AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

โ† Trials
๐Ÿงช

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

RecruitingPhase 3Ages 5 Years - 20 YearsFemaleBiomed Industries, Inc.
๐Ÿ’ฌ

In plain words

What the trial is, and whether it could suit you

Primary Objective โ€ข To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective โ€ข To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
INCLUSION CRITERIA 1. Female subjects 5 to 20 years of age, inclusive, at Screening 2. Body weight โ‰ฅ12 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
๐Ÿ’Š

What it tests

  • Bionetide
  • Placebo
๐Ÿ“

Where

18 sites
Biomed Testing Facility # BIO-01-85012
Phoenix, United States
Biomed Testing Facility # BIO-05-92093
La Jolla, United States
Biomed Testing Facility # BIO-04-90095
Los Angeles, United States
Biomed Testing Facility # BIO-03-95817
Sacramento, United States
Biomed Testing Facility # BIO-02-94104
San Francisco, United States
Biomed Testing Facility #BIO-06-80042
Aurora, United States
Biomed Testing Facility #BIO-07-33606
Tampa, United States
Biomed Testing Facility #BIO-08-60612
Chicago, United States

โ€ฆand 10 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
๐Ÿ›ก๏ธ

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it โ€” not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use ยท Privacy