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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

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

Key facts

Study ID

NCT06840496

Run by

Biomed Industries, Inc.

People needed

210

Starts

2026-09-30

Expected to finish

2027-12-30

Last updated by the study team

2026-03-27

Who can join

Age: 5 and older, up to 20. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Female subjects 5 to 20 years of age, inclusive, at Screening
• Body weight ≥12 kg at Screening
• Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• Has classic/typical Rett syndrome (RTT)
• Has a documented disease-causing mutation in the MECP2 gene
• Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
• 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.
• The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
• 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

You may not qualify if…

• Has been treated with insulin within 12 weeks of Baseline
• 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
• Has a history of, or current, cerebrovascular disease or brain trauma
• Has significant, uncorrected visual or uncorrected hearing impairment
• Has a history of, or current, malignancy
• Has a known history or symptoms of long QT syndrome

Where it is running

• Biomed Testing Facility # BIO-01-85012 — Phoenix, Arizona, United States (enrolling)
• Biomed Testing Facility # BIO-05-92093 — La Jolla, California, United States (enrolling)
• Biomed Testing Facility # BIO-04-90095 — Los Angeles, California, United States (enrolling)
• Biomed Testing Facility # BIO-03-95817 — Sacramento, California, United States (enrolling)
• Biomed Testing Facility # BIO-02-94104 — San Francisco, California, United States (enrolling)
• Biomed Testing Facility #BIO-06-80042 — Aurora, Colorado, United States (enrolling)
• Biomed Testing Facility #BIO-07-33606 — Tampa, Florida, United States (enrolling)
• Biomed Testing Facility #BIO-08-60612 — Chicago, Illinois, United States (enrolling)
• Maryland Locations Biomed Testing Facility #BIO-9-21205 — Baltimore, Maryland, United States (enrolling)
• Biomed Testing Facility #BIO-10-02115 — Boston, Massachusetts, United States (enrolling)
• Biomed Testing Facility #BIO-13-10467 — The Bronx, New York, United States (enrolling)
• Biomed Testing Facility #BIO-17-19104 — Philadelphia, Pennsylvania, United States (enrolling)
• Biomed Testing Facility #BIO-19-77030 — Houston, Texas, United States (enrolling)
• Biomed Testing Facility #BIO-20-98105 — Seattle, Washington, United States (enrolling)
• Biomed Research Unit-BIO-23-NSW 2050 — Camperdown, New South Wales, Australia (enrolling)
• Biomed Research Unit-BIO-24-NSW-4101 — South Brisbane, Queensland, Australia (enrolling)
• Biomed Research Unit-BIO-21-VIC-3084 — Heidelberg West, Victoria, Australia (enrolling)
• Biomed Research Unit- BIO-22-VIC 3010 — Parkville, Victoria, Australia (enrolling)

Full record on ClinicalTrials.gov

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