Myasthenia GravisEarly Phase 1NCT01555580
A Pilot Study of Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Generalized Myasthenia Gravis
A Early Phase 1 study testing Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in people with Myasthenia Gravis, run by Muscular Dystrophy Association at 1 site in United States.
Trial summary
The purpose of this study is to determine whether the drug Leukine (GM-CFS) is safe and tolerated by patients with autoimmune myasthenia gravis (MG). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2012to Dec 2013
- Phase
- Early 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be between 18 and 80 years of age
- Established diagnosis of myasthenia based on: the presence of fatigable weakness of ocular, oropharyngeal, and/or limb muscles AND the presence of abnormal acetylcholine receptor binding antibodies ≥ 0.4 nmol/l.
- Patients of childbearing potential must agree to use a medically acceptable form of contraception defined by consistent use of oral contraceptive medications or history of tubal ligation or men who are in sexual relationship with such women during and for at least 8 weeks following completion of the study.
- Patient or designee must have the ability to self-inject investigational product
- If thymectomized, the procedure must have been performed at least one year prior to screening.
- Dose of current anticholinesterase drugs must be constant for 2 weeks prior to screening.
- If taking prednisone, dose must be stable for ≥4 weeks prior to screening.
Who cannot join
- exclusively ocular MG (MGFA Class I)
- severe respiratory and/ or swallowing muscle weakness (MGFA Class Vb or V)
- presence of thymoma
- Must not have received plasm exchange or IVIG within 4 weeks of screening
- Must not have received immuno-modulating agents within the 4 weeks of screening, including Azathioprine (Imuran), Cyclosporine (Sandimmune, Neoral), Mycophenolate mofetil (CellCept), GM-CSF (Filgrastim; Neupogen; pegfilgrastim, sargramostim), or any other chronic immunosuppressive agent
- History of tuberculosis or evidence of latent tuberculosis (positive PPD skin test or a chest X-ray with evidence of tuberculosis)
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- vital capacity of less than 1.2 liters or on supplemental oxygen therapy.
- severe comorbidities including lung disease, stroke, congestive heart failure of any severity, myocardial infarction, EKG abnormalities, uncontrolled hypertension - (sitting systolic BP <80 or > 160 mm Hg or diastolic BP > 100 mm Hg, unstable angina pectoris, hepatic or renal disease, insulin-dependent diabetes mellitus, history of cancer (other than in-situ cervical cancer or resected, cutaneous basal cell or squamous cell carcinoma), open cutaneous ulcers, known hepatitis B surface antigen (HbsAg) or hepatitis C virus (HCV) positive, or any other concurrent medical condition, which would make it unsafe for subjects to participate in the trial or interfere with the interpretation of the results.
- Laboratories values which, at the time of the screening visit or at any time during the study that in the opinion of the Investigator would preclude participation in the study including: serum creatinine > 2.5 mg/dL, serum potassium < 3.5 mmol/L or > 5.5 mmol/L, serum aspartate transaminase (AST), alanine transaminase (ALT), or alkaline phosphatase (ALP)> 3 times the upper limit of normal, platelet count < 100,000/mm3, WBC count < 3,000 cells/mm3, Hemoglobin, hematocrit, or red blood cell count outside 30% of the upper or lower limits of normal
- Receipt of a live vaccine within 3 months of screening
- participation in another investigational drug study within 90 days of screening.
- known hypersensitivity to GM-CSF or any of its components
- Known HIV-positive status or known history of any other immuno-suppressing disease.
- Any mycobacterial disease.
- Active severe infections within 4 weeks before screening visit, or between the screening and baseline visits.
- Untreated Lyme disease.
- History of TB or TB exposure, chronic hepatitis B or hepatitis C, SLE, history of multiple sclerosis, transverse myelitis, optic neuritis or epilepsy.
