AMA titer greater or equal to 1:40 or AMA greater than 0.1U. Liver histology in the past with features consistent with or diagnostic of PBC.
- A previous ultrasound, computed tomography (CT), or cholangiography of the biliary tree excludes biliary obstruction.
- Verbal report of fatigue for greater than 6 months.
Who cannot join
- Patients with other serious coexistent conditions such as pre-existing advanced malignancy or severe cardiopulmonary disease which would be expected to limit their expectancy to less than three years.
- Findings highly suggestive of liver disease of other etiology such as chronic alcoholic liver disease, chronic hepatitis B or C infection, hemochromatosis, Wilson's disease, 1-antitrypsin deficiency, non-alcoholic steatohepatitis or sclerosing cholangitis.
- Treatment of underlying PBC has been modified in the preceding six months.
- Anticipated need for transplantation in one year (Mayo survival model <80% one-year survival without transplant) or MELD above 15.
- Recurrent variceal bleeding, presence of diuretic-resistant ascites, or spontaneous encephalopathy.
- Active drug or alcohol use.
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- History of drug and/or stimulant (e.g. methylphenidate, amphetamine, or cocaine) abuse.
- Serum bilirubin >4 mg/dl.
- Serum creatinine over 1.4 mg/dl.
- Pregnancy.
- Breast-feeding.
- Inability or unwillingness to practice contraceptive measures for the prevention of pregnancy if appropriate.
- Other fatigue related diagnoses such as anemia, thyroid disease, renal failure, use of beta-blockers and untreated depression.
- Known hypersensitivity to modafinil.
- Uncontrolled hypertension.
- Patients with hypertension and left ventricular hypertrophy documented on ECG in the last 2 years.
- Recent (<6 months) history of myocardial infarction or unstable angina.
- Patients with history of psychosis.
- Patients receiving cyclosporine, warfarin, tricyclic agents, carbamazepine, phenobarbital, rifampin, ketoconazole or itraconazole in the last 3 months.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis
The purpose of this study is to evaluate the use of modafinil in the treatment of fatigue in patients with Primary Biliary Cirrhosis.
The general aim of the study is to identify a safe and effective therapy for fatigue in patients with primary biliary cirrhosis.
Read the detailed description
The general aim of the study is to identify a safe and effective therapy for fatigue in patients with primary biliary cirrhosis. Specific aims include: 1) to determine the safety profile of modafinil in patients with primary biliary cirrhosis; 2) to evaluate the beneficial effects of modafinil on patients with primary biliary cirrhosis and fatigue as documented by a well-validated questionnaire (Fisk Fatigue Severity Score) to be applied at the beginning and end of the study period; and 3) to compare the performance of three questionnaires (Fisk Fatigue Severity Score, Fatigue Severity Scale and the fatigue domain of the PBC-40) as instruments to determine the benefits of a therapeutic intervention on fatigue by establishing the minimally important clinical difference of these measures of fatigue.
- Conditions
- Fatigue, Primary Biliary Cirrhosis
- Being tested
- Modafinil
- Keywords
- Fatigue
- Medical subject headings
- Fatigue, Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ModafinilDrug
Also called Provigil
100 mg tablets of modafinil or matched placebo will be given for a total of 12 weeks. Patients will be instructed to start one capsule once daily (modafinil or placebo), and dose will be titrated according to tolerability and response.
| Study group | Role | What they receive |
|---|---|---|
| Sugar Pill | Placebo comparator | Not described Receives: Drug: Modafinil |
| Modafinil | Experimental | Not described Receives: Drug: Modafinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
The main endpoint will be the change in fatigue severity (quantified by the FFSS) following 12 weeks of treatment, compared to baseline values.
Measured: after 12 weeks of treatment
Secondary outcomes (1)
Secondary outcome includes frequency of adverse events, change in alkaline phosphatase, AST, total bilirubin and albumin levels after 12 weeks of therapy compared to baseline values, change in fatigue severity compared to baseline.
Measured: after 12 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2009 (estimated)
- Study startJun 2009
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for FatigueStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- American College of Gastroenterology
- Investigators
- Keith D Lindor, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00943176
- Sponsor's protocol ID
- 08-007692
- Acronym
- PBC
- Status last verified
- Jan 2012
- First submitted
- 17 Jul 2009
- Last updated
- 18 Jan 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Silveira MG, Gossard AA, Stahler AC, Jorgensen RA, Petz JL, Ali AH, Lindor KD. A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety: Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis. Am J Ther. 2017 Mar/Apr;24(2):e167-e176. doi: 10.1097/MJT.0000000000000387.
