Myasthenia GravisPhase 1SIMMNCT01828294
Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis
A Phase 1 study testing Subcutaneous immunoglobulins in people with Myasthenia Gravis, run by St. Louis University at 2 sites in United States.
Trial summary
The study is being done with patients with Myasthenia Gravis (MG), age 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents. 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?
- Subcutaneous immunoglobulins
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2011to Dec 2017
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- AChR Ab positive myasthenia gravis (acetylcholine receptor antibody).
- Age 18-80 years.
- MGFA Classification II-IV (The scale used to determine the severity of symptoms of MG).
- Receiving > or equal 30mg of Prednisone per day.
- No new MG-specific treatments in prior 3 months.
- Willingness to participate in study protocol.
- QMG > 10 (quantitative myasthenia gravis score: the sum of grades given for symptoms of MG).
- Treatment with any immunomodulator > than or equal to 3 months prior to trial initiation.
Who cannot join
- IgA deficiency (a major class of immunoglobulins found in serum and external body secretions such as saliva, tears, and sweat as well as in the gastrointestinal, respiratory, and genitourinary tracts).
- Previous thromboembolic events, including deep vein thrombosis, stroke and myocardial infarction
- MGFA Class I, IV (if patient requires hospitalization) or V
- History of thymoma
- Thymectomy in previous year or planning to undergo thymectomy in next six months
- Pregnancy or lactation; unwillingness to avoid pregnancy
Show all 11Show fewer
- Serious concurrent medical, neurological or psychiatric condition that would interfere with IGSC administration or subsequent clinical assessments
- Unwillingness or incapacity to participate, agree to necessary follow-up visits, or give written and informed consent
- Patients who have had an anaphylactic or severe systemic reaction to the administration of human immune globulin or to components of Hizentra, such as polysorbate 80, or patients with hyperprolinemia because it contains the stabilizer L-proline
- Cholinesterase inhibitor no more than 240 mg/day
- Body weight greater than 120 kg.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Saint Louis University
St Louis, Missouri, 63110
George Washington University
Washington D.C., District of Columbia, 20037
About this study
The study is being done with patients with Myasthenia Gravis (MG), age 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents. A common treatment for patients with this disease includes the administration of intravenous immunoglobulin (IVIG), which is a plasma protein that is given to help maintain adequate antibody levels to prevent infections and decrease the symptoms of the disease of Myasthenia Gravis. This study is being done to test if giving this medication in a subcutaneous form (into the fat of the abdomen, legs and thighs) will be better tolerated for patients with Myasthenia Gravis.
Read the detailed description
The study is a pilot study to ascertain the feasibility and tolerability of subcutaneous immunoglobulin (SCIG or IGSC) as a maintenance therapy for patients with non-thymomatous MG patients (MGFA class II-IV) at entry, aged 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents.
The neurologist principal investigator at each site will have the overall responsibility for study performance is designated the medical coordinator (MC). The MC will assess patients from the sites clinic populations and identify potential subjects for inclusion and exclusion criteria. Once a subject is identified and provided informed consent to participate the Visit Schedule will be initiated.
At the initial visit the MC will perform the acetylcholine receptor antibody level, and record the prednisone and anticholinesterase doses. The MC will be responsible for assessment of adverse events. The research coordinator will arrange for the initial laboratory testing at the patient's local Quest, where the blood will be drawn. Baseline lab tests to be done will include IgA level to evaluate for deficiency, IgG level, CBC, AchR antibody, pregnancy tests in women, LFT's, PT/PTT and BUN/Creatinine. The patient will complete the SF-36 quality of life, MG, and MGFA ADL The research coordinator will be responsible for training subjects in performance of IGSC infusion. Subjects will have outpatient clinic assessments in one week and then monthly for the remainder of the study. Patients will receive 2gms/kg divided over 4 weeks initially and then will be given 250mgs/kg/wk for total of 6 months. This is similar to the standard IV treatment for patients which is 2 gm/kg given over 2-5 days for the initial dose. After the initial dose, a patient is started on monthly IV maintenance dose of 1 gm/kg each month given over 1-3 days.
The subject will be evaluated monthly for assessment of whether minimal manifestation (MM) status has been reached, which then allows reduction of corticosteroids by 5mg or more if clinically indicated. The MC will record adverse events and symptoms. The dose of anticholinesterase drugs will be decreased at the discretion of the MC. The prednisone dose will be decreased unless the MM status is lost; in that situation the prednisone dose will be increased 10mg every 2 weeks until the MM is again achieved. Titration of the prednisone and cholinesterase inhibitor medications will be at the discretion of the physician and will be based on the patient's symptoms as measured by symptoms and examination, leading to a determination of the MM. The patient will complete the SF-36 quality of life assessment.
