A chance to help shape the future of Duchenne muscular dystrophy (DMD) treatment
AGAMREE® SUMMIT Study
A chance to help shape the future of Duchenne muscular dystrophy (DMD) treatment
The AGAMREE SUMMIT Study joins other important research opportunities studying DMD, offering the benefits of an observational registry with the inclusion of state-of-the-art imaging and data collection. The study will follow 250 participants who are taking AGAMREE for the treatment of DMD for up to 5 years. Healthcare providers at 25 sites across the United States will gather a wide range of information from participants. Along with supplementing existing DMD research with real-world information, the main focus of the AGAMREE SUMMIT Study is to better understand outcomes associated with long-term AGAMREE treatment, including bone health, behavior, sexual development, and cataract formation.
Participants will be followed for approximately 5 years and will be expected to return to the study site for yearly follow-up visits.
Participants may receive travel assistance depending on the distance from the study site.
Participants will receive a physical examination at each visit, and blood work and state-of-the-art imaging tests may be conducted.
There will be no cost to participants in the study—the study will pay for all out-of-pocket expenses associated with procedures (physical exams,
blood draws, study scans, etc.). Procedure results will be available to the patient.
Quality-of-life questionnaires will also be completed at each return visit.
All members of the DMD community understand the importance of research. By participating in the AGAMREE SUMMIT Study, your family will provide important data that may be helpful in developing better treatment options for DMD patients in the future.
Participants in SUMMIT must be at least 2 years old, have received a confirmed diagnosis of DMD, and be currently taking AGAMREE. Participants or their parent/legal guardian (for those younger than 18 years old) must provide written informed consent after hearing about the AGAMREE SUMMIT Study and be willing to complete quality-of-life questionnaires.
Note: Not all care centers are participating in the AGAMREE SUMMIT Study.
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What is AGAMREE?
AGAMREE is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
IMPORTANT SAFETY INFORMATION
Patients should not take AGAMREE if they are allergic to vamorolone or any of the inactive ingredients in AGAMREE.
What is the most important information I should know about AGAMREE?
Do not stop AGAMREE, or change the amount taken, without first checking with your healthcare provider. There may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis, which can be fatal.
There is an increased risk of infection when taking corticosteroids like AGAMREE. Tell your healthcare provider if the patient has had recent or ongoing infections or has recently received a vaccine. Seek immediate medical advice in the case of fever or other signs of infection. Some infections can be severe, and sometimes fatal. Patients should avoid exposure to chickenpox or measles; alert your healthcare provider immediately if exposure occurs.
Corticosteroids, including AGAMREE, can cause an increase in blood pressure and water retention. Your healthcare provider may monitor for these increases during treatment.
There is an increased risk of developing a hole in the stomach or intestines in patients with certain gastrointestinal disorders when taking corticosteroids like AGAMREE.
Corticosteroids, including AGAMREE, can cause severe behavioral and mood changes. Seek medical attention if behavioral or mood changes develop.
There is a risk of osteoporosis with prolonged use of corticosteroids like AGAMREE, which can lead to vertebral and long bone fractures.
Corticosteroids like AGAMREE may cause cataracts or glaucoma. Your healthcare provider should monitor for these conditions if AGAMREE treatment continues for more than 6 weeks.
Immunizations should be up to date according to immunization guidelines prior to starting therapy with AGAMREE.Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting AGAMREE. Live-attenuated or live vaccines should not be administered in patients taking AGAMREE.
Rare instances of severe allergic reaction have occurred in patients receiving corticosteroid therapy.
Before taking AGAMREE, tell your healthcare provider about all medical conditions, including if the patient:
has decreased liver function
is pregnant or planning to become pregnant. AGAMREE can harm an unborn baby.
is breastfeeding or planning to breastfeed. AGAMREE may appear in breastmilk and could affect a nursing child.
Certain medications can cause an interaction with AGAMREE.
Tell your healthcare provider about all the medicines the patient takes, including prescription and over-the-counter medicines, dietary supplements, and herbal products.
What are the possible side effects of AGAMREE?
The most common side effects with AGAMREE include facial puffiness (cushingoid features), psychiatric disorders, vomiting, weight gain, and vitamin D deficiency. These are not all the possible side effects of AGAMREE.
Call your doctor for medical advice about side effects.
AGAMREE is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
IMPORTANT SAFETY INFORMATION
Patients should not take AGAMREE if they are allergic to vamorolone or any of the inactive ingredients in AGAMREE.
What is the most important information I should know about AGAMREE?
Do not stop AGAMREE, or change the amount taken, without first checking with your healthcare provider. There may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis, which can be fatal.
There is an increased risk of infection when taking corticosteroids like AGAMREE. Tell your healthcare provider if the patient has had recent or ongoing infections or has recently received a vaccine. Seek immediate medical advice in the case of fever or other signs of infection. Some infections can be severe, and sometimes fatal. Patients should avoid exposure to chickenpox or measles; alert your healthcare provider immediately if exposure occurs.
Corticosteroids, including AGAMREE, can cause an increase in blood pressure and water retention. Your healthcare provider may monitor for these increases during treatment.
There is an increased risk of developing a hole in the stomach or intestines in patients with certain gastrointestinal disorders when taking corticosteroids like AGAMREE.
Corticosteroids, including AGAMREE, can cause severe behavioral and mood changes. Seek medical attention if behavioral or mood changes develop.
There is a risk of osteoporosis with prolonged use of corticosteroids like AGAMREE, which can lead to vertebral and long bone fractures.
Corticosteroids like AGAMREE may cause cataracts or glaucoma. Your healthcare provider should monitor for these conditions if AGAMREE treatment continues for more than 6 weeks.
Immunizations should be up to date according to immunization guidelines prior to starting therapy with AGAMREE.Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting AGAMREE. Live-attenuated or live vaccines should not be administered in patients taking AGAMREE.
Rare instances of severe allergic reaction have occurred in patients receiving corticosteroid therapy.
Before taking AGAMREE, tell your healthcare provider about all medical conditions, including if the patient:
has decreased liver function
is pregnant or planning to become pregnant. AGAMREE can harm an unborn baby.
is breastfeeding or planning to breastfeed. AGAMREE may appear in breastmilk and could affect a nursing child.
Certain medications can cause an interaction with AGAMREE.
Tell your healthcare provider about all the medicines the patient takes, including prescription and over-the-counter medicines, dietary supplements, and herbal products.
What are the possible side effects of AGAMREE?
The most common side effects with AGAMREE include facial puffiness (cushingoid features), psychiatric disorders, vomiting, weight gain, and vitamin D deficiency. These are not all the possible side effects of AGAMREE.
Call your doctor for medical advice about side effects.
