Elan: A Study Involving a Drug that Might Improve Cognitive Capacities in People with Down Syndrome
This study is being funded by Elan, who are developing the study drug. The upcoming clinical trial will evaluate the effects of their study drug on learning, memory, and language abilities in people with Down syndrome. Elan expects that approximately 24 people with Down syndrome will participate in the U.S. Our MassGeneral Hospital Down Syndrome Program will be one of the sites enrolling interested and eligible participants. There is no guarantee that your family member will benefit from taking part in this research study.
To see if your family member is eligible to take part in this study, please review these questions first. If you would like your family member to participate in this study, or if you have further questions, please contact Mary Ellen McDonough, RN, Senior Clinical Research Coordinator in the MassGeneralHospital Down Syndrome Program, at (617)-643-5572 or email@example.com
Q: Why is this research study being done? How is this experimental drug supposed to work?
A: In this study we will be examining the effect of scyllo-inositol (ELND005) in patients with Down syndrome. Patients with Down syndrome have life-long increased levels of amyloid and myo-inositol, both of which can affect the health and functions of neurons. Scyllo-inositol has affects on both amyloid and myo-inositol that could have beneficial effects on cognition and behavior in Down syndrome. Elan is initiating this study to evaluate the use of scyllo-inositol in young adults with Down syndrome.
ELND005, an investigational product that has not been approved for sale by the FDA, will be administered as a pill for 4 weeks.
Q: How long will I take part in the research study?
A: The total amount of time in this study is approximately 10 weeks. A caregiver must personally accompany your family member with Down syndrome to all study visits. The caregiver must have contact with this family member at least 5 times a week. Someone else cannot take the caregiver’s place. That is why, before you agree to participate, a caregiver must make sure that he or she can meet these requirements.
Q: What does this study involve?
A: Each person in the study will be given either the study drug (called ELND005) or a pill that contains no medicine (placebo). A placebo looks the same as the study drug (ELND005) but has no medicine in it. Neither you nor the study doctor or study staff will be able to pick which study group your family member with Down syndrome is assigned to. Neither you nor the study doctor will know which drug is given.
If you agree to take part in this study you will be asked to make a total 5 visits to the study clinic. The length of the whole study will be about 10 weeks. Your first and last clinic visits could take as little as 6 hours or as long as 9 hours. All other visits will take approximately 2-6 hours. In addition, we will schedule 2 visits by phone.
Q: Will being in this study help my family member with Down syndrome or others?
A: There is no guarantee that your family member will benefit from this study. Information from the study might help researchers to come up with new tests or medications to help people with Down syndrome in the future.
Q: What are the requirements for my family member to participate in this study?
Your family member with Down syndrome must meet ALL of the following requirements to be able to participate in the drug study:
- Must provide signed and dated written consent form. From the patient if capable or from the patient’s legally authorized representative, with the patient’s assent (if the patient is not competent to provide informed consent).
- The patient’s study partner / caregiver must also be able to participate in the study.
- Is male or female, aged 18 to 45 years inclusive.
- Has a diagnosis of Down syndrome confirmed by karyotype; patients may have free trisomy 21 or Robertsonian translocations; mosaic Down syndrome will be excluded.
- Has an IQ of ≥ 40 as assessed by the Kaufman-Brief Intelligence Test (K-BIT), which will be administered during the first Screening Visit to our clinic to determine eligibility.
- Has been on stable doses of medication for the treatment of non-excluded medical conditions, if any, for at least 4 weeks before the Screening Visit and is expected to remain on the same doses of these medications throughout the duration of the study.
- Is able and willing to have a brain MRI.
- Has a study partner/caregiver who is in contact with the patient at least five times a week. The study partner/ caregiver must be:
a. willing and able to accompany the patient to all clinic visits, and
b. capable of monitoring and reporting the study drug’ use and adequately reporting he patient’s functional status.
- Both patients and study partner/caregiver are willing and able to participate in all scheduled evaluations and complete all required tests.
- Females of childbearing potential must agree to use two medically approved forms of contraception, one of which must be a barrier method, for the duration of the study and for 3 months after the last study drug administration.
- If a male patient with a partner of childbearing potential, must agree to use dual methods of contraception from first dose of study drug and until 3 months after the last dose of study drug.