- Conditions
- Myasthenia Gravis
- Being tested
- Subcutaneous immunoglobulins
- Keywords
- MG
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Subcutaneous immunoglobulinsDrug
Also called Hizentra
Immunoglobulins used subcutaneously for maintenance of other immune mediated disorders.
| Study group | Role | What they receive |
|---|---|---|
| Study Population | Other | Study population will include patients (18-80 years old) with non-thymomatous myasthenia gravis MGFA Class II-IV receiving a minimum of 30mg of Prednisone daily and no other immunosuppression and no more 240 mgs per day of Cholinesterase inhibitor. Patients will receive Subcutaneous immunoglobulins weekly for 6 months. Receives: Drug: Subcutaneous immunoglobulins |
What is being measured
The primary outcome is the main question the study is designed to answer.
To monitor number of participants completing the study for the six months period
To evaluate feasibility and tolerability of IGSC as a maintenance treatment of generalized MG as measured by treatment adherence and adverse events experienced by the patient. Patients will be asked if they have experienced any of the most commonly known side effects of Hizentra. A case report form detailing any adverse events will be included with every study visit and reviewed with the patient at each visit.
Measured: Baseline to 6 months
Secondary outcomes (7)
To monitor number of adverse events in participants
•To evaluate safety IGSC for treatment of generalized MG as measured by adverse events experienced by each patient and reviewed monthly by the principal investigator.
Measured: Baseline to 6 months
Number of participants able to decrease prednisone dose below 30 mgs
- To evaluate the potential steroid-sparing effect of IGSC treatment as measured by the total dose of prednisone required by each patient over the six month period.
Measured: Baseline to 6 months
To monitor effect on manual muscle testing
- To evaluate the effect of IGSC treatment on symptoms associated with MG as measured by the QMG and MG-ADL.
Measured: Baseline to 6 months
To measure changes on SF-36 quality of life measurement tool before and after completion of study
- To evaluate Quality of Life with IGSC treatment for MG as measured by the SF-36.
Measured: Baseline to 6 months
To measure and correlate levels of serum IgG with clinical response of the participants
- To evaluate pharmacokinetics (IgG) levels associated with IGSC treatment for MG as measured by the monthly blood draws.
Measured: Baseline to 6 months
To monitor minimal manifestation of Myasthenia Gravis
To evaluate the effect of IGSC treatment on symptoms associated with MG
Measured: Baseline to 6 months
To monitor effect on Quantitative Myasthenia Gravis Score
To evaluate the effect of IGSC treatment on the Quantitative Myasthenia Score. Data collected will be qualitative and quantitative, and scores will be compared over time. Data will be captured on case report forms, and entered into an SPSS database. Dr. Gary Cutter, who is serving as a statistician for this study, will review the data monthly for safety, and a report will be sent to Dr. Hayat.
Measured: Baseline to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2013 (estimated)
- Study startOct 2011
- Primary completionDec 2017
- Study completionDec 2017
Why it stopped: Study stopped by sponsor due to difficulty enrolling at site
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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Louis University
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring
- Responsible party
- Principal investigator, Ghazala Hayat, St. Louis University
- Investigators
- Ghazala Hayat, M.D., Principal investigator, St. Louis UniversityJafar Kafaie, M.D., Principal investigator, St. Louis University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01828294
- Sponsor's protocol ID
- 17005
- Acronym
- SIMM
- Status last verified
- Apr 2019
- First submitted
- 1 Nov 2011
- Last updated
- 3 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT01828294
- Acronym / sponsor ID
- SIMM / 17005
- Conditions
- Myasthenia Gravis
- Keywords
- MG
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study stopped by sponsor due to difficulty enrolling at site
- Enrolment
- 4 participants (actual)
- Lead sponsor
- St. Louis UniversityUniversity, hospital or other
- Collaborators
- CSL Behring
- Study start
- Oct 2011
- Primary completion
- Dec 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 10 Apr 2013 (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 study is being done with patients with Myasthenia Gravis (MG), age 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents. A common treatment for patients with this disease includes the administration of intravenous immunoglobulin (IVIG), which is a plasma protein that is given to help maintain adequate antibody levels to prevent infections and decrease the symptoms of the disease of Myasthenia Gravis. This study is being done to test if giving this medication in a subcutaneous form (into the fat of the abdomen, legs and thighs) will be better tolerated for patients with Myasthenia Gravis.
Detailed description
The study is a pilot study to ascertain the feasibility and tolerability of subcutaneous immunoglobulin (SCIG or IGSC) as a maintenance therapy for patients with non-thymomatous MG patients (MGFA class II-IV) at entry, aged 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents.
The neurologist principal investigator at each site will have the overall responsibility for study performance is designated the medical coordinator (MC). The MC will assess patients from the sites clinic populations and identify potential subjects for inclusion and exclusion criteria. Once a subject is identified and provided informed consent to participate the Visit Schedule will be initiated.
