Efficacy of Bionetide for treating Rett syndrome in females

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the effectiveness of oral Bionetide compared to a placebo in females aged 5 to 20 with Rett syndrome. The primary focus is on measuring changes in behavior and communication abilities over a 12-week period. Key assessments include the Rett Syndrome Behavior Questionnaire and the Clinical Global Impression-Improvement score. The study aims to provide insights into the potential benefits of Bionetide for improving the quality of life in affected individuals.

Why it matters

Who can join: the full rules

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

Where it's running

• Biomed Testing Facility # BIO-01-85012
  Phoenix, Arizona, United States Recruiting
• Biomed Testing Facility # BIO-05-92093
  La Jolla, California, United States Recruiting
• Biomed Testing Facility # BIO-04-90095
  Los Angeles, California, United States Recruiting
• Biomed Testing Facility # BIO-03-95817
  Sacramento, California, United States Recruiting
• Biomed Testing Facility # BIO-02-94104
  San Francisco, California, United States Recruiting
• Biomed Testing Facility #BIO-06-80042
  Aurora, Colorado, United States Recruiting

Study contacts

• Study coordinator: David L Nguyen, PhD
• Email: research@biomedind.com
• Phone: 1-800-824-5135

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Auditory EEG and behavioral testing for people with Rett syndrome

  This project will record noninvasive EEG while people with Rett syndrome and matched controls listen to simple sounds to see how their brains process sound.

  Recruiting University of Rochester · Rochester, New York · NCT07569445

• Fenfluramine HCl for Rett Syndrome in a Phase 3 study

  This study tests whether fenfluramine hydrochloride helps people with Rett syndrome when compared to a placebo.

  Recruiting at 40 sites Phase 3 UCB Pharma · Little Rock, Arkansas and 39 other locations · NCT07503444

• Daily oral vorinostat for girls and young women with Rett syndrome

  This trial will try daily oral vorinostat at two escalating dose levels, using a four-week placebo baseline and an N-of-1 design so each participant serves as her own control, to see if the drug is safe and shows clinical or molecular benefit in girls and young women with typical Rett syndrome.

  Recruiting Phase 1 Unravel Biosciences, Inc. · Medellín · NCT07150013

• Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

  This study is testing how well eye-tracking tasks can measure cognitive abilities in people with Rett syndrome who are starting treatment with trofinetide.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT06705816

• Identifying biomarkers in Rett Syndrome

  This study is trying to find specific brain signals in girls with Rett Syndrome to see how they differ from girls without the condition and how these signals relate to the severity of their symptoms over time.

  Recruiting at 6 sites Children's Hospital of Philadelphia · Los Angeles, California and 5 other locations · NCT05932589

• Gene editing to correct common MECP2 mutations in Rett syndrome

  This project will test CRISPR/Cas9 delivered with AAV to correct common MECP2 mutations in females with Rett syndrome using lab and animal work.

  Recruiting University of Siena · Siena, Siena · NCT05740761

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.