If ANY of the following apply to the person with Down syndrome at this time, he or she would NOT qualify for this study:
- Has a co-occurring disease or condition (other than Down syndrome) or any clinically significant finding at the Screening Visit that could interfere with, or for which the treatment might interfere with, the conduct of the study, or that would, in the opinion of the Investigator (Dr. Brian Skotko), pose an unacceptable risk to the patient in this study.
- Has symptoms of dementia or worsening cognition over the past year.
- Has major depressive disorder as defined in the Diagnostic and Statistical Manual of Mental disorders- Fourth Edition Text Revision.
- Has brain MRI findings showing significant central nervous system pathology (such as, but not limited to, tumor, hydrocephalus, or vascular malformation), other than radiologic findings expected in patients with Down syndrome.
- Has a history of epilepsy or seizures other than benign febrile convulsions of childhood.
- Has a history of hepatitis B, hepatitis C, or human immunodeficiency virus infection (HIV).
- Is taking prescription or non-prescription medication with the potential to affect cognition after 4 weeks of Screening Visit (including, but not limited to, cholinesterase inhibitors, memantine, herbal preparations, opiod analgesics, or sedating antiallergy medications).
- Has taken inositol supplements within 4 weeks of the screening Visit.
- Has participated or is participating in a clinical trial of any investigational drug, device, or experimental medication or intervention, and received active drug within 4 weeks (or five half-lives, whichever is longer) of the Screening Visit.
- Has a positive urine test for drugs of abuse at the Screening Visit.
- Is a pregnant or lactating woman.
- Patient or study partner/caregiver has uncorrected sensory impairments (e.g., hearing, vision) that would impair the ability to perform the study assessments.
- Has a history of cancer, diagnosed and treated within the last 5 years, with the exception of basal cell carcinoma of the skin documented to have been excised with clean margins before the Screening Visit.
- Has a hemoglobin level < 11 g/dL(<110g/dL) male patients or < 10g/dL (<100g/dL) female patientsor has donated blood within 4 weeks before the Screening Visit.
- Has clinically significant abnormalities in serum thyroid-stimulating hormone (TSH) or in B12 levels. If patient is taking thyroid hormone replacement or vitamin B12 injections, corresponding screening test values must be within the reference range.
- Diet in renal disease formula.
- Has glycosylated hemoglobin (HbA1c) > 8% at the Screening Visit.
- Has a history of alcohol or drug dependence or abuse within the last 2 years.
- Has had any significant medical/psychiatric condition that is unstable and that would either:
- place the patient at undue risk from administration of study drug or undergoing study procedures, or
- interfere with the interpretation of safety, pharmacokinetics, or efficacy evaluations obtained in the course of the study, or
- interfere with the absorption of ELND005.
- Has any disease or condition that might compromise the cardiovascular, hematological, renal, hepatic, pulmonary, endocrine, central nervous, immune, or gastrointestinal systems (unless deemed not clinically significant by the Investigator and Elan’s Medical Monitior).
During the study you may take medications for the following reasons:
- Heart problems, including medications such as Plavix® (clopidogrel), aspirin, and related medications
- Stomach problems
- Lung or breathing problems
- Kidney problems
- Pain (all over the counter medications)
During the study you may NOT take any of the following medications:
- Sleep medications (all over the counter medications containing benadryl), except trazadone
- Benzodiazepines (Valium® and similar medications), except lorazepam
- Regular use of narcotics – please check with your doctor for specific medications
- Blood thinners such as Coumadin® (warfarin) – please check with your doctor for specific medications
- Seizures or epilepsy, except for Neurontin® (gabapentin) and Lyrica® (pregabalin) when prescribed for nerve pain
- For Alzheimer’s Disease/dementia, such as Aricept® (donepezil), Exelon® (rivastigmine), Namenda® (memantine), Razadyne® (galantamine)
- Medications with the potential to affect cognition including prescription medications (opioid analgesics, thyroid supplements, or vitamin B12 supplements by injection) or nonprescription medications (including, but not limited to, herbal preparations [e.g., Ginkgo biloba], over the-counter sleeping aids [e.g., diphenhydramine], and sedating anti-allergy medications)
- Stimulants such as methamphetamines, Provigil® (modafinil), and Nuvigil® (armodafinil)
- Experimental medications
For other medications, or if you do not know your medications well, please check with the study staff for or any questions as to whether these are permitted or not.
Learn more about the Down Syndrome Program at Mass General, including prenatal services and clinics for infants and toddlers, school-aged children, adolescents and adults.
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