At the initial visit the MC will perform the acetylcholine receptor antibody level, and record the prednisone and anticholinesterase doses. The MC will be responsible for assessment of adverse events. The research coordinator will arrange for the initial laboratory testing at the patient's local Quest, where the blood will be drawn. Baseline lab tests to be done will include IgA level to evaluate for deficiency, IgG level, CBC, AchR antibody, pregnancy tests in women, LFT's, PT/PTT and BUN/Creatinine. The patient will complete the SF-36 quality of life, MG, and MGFA ADL The research coordinator will be responsible for training subjects in performance of IGSC infusion. Subjects will have outpatient clinic assessments in one week and then monthly for the remainder of the study. Patients will receive 2gms/kg divided over 4 weeks initially and then will be given 250mgs/kg/wk for total of 6 months. This is similar to the standard IV treatment for patients which is 2 gm/kg given over 2-5 days for the initial dose. After the initial dose, a patient is started on monthly IV maintenance dose of 1 gm/kg each month given over 1-3 days.
The subject will be evaluated monthly for assessment of whether minimal manifestation (MM) status has been reached, which then allows reduction of corticosteroids by 5mg or more if clinically indicated. The MC will record adverse events and symptoms. The dose of anticholinesterase drugs will be decreased at the discretion of the MC. The prednisone dose will be decreased unless the MM status is lost; in that situation the prednisone dose will be increased 10mg every 2 weeks until the MM is again achieved. Titration of the prednisone and cholinesterase inhibitor medications will be at the discretion of the physician and will be based on the patient's symptoms as measured by symptoms and examination, leading to a determination of the MM. The patient will complete the SF-36 quality of life assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study Population | Other | Drug: Subcutaneous immunoglobulinsStudy population will include patients (18-80 years old) with non-thymomatous myasthenia gravis MGFA Class II-IV receiving a minimum of 30mg of Prednisone daily and no other immunosuppression and no more 240 mgs per day of Cholinesterase inhibitor. Patients will receive Subcutaneous immunoglobulins weekly for 6 months. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- AChR Ab positive myasthenia gravis (acetylcholine receptor antibody).
- Age 18-80 years.
- MGFA Classification II-IV (The scale used to determine the severity of symptoms of MG).
- Receiving > or equal 30mg of Prednisone per day.
- No new MG-specific treatments in prior 3 months.
Key exclusion criteria (11 listed)
- IgA deficiency (a major class of immunoglobulins found in serum and external body secretions such as saliva, tears, and sweat as well as in the gastrointestinal, respiratory, and genitourinary tracts).
- Previous thromboembolic events, including deep vein thrombosis, stroke and myocardial infarction
- MGFA Class I, IV (if patient requires hospitalization) or V
- History of thymoma
- Thymectomy in previous year or planning to undergo thymectomy in next six months
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 |
|---|---|---|
| Subcutaneous immunoglobulinsAlso: HizentraImmunoglobulins used subcutaneously for maintenance of other immune mediated disorders. | Drug | Study Population |
Primary outcome
To monitor number of participants completing the study for the six months period
Time frame: Baseline to 6 months
To evaluate feasibility and tolerability of IGSC as a maintenance treatment of generalized MG as measured by treatment adherence and adverse events experienced by the patient. Patients will be asked if they have experienced any of the most commonly known side effects of Hizentra. A case report form detailing any adverse events will be included with every study visit and reviewed with the patient at each visit.
7 secondary outcomes
To monitor number of adverse events in participants
Time frame: Baseline to 6 months
Number of participants able to decrease prednisone dose below 30 mgs
Time frame: Baseline to 6 months
To monitor effect on manual muscle testing
Time frame: Baseline to 6 months
To measure changes on SF-36 quality of life measurement tool before and after completion of study
Time frame: Baseline to 6 months
To measure and correlate levels of serum IgG with clinical response of the participants
Time frame: Baseline to 6 months
To monitor minimal manifestation of Myasthenia Gravis
Time frame: Baseline to 6 months
To monitor effect on Quantitative Myasthenia Gravis Score
Time frame: Baseline to 6 months
Results and safety
The study was stopped early (reason given: Study stopped by sponsor due to difficulty enrolling at site). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2019.
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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.
Ghazala Hayat, M.D. principal investigator
St. Louis University
Jafar Kafaie, M.D. principal investigator
St. Louis University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Subcutaneous immunoglobulins in people with Myasthenia Gravis, run by St. Louis University at 2 sites 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 8 inclusion and 11 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: stopped early, as of the sponsor's last update on 3 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 was stopped early (reason given: Study stopped by sponsor due to difficulty enrolling at site). Results are not available in the source record.
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?
To monitor number of participants completing the study for the six months period (time frame: Baseline to 6 months). 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 3 Apr 2019.