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

Biomed Industries

Status and phase: Enrolling; Phase 3

Conditions: Rett Syndrome

Treatments: Drug: Placebo; Drug: Bionetide

Study type: Interventional

Funder types: Industry

Identifiers

NCT06840496

BIO-2025R02

Details and patient eligibility

About

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

Full description

Co-Primary Endpoints

• Rett Syndrome Behavior Questionnaire (RSBQ) total score - Change from Baseline to Week 12
• Clinical Global Impression-Improvement (CGI-I) Score at Week 12

Key Secondary Endpoint

Change from Baseline to Week 12 in:

• Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist- Social Composite Score (CSBS-DP-IT Social)

Enrollment: 210 estimated patients

Sex: Female

Ages: 5 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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210 participants in 2 patient groups, including a placebo group

Bionetide

Experimental group

Description:

Experimental: Bionetide Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Treatment:

Drug: Bionetide

Placebo

Placebo Comparator group

Description:

Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Treatment:

Drug: Placebo