To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Launched by BIOMED INDUSTRIES, INC. · Feb 18, 2025

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of a medication called Bionetide in girls and women with Rett syndrome, a rare genetic disorder that mainly affects females and can lead to difficulties with communication and movement. The main goal is to see if Bionetide can improve symptoms compared to a placebo, which is a dummy treatment with no active medication. Researchers also want to find out if this treatment helps improve communication abilities in the participants.

To participate in this study, girls and women aged 5 to 20 who have been diagnosed with typical Rett syndrome and have a specific genetic mutation may be eligible. They should be able to swallow the medication or take it through a feeding tube and must not have had any major health issues recently. Participants can expect to take the treatment for a certain period while being monitored by healthcare professionals. It's important to note that caregivers must speak English well enough to help with assessments, and participants should not be pregnant or breastfeeding. This trial is currently recruiting participants, and those interested should discuss it with their healthcare provider for more information.

Gender

FEMALE

Eligibility criteria

• • INCLUSION CRITERIA
• • 1. Female subjects 5 to 20 years of age, inclusive, at Screening
• • 2. Body weight ≥12 kg at Screening
• • 3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• • 4. Has classic/typical Rett syndrome (RTT)
• • 5. Has a documented disease-causing mutation in the MECP2 gene
• • 6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
• • 7. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
• • 8. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
• • 9. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening
• • EXCLUSION CRITERIA
• • 1. Has been treated with insulin within 12 weeks of Baseline
• • 2. Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
• • 3. Has a history of, or current, cerebrovascular disease or brain trauma
• • 4. Has significant, uncorrected visual or uncorrected hearing impairment
• • 5. Has a history of, or current, malignancy
• • 6. Has a known history or symptoms of long QT syndrome