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

Conditions

• Rett Syndrome

Eligibility Criteria

Sex

FEMALE

Age

5 Years - 20 Years

Healthy Volunteers

Not accepted

Interventions

• Bionetide — DRUG
  Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
• Placebo — DRUG
  Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Study Details

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 Dates

Start date

Sep 30, 2026

Status verified

Mar 2026

Primary completion

Sep 30, 2027

Completion

Dec 30, 2027

Study Design

Enrollment

210 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

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

Primary Outcome Measure

Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 [ Time Frame: Baseline and Week 12 ]

Central Contacts

• David L Nguyen, PhD
  1-800-824-5135
  [email protected]
• Jennifer Thomposn, MS
  1-800-824-5135
  [email protected]

Locations (14)

Find similar trials in Phoenix, AZ

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