- History of recent alcohol or substance abuse (< 1 year)
- Pregnant or lactating females
- History of non-compliance with other therapies
- abnormal mental status sufficient to exclude informed consent
- History of any opportunistic infection - to include but not limited to Pneumocystis carinii, aspergillosis, histoplasmosis, or atypical mycobacterium
- History of Sickle cell disease.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Illinois at Chicago, Department of Neurology
Chicago, Illinois, 60612
Matthew N Meriggioli, MD, 312-996-4780, mmerig@uic.edu
Margaret O'Connor, RN, 312-413-8605, moconn@uic.edu
About this study
Official title: A Pilot Study of Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Symptomatic Autoimmune Generalized Myasthenia Gravis
The purpose of this study is to determine whether the drug Leukine (GM-CFS) is safe and tolerated by patients with autoimmune myasthenia gravis (MG).
Read the detailed description
Twelve patients aged 18-80 with symptomatic generalized autoimmune MG that are not being treated with medication that suppresses their immune system, other than prednisone, will enter the study at UIC over a two year period. The study will involve a screening visit and visits at baseline and at days 5, 15, 30, 45, 60, 90, and 120. The study drug, Leukine (GM-CFS), is given by injection. Subjects will give themselves one dose of GM-CSF every day for 10 days. Study visits will include muscle testing, immunologic studies and quality-of-life studies.
- Conditions
- Myasthenia Gravis
- Being tested
- Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)
- Keywords
- myasthenia gravis, autoimmune, GM-CSF
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)Drug
Also called LEUKINE® (sargramostim)
Participants will receive one dose of GM-CSF (5 µg/kg) by subcutaneous injection for ten (10) consecutive days. The first dose of GM-CSF will be administered by the subject or caregiver under the observation and direction of the study staff during the baseline visit. The subject or caregiver will administer subsequent injections at home.
| Study group | Role | What they receive |
|---|---|---|
| GM-CSF | Experimental | Not described Receives: Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of GM-CSF in patients with myasthenia gravis
The number of subjects experiencing a Class II-V adverse event within 120 days of the start of treatment that is probably or definitely related to the study medication
Measured: 150 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 30 days post-treatment
Measured: 30 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 60 days post-treatment
Measured: 60 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 120 days post-treatment
Measured: 120 days
Secondary outcomes (14)
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at 60 days
Measured: 60 days
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 120
Measured: 120 days
Change from baseline in the Myasthenia Gravis Composite score at day 60
Measured: 60 days
Change from baseline in the Myasthenia Gravis Composite score at day 120
Measured: 120 days
Change from baseline in Manual Muscle Testing (MMT) score at day 60
Measured: 60 days
Change from baseline in Manual Muscle Testing (MMT) score at day 120
Measured: 120 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 60
Measured: 60 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 120
Measured: 120 days
Change from baseline in Quality of Life Assessment (SF-36) at day 60
Measured: 60 days
Change from baseline in Quality of Life Assessment (SF-36) at day 120
Measured: 120 days
Change from baseline in acetylcholine receptor antibody titre level at day 60
Measured: 60 days
Change from baseline in acetylcholine receptor antibody titre level at day 120
Measured: 120 days
Change from baseline in prednisone dose at day 60
Measured: 60 days
Change from baseline in prednisone dose at day 120 Days
Measured: 120 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2012 (estimated)
- Study startApr 2012
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Muscular Dystrophy Association
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Matthew N. Meriggioli, MD, University of Illinois Chicago
- Investigators
- Matthew N Meriggioli, MD, Principal investigator, University of Illinois at Chicago, 912 S. Wood St., Rm 855-N, M/C 796, Chicago IL 60612
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01555580
- Sponsor's protocol ID
- MDA 2011 MG GM-CSF
- Status last verified
- Mar 2012
- First submitted
- 13 Mar 2012
- Last updated
- 15 Mar 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Symptomatic Autoimmune Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT01555580
- Acronym / sponsor ID
- MDA 2011 MG GM-CSF
- Conditions
- Myasthenia Gravis
- Keywords
- myasthenia gravis, autoimmune, GM-CSF
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Muscular Dystrophy AssociationUniversity, hospital or other
- Study start
- Apr 2012
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 15 Mar 2012 (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 determine whether the drug Leukine (GM-CFS) is safe and tolerated by patients with autoimmune myasthenia gravis (MG).
