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Tests treatment safety and results for Generalized Myasthenia Gravis
Official title A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)
ClinicalTrials.gov ID: NCT06987539
What this study is testing
What is Inebilizumab?
Inebilizumab is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for generalized myasthenia gravis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent...
- Age ≥ 2 to \< 18 years of age on the day of enrollment.
- Diagnosis of gMG defined as:
- Positive serologic test for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody (Ab) titers as confirmed at...
- At least 1 of the following:
You likely can't join if
- Employees of the Sponsor, contract research organization (CRO), site staff, and their family members.
- Thymectomy within 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the treatment period.
- Unresected thymoma- Participants with benign thymoma resected \> 12 months prior to screening may enroll.
- History of recurrent significant infections.
- Known immunodeficiency disorder, including current infection or positive test for human immunodeficiency virus (HIV).
- Positive test for chronic hepatitis B infection at screening.
See the full eligibility criteria
- Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local...
- Age ≥ 2 to \< 18 years of age on the day of enrollment.
- Diagnosis of gMG defined as:
- Positive serologic test for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody (Ab) titers as confirmed at screening (1 retest allowed), and
- At least 1 of the following:
- History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or
- History of positive anticholinesterase test (eg, edrophonium chloride test); or
- Participant demonstrated improvement in gMG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or
- Clinical syndrome consistent with a diagnosis of gMG, and not otherwise explained by another condition.
- Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.
- Participants must be on:
- Corticosteroids only, with no dose increase within 4 weeks prior to screening, or
- One allowed non-steroidal immunosuppressive therapies (IST) (azathioprine, mycophenolate mofetil, or mycophenolic acid) with continuous use for at least 6 months prior to screening and no dose increase within 4 months...
- Combination of (1) corticosteroids with no dose increase within 4 weeks prior to screening and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to screening and no dose increase within 4...
- Participants may enter the study on a stable dose of acetylcholinesterase inhibitors (pyridostigmine dose). The acetylcholinesterase inhibitor dose must have been stable for at least 2 weeks prior to enrollment.
- Vital signs and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the investigator.
- Employees of the Sponsor, contract research organization (CRO), site staff, and their family members.
- Thymectomy within 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the treatment period.
- Unresected thymoma- Participants with benign thymoma resected \> 12 months prior to screening may enroll.
- History of recurrent significant infections.
- Known immunodeficiency disorder, including current infection or positive test for human immunodeficiency virus (HIV).
- Positive test for chronic hepatitis B infection at screening.
- History of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV).
- History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines.
- History of progressive multifocal leukoencephalopathy.
- Participants diagnosed with congenital myasthenic syndromes.
- Receipt of any biologic B-cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) or any experimental B-cell-depleting agent in the 6 months prior to screening.
- Receipt of any other monoclonal antibody (mAb) or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-janus kinase (JAK) Stat mAbs, and complement inhibitors within 6 months prior...
- Receipt of the following medications or treatments at any time prior to randomization: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy, natalizumab.
- Participants who are pregnant or breastfeeding or planning to get pregnant.
The study team makes the final eligibility decision.
Where it's taking place
- Austin, Texas, United States
- Paris, France
- Valencia, Spain
- Headington, Oxford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Austin, Texas, United States; Paris, France; Valencia, Spain; Headington, Oxford, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.