PubMed 27148676
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis
- ClinicalTrials.gov ID
- NCT00943176
- Acronym / sponsor ID
- PBC / 08-007692
- Conditions
- Fatigue, Primary Biliary Cirrhosis
- Keywords
- Fatigue
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- American College of Gastroenterology
- Study start
- Jun 2009
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 22 Jul 2009 (estimated)
- 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 purpose of this study is to evaluate the use of modafinil in the treatment of fatigue in patients with Primary Biliary Cirrhosis.
The general aim of the study is to identify a safe and effective therapy for fatigue in patients with primary biliary cirrhosis.
Detailed description
The general aim of the study is to identify a safe and effective therapy for fatigue in patients with primary biliary cirrhosis. Specific aims include: 1) to determine the safety profile of modafinil in patients with primary biliary cirrhosis; 2) to evaluate the beneficial effects of modafinil on patients with primary biliary cirrhosis and fatigue as documented by a well-validated questionnaire (Fisk Fatigue Severity Score) to be applied at the beginning and end of the study period; and 3) to compare the performance of three questionnaires (Fisk Fatigue Severity Score, Fatigue Severity Scale and the fatigue domain of the PBC-40) as instruments to determine the benefits of a therapeutic intervention on fatigue by establishing the minimally important clinical difference of these measures of fatigue.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sugar Pill | Placebo comparator | Drug: Modafinil |
| Modafinil | Experimental | Drug: Modafinil |
Who may qualify
- Ages
- 21 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Primary biliary cirrhosis will be defined as present when 2 of 3 of the following criteria are met:
- A previous ultrasound, computed tomography (CT), or cholangiography of the biliary tree excludes biliary obstruction.
- Verbal report of fatigue for greater than 6 months.
Key exclusion criteria (19 listed)
- Patients with other serious coexistent conditions such as pre-existing advanced malignancy or severe cardiopulmonary disease which would be expected to limit their expectancy to less than three years.
- Findings highly suggestive of liver disease of other etiology such as chronic alcoholic liver disease, chronic hepatitis B or C infection, hemochromatosis, Wilson's disease, 1-antitrypsin deficiency, non-alcoholic steatohepatitis or sclerosing cholangitis.
- Treatment of underlying PBC has been modified in the preceding six months.
- Anticipated need for transplantation in one year (Mayo survival model <80% one-year survival without transplant) or MELD above 15.
- Recurrent variceal bleeding, presence of diuretic-resistant ascites, or spontaneous encephalopathy.
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 |
|---|---|---|
| ModafinilAlso: Provigil100 mg tablets of modafinil or matched placebo will be given for a total of 12 weeks. Patients will be instructed to start one capsule once daily (modafinil or placebo), and dose will be titrated according to tolerability and response. | Drug | Modafinil, Sugar Pill |
Primary outcome
The main endpoint will be the change in fatigue severity (quantified by the FFSS) following 12 weeks of treatment, compared to baseline values.
Time frame: after 12 weeks of treatment
1 secondary outcome
Secondary outcome includes frequency of adverse events, change in alkaline phosphatase, AST, total bilirubin and albumin levels after 12 weeks of therapy compared to baseline values, change in fatigue severity compared to baseline.
Time frame: after 12 weeks of treatment
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 18 Jan 2012.
- Derived from this study: PubMed 27148676Silveira MG, Gossard AA, Stahler AC, Jorgensen RA, Petz JL, Ali AH, Lindor KD. A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety: Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis. Am J Ther. 2017 Mar/Apr;24(2):e167-e176. doi: 10.1097/MJT.0000000000000387.
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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.
Keith D Lindor, M.D. principal investigator
Mayo Clinic
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 Modafinil in people with Fatigue and Primary Biliary Cirrhosis, run by Mayo Clinic 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 5 inclusion and 19 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 18 Jan 2012. 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. 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?
The main endpoint will be the change in fatigue severity (quantified by the FFSS) following 12 weeks of treatment, compared to baseline values. (time frame: after 12 weeks of treatment). 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 NCT00943176, which also lists 1 linked publication.
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 18 Jan 2012.