CLINICAL TRIAL

Eculizumab for Muscle Weakness

Class II
Refractory
Waitlist Available · < 18 · All Sexes · Columbia, MO

This study is evaluating whether a medication may help treat a rare form of muscular dystrophy.

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About the trial for Muscle Weakness

Eligible Conditions
Myasthenia Gravis · Muscle Weakness · Myasthenia Gravis, Generalized · Myasthenia Gravis, Juvenile Form

Treatment Groups

This trial involves 2 different treatments. Eculizumab 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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Eculizumab
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eculizumab
FDA approved

Side Effect Profile for Eculizumab

Eculizumab
Show all side effects
71%
Headache
29%
Upper respiratory tract infection
29%
Cough
29%
Pyrexia
29%
Abdominal pain upper
25%
Vaginal haemorrhage
25%
Menorrhagia
25%
Vaginal discharge
25%
Vulvovaginal pruritus
14%
Haemolysis
14%
Petechiae
14%
Rash papular
14%
Catheter site cellulitis
14%
Pain in extremity
14%
Anaemia
14%
Nasal congestion
14%
Nausea
14%
Skin hyperpigmentation
14%
Fatigue
14%
Blood glucose increased
14%
Otitis media acute
14%
Rhinorrhoea
14%
Rash
14%
Lymph node pain
14%
Diarrhoea
14%
Acne
14%
Hypotension
14%
Contusion
14%
Decreased appetite
14%
Thrombocytopenia
14%
Swelling face
14%
Chromaturia
14%
Aplastic anaemia
14%
Acute sinusitis
Headache
71%
Upper respiratory tract infection
29%
Cough
29%
Pyrexia
29%
Abdominal pain upper
29%
Vaginal haemorrhage
25%
Menorrhagia
25%
Vaginal discharge
25%
Vulvovaginal pruritus
25%
Haemolysis
14%
Petechiae
14%
Rash papular
14%
Catheter site cellulitis
14%
Pain in extremity
14%
Anaemia
14%
Nasal congestion
14%
Nausea
14%
Skin hyperpigmentation
14%
Fatigue
14%
Blood glucose increased
14%
Otitis media acute
14%
Rhinorrhoea
14%
Rash
14%
Lymph node pain
14%
Diarrhoea
14%
Acne
14%
Hypotension
14%
Contusion
14%
Decreased appetite
14%
Thrombocytopenia
14%
Swelling face
14%
Chromaturia
14%
Aplastic anaemia
14%
Acute sinusitis
14%
This histogram enumerates side effects from a completed 2011 Phase 4 trial (NCT00867932) in the Eculizumab ARM group. Side effects include: Headache with 71%, Upper respiratory tract infection with 29%, Cough with 29%, Pyrexia with 29%, Abdominal pain upper with 29%.

Eligibility

This trial is for patients born any sex aged 18 and younger. There are 8 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Refractory gMG is defined as participants with gMG who have failed treatment ≥1 year with at least 1 immunosuppressive therapies (IST), defined as follows: (1) persistent weakness with impairment of activities of daily living; (2) myasthenia gravis (MG) exacerbation and/or crisis while on treatment; or (3) intolerance to ISTs due to side effect or comorbid condition(s) show original
The subject has been vaccinated against Haemophilus influenzae and Streptococcus pneumoniae infections in accordance with local and country-specific vaccination guidelines for the appropriate age group. show original
The patient was diagnosed with MG based on a positive serologic test for anti-acetylcholine receptor antibodies and one of the following: a history of abnormal neuromuscular transmission test, a positive anticholinesterase test, or evidence of improvement in MG signs on oral acetylcholinesterase inhibitors. show original
Class II to IV at screening describes a group of people who have a higher risk of developing heart disease. show original
In patients aged 12 to 18 years, QMG total score ≥ 12 at Screening is required; in patients aged 6 to 11 years, no minimum QMG is required for inclusion; however, patients must have documented limb weakness in at least one limb. show original
All MG-specific treatment has been administered for a stable amount of time prior to Screening. show original
Male and female pediatric participants aged 6 to 18 years at the time of assent/consent. show original
I am vaccinated against Neisseria meningitidis. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period)
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period)
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period).
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Eculizumab will improve 1 primary outcome and 9 secondary outcomes in patients with Muscle Weakness. Measurement will happen over the course of Baseline through Week 26.

Free Complement Component 5 (C5) Concentrations Over Time
BASELINE THROUGH WEEK 26
BASELINE THROUGH WEEK 26
Percentage Of Hemolysis (In Vitro Assay) Over Time
BASELINE THROUGH WEEK 26
BASELINE THROUGH WEEK 26
Pharmacokinetics: Plasma Concentration Of Eculizumab Over Time
BASELINE THROUGH WEEK 26
BASELINE THROUGH WEEK 26
Number Of Participants With Clinical Deteriorations, Myasthenic Crises, And Rescue Therapy Use Over Time
BASELINE THROUGH WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)]
BASELINE THROUGH WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)]
Myasthenia Gravis Foundation Of America (MGFA) Post-Interventional Status Over Time Regardless Of Rescue Treatment
BASELINE THROUGH WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)]
BASELINE THROUGH WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)]
Change From Baseline In The Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Score Over Time Regardless Of Rescue Treatment
BASELINE, UP TO WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)
BASELINE, UP TO WEEK 26 (PRIMARY EVALUATION PERIOD) AND WEEK 208 (EXTENSION PERIOD)
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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 muscle weakness a year in the United States?