Detailed description
Twelve patients aged 18-80 with symptomatic generalized autoimmune MG that are not being treated with medication that suppresses their immune system, other than prednisone, will enter the study at UIC over a two year period. The study will involve a screening visit and visits at baseline and at days 5, 15, 30, 45, 60, 90, and 120. The study drug, Leukine (GM-CFS), is given by injection. Subjects will give themselves one dose of GM-CSF every day for 10 days. Study visits will include muscle testing, immunologic studies and quality-of-life studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GM-CSF | Experimental | Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Must be between 18 and 80 years of age
- Established diagnosis of myasthenia based on: the presence of fatigable weakness of ocular, oropharyngeal, and/or limb muscles AND the presence of abnormal acetylcholine receptor binding antibodies ≥ 0.4 nmol/l.
- Patients of childbearing potential must agree to use a medically acceptable form of contraception defined by consistent use of oral contraceptive medications or history of tubal ligation or men who are in sexual relationship with such women during and for at least 8 weeks following completion of the study.
- Patient or designee must have the ability to self-inject investigational product
- If thymectomized, the procedure must have been performed at least one year prior to screening.
Key exclusion criteria (23 listed)
- exclusively ocular MG (MGFA Class I)
- severe respiratory and/ or swallowing muscle weakness (MGFA Class Vb or V)
- presence of thymoma
- Must not have received plasm exchange or IVIG within 4 weeks of screening
- Must not have received immuno-modulating agents within the 4 weeks of screening, including Azathioprine (Imuran), Cyclosporine (Sandimmune, Neoral), Mycophenolate mofetil (CellCept), GM-CSF (Filgrastim; Neupogen; pegfilgrastim, sargramostim), or any other chronic immunosuppressive agent
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 |
|---|---|---|
| Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)Also: LEUKINE® (sargramostim)Participants will receive one dose of GM-CSF (5 µg/kg) by subcutaneous injection for ten (10) consecutive days. The first dose of GM-CSF will be administered by the subject or caregiver under the observation and direction of the study staff during the baseline visit. The subject or caregiver will administer subsequent injections at home. | Drug | GM-CSF |
Primary outcome
Safety of GM-CSF in patients with myasthenia gravis
Time frame: 150 days
The number of subjects experiencing a Class II-V adverse event within 120 days of the start of treatment that is probably or definitely related to the study medication
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 30 days post-treatment
Time frame: 30 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 60 days post-treatment
Time frame: 60 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 120 days post-treatment
Time frame: 120 days
14 secondary outcomes
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at 60 days
Time frame: 60 days
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 120
Time frame: 120 days
Change from baseline in the Myasthenia Gravis Composite score at day 60
Time frame: 60 days
Change from baseline in the Myasthenia Gravis Composite score at day 120
Time frame: 120 days
Change from baseline in Manual Muscle Testing (MMT) score at day 60
Time frame: 60 days
Change from baseline in Manual Muscle Testing (MMT) score at day 120
Time frame: 120 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 60
Time frame: 60 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 120
Time frame: 120 days
Change from baseline in Quality of Life Assessment (SF-36) at day 60
Time frame: 60 days
Change from baseline in Quality of Life Assessment (SF-36) at day 120
Time frame: 120 days
Change from baseline in acetylcholine receptor antibody titre level at day 60
Time frame: 60 days
Change from baseline in acetylcholine receptor antibody titre level at day 120
Time frame: 120 days
Change from baseline in prednisone dose at day 60
Time frame: 60 days
Change from baseline in prednisone dose at day 120 Days
Time frame: 120 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2012.
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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.
Matthew N Meriggioli, MD principal investigator
University of Illinois at Chicago, 912 S. Wood St., Rm 855-N, M/C 796, Chicago IL 60612
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in people with Myasthenia Gravis, run by Muscular Dystrophy Association 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 7 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: status not confirmed, as of the sponsor's last update on 15 Mar 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Matthew N Meriggioli, MD (312-996-4780, mmerig@uic.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of GM-CSF in patients with myasthenia gravis (time frame: 150 days). The study lists 4 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 15 Mar 2012.