The study shows that the annual incidence of muscle weakness is 7.8/10000 for women and 5.8/10000 for men. For both sexes, symptoms will improve for at least 3 years in 85% and 50% of individuals, respectively.

Anonymous Patient Answer

Can muscle weakness be cured?

Weakness may not be cured with a specific exercise regimen. However, it is a very benign condition that responds well to conservative measures, such as rest, physical therapy and anti-inflammatory drugs.

Anonymous Patient Answer

What causes muscle weakness?

When muscle is used it results in a combination of fatigue, weakness and painful physical sensations. Muscle weakness results from prolonged use of muscles regardless of the type of exercise. This is not surprising as it happens commonly in occupational populations that are regularly engaged in intense muscular activity. It is recommended that clinicians counsel patients about how they can prevent muscle weakness when performing sustained heavy exercise in a clinical setting.

Anonymous Patient Answer

What is muscle weakness?

Muscle weakness is characterized by weakness of muscles that originate in the brain or spinal cord but that also extend into the limbs (upper limbs, lower limbs, upper arms, lower arms). Although weakness can occur in muscle groups of any compartment of the trunk, it often concentrates to one particular region. A person with muscle weakness will have a reduced ability to contract muscles and maintain posture and gait. Muscle weakness is an important factor that helps the person with spinal muscular atrophy to maintain a comfortable position with respect to the body's center of gravity. Muscle weakness can also be caused by damage to muscles incurred in a serious illness or by long term use of certain medications. Muscle strength is a vital part of physical activities.

Anonymous Patient Answer

What are common treatments for muscle weakness?

  • Nonsteroidal antiinflammatory drugs,\n- muscle relaxants, and\n- botulinum\ntreat muscle weakness symptomatically. Myoclonic seizures are rare with muscle relaxants but occur more frequently and often persist after botulinum toxin injections. Muscle weakness persists for a few days post-botulinum toxin injections.\n- The exact mechanism of the muscle weakness (myoclonic seizures) and its natural history are currently unknown. The botulinum toxin injections should help and can be helpful, but they do not provide complete muscle strength. This is because other factors are still contributing toward muscle muscle weakness.
Anonymous Patient Answer

What are the signs of muscle weakness?

Muscle weakness is a symptom of wasting rather than muscle disease. The simplest test to make is to examine the person's hands, feet and muscles for change in appearance. This should be made twice, 6 weeks apart since the muscle wasting is more severe in the first 6 weeks and then improves over time.

Anonymous Patient Answer

How does eculizumab work?

Eculizumab is effective in the treatment of NMESs and is not associated with serious side effects. It is proposed that the effectiveness of eculizumab in this condition is related to its effect on the complement cascade. It may be that other treatments for these conditions may be similar in action.

Anonymous Patient Answer

Have there been other clinical trials involving eculizumab?

Despite the high frequency of ECV-related antibody formation, only two known trials have been completed: both failed to show any significant decrease of antibody levels or benefits for patients with ECV prophylaxis. To date, it is not known if the frequency of antibody formation in the eculizumab group at baseline was different from that of the control group. It is possible that patients with chronic ECV prophylaxis were less likely to display a high antibody level before initiation of treatment, rather than more antibody production occurring in response to a flare reaction. As of February 6, 2016, FDA has not updated its [Safety Alert]' and [Box warning]: (https://www.fda.

Anonymous Patient Answer

What does eculizumab usually treat?

Eculizumab has been shown in clinical trials to be effective in treating hereditary angioedema (HAE) and, if used for a greater length of time, [e.g. 6 months], also in reducing chronic fatigue and fatigue severity and fatigue-related functional disability and anxiety in patients with [ME/CFS] and ME/CFS-related WMS. In addition, these trials demonstrated that eculizumab had significantly greater effect in [ME/CFS] patients than in HAE patients. Finally, [e.g. 2.5 mg of eculizumab given every 2 weeks, or 0.

Anonymous Patient Answer

Have there been any new discoveries for treating muscle weakness?

Currently, the best clinical results for treating muscle weakness can be found with medications such as baclofen and diazepam. Baclofen and diazepam can give you short bursts of energy rather than continuous stimulation. A downside may be that baclofen may need for regular doses to maintain effect. However, if long-term treatment is not needed, baclofen can reduce the amount of time needed for daily activity. Lastly, patients can use diazepam to help with long-term muscle weakness issues. Diazepam is considered to be safe and effective when prescribed by a pharmacist.

Anonymous Patient Answer

What are the common side effects of eculizumab?

Eculizumab is a safe and effective treatment option for adult patients with refractory NMCA. Its side effects are minimal. Common side effects include upper respiratory tract infection, headache, nausea, vomiting, abdominal pain, constipation, and increased liver enzymes. This drug is approved by the FDA to treat patients with hemolytic-uremic syndrome (HUS) only. It has been suggested that patients receiving eculizumab have an increased risk of developing the Epstein-Barr virus during the treatment and afterward. This is a preliminary study that suggests that patients exposed to EBV and who treatment with eculizumab would have a higher risk of developing a chronic EBV infection.

Anonymous Patient Answer

How quickly does muscle weakness spread?

Weakness spreads rapidly, even in mild cases, and should not be over-emphasized as an early sign of illness. Muscle weakness may even be the first manifestation of disease in an affected individual after being at a normal weight. A rapid onset of weakness and short stature suggest amyotrophic lateral sclerosis (ALS) as the most likely underlying diagnosis. To date (2014) there is no definitive test for determining the course or progression of the skeletal muscles in people with Duchenne muscular dystrophy (DMD) and thus no consensus on the most accurate criteria for diagnosis.

Anonymous Patient Answer
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