Bionetide for Rett Syndrome

Recruiting at 25 trial locations

Overseen By Jennifer Thomposn, MS

Age: < 65

Sex: Female

Trial Phase: Phase 3

Sponsor: Biomed Industries, Inc.

Must not be taking: Insulin

Disqualifiers: Cardiovascular, Endocrine, Renal, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

210 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have been treated with insulin within 12 weeks of the study's start. It's best to discuss your current medications with the trial team.

Is Bionetide safe for humans?

Trofinetide, which may be related to Bionetide, has been studied for safety in children with Rett Syndrome. While further research is needed to fully understand potential side effects, it has been approved by the FDA, indicating a level of safety for use in this condition. ^{1 2 3 4 5}

What data supports the effectiveness of the drug Bionetide for Rett Syndrome?

Trofinetide, a drug similar to Bionetide, has been approved by the FDA for treating Rett Syndrome and has shown significant improvements in behavioral scores in clinical studies, indicating potential effectiveness for similar treatments. ^{5 6 7 8 9}

Who Is on the Research Team?

Lloyd L Tran, PhD

Principal Investigator

Biomed Industries, Inc.

David Nguyen, PhD

Principal Investigator

Biomed Industries, Inc.

Zung V Tran, PhD

Principal Investigator

Biomed Industries, Inc.

Are You a Good Fit for This Trial?

This trial is for females with Rett Syndrome, a neurological disorder. Participants should be able to swallow pills and not have other conditions that could interfere with the study or pose risks.

Inclusion Criteria

Body weight ≥12 kg at 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

Exclusion Criteria

I have or had cancer before.

I have had a stroke or brain injury.

I have a history or symptoms of long QT syndrome.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral Bionetide or placebo twice daily for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Bionetide

Trial Overview The trial is testing Bionetide, an oral medication, against a placebo to see if it helps improve symptoms of Rett Syndrome. It also looks at whether Bionetide can help these individuals communicate better.

How Is the Trial Designed?

Group I: Bionetide Experimental Treatment 1 Intervention

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)

Group II: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Biomed Industries, Inc.

Lead Sponsor

Trials

9

Recruited

5,100+

Published Research Related to This Trial

The expert panel developed comprehensive clinical guidelines for managing bone health in individuals with Rett syndrome, emphasizing the importance of assessing fracture history, mutation type, medication, and dietary intake.

The guidelines recommend baseline bone density assessments and suggest that increasing physical activity and supplementing with calcium and vitamin D are key initial strategies, with bisphosphonates recommended for those diagnosed with osteoporosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Guidelines for Management of Bone Health in Rett Syndrome Based on Expert Consensus and Available Evidence. Jefferson, A., Leonard, H., Siafarikas, A., et al.[2020]

Trofinetide was found to be safe and well-tolerated in a phase 2 study involving 82 female children and adolescents with Rett syndrome, with no significant adverse effects reported across all dose levels.

At a dosage of 200 mg/kg twice daily, trofinetide showed statistically significant improvements in key symptoms of Rett syndrome compared to placebo, indicating its potential as a treatment option for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome. Glaze, DG., Neul, JL., Kaufmann, WE., et al.[2020]

In a pilot study involving 12 girls with Rett syndrome, Lamotrigine (LTG) showed promise in reducing seizure frequency and improving overall mood and alertness in some participants, indicating potential efficacy as an adjunct treatment.

The treatment was generally well-tolerated, with only mild side effects like rash and tremor reported, suggesting that LTG could be a safe option for managing symptoms in girls with Rett syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lamotrigine in Rett syndrome: treatment experience from a pilot study. Stenbom, Y., Tonnby, B., Hagberg, B.[2019]

In a double-blind crossover study involving 30 girls with Rett syndrome, mecasermin (rhIGF-1) did not show significant improvement in symptoms compared to placebo, and some measures indicated worsening of symptoms.

While the treatment was generally safe with mostly mild to moderate adverse events, serious adverse events were reported, and EEG parameters also showed deterioration, suggesting caution in its use for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome. O'Leary, HM., Kaufmann, WE., Barnes, KV., et al.[2019]

A comprehensive set of recommendations for managing poor growth and weight gain in Rett syndrome was developed through expert consensus, emphasizing the importance of addressing feeding difficulties and nutritional needs.

A target body mass index around the 25th centile is suggested for clinical practice, and gastrostomy may be necessary for patients with extremely poor growth or feeding challenges.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment and management of nutrition and growth in Rett syndrome. Leonard, H., Ravikumara, M., Baikie, G., et al.[2021]

Rett syndrome (RTT) is a complex neurodevelopmental disorder primarily affecting females, characterized by developmental regression and severe disabilities, with no known treatments beyond symptom management.

Future drug trials for RTT should focus on neurobiological mechanisms and utilize innovative strategies, such as targeting growth factors and glutamate modulators, while ensuring that outcome measures are robust to effectively assess safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neurobiologically-based treatments in Rett syndrome: opportunities and challenges. Kaufmann, WE., Stallworth, JL., Everman, DB., et al.[2020]

The study involving 6 girls with Rett syndrome showed that fingolimod treatment was safe, with no serious adverse events reported during the 12-month treatment period.

However, the study did not find evidence that fingolimod improved clinical outcomes or laboratory measures, indicating that further research is needed to explore its efficacy in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod in children with Rett syndrome: the FINGORETT study. Naegelin, Y., Kuhle, J., Schädelin, S., et al.[2021]

In a study of 165 Italian patients with Rett Syndrome, sodium valproate (VPA) and carbamazepine (CBZ) were found to be equally effective as first-line treatments for seizures that began at the typical age of 4-5 years, while lamotrigine (LTG) was effective for later-onset epilepsy.

The overall frequency of side effects from these antiepileptic drugs was low, with restlessness and somnolence being the most common, suggesting that these medications are relatively safe for use in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antiepileptic drugs in Rett Syndrome. Pintaudi, M., Calevo, MG., Vignoli, A., et al.[2018]

Trofinetide is the first FDA-approved drug for treating Rett Syndrome in children aged 2 years and older, marking a significant milestone in the management of this condition.

Clinical studies have shown that Trofinetide significantly improves behavioral scores in children with Rett Syndrome, indicating its efficacy as a new treatment option, although further research is needed to evaluate potential side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Trofinetide-a new chapter in rett syndrome's treatment. Furqan, M.[2023]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Assessment and management of nutrition and growth in Rett syndrome. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Trofinetide-a new chapter in rett syndrome's treatment. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Clinical Guidelines for Management of Bone Health in Rett Syndrome Based on Expert Consensus and Available Evidence. [2020]

5. England pubmed.ncbi.nlm.nih.gov

Neurobiologically-based treatments in Rett syndrome: opportunities and challenges. [2020]

6. Germany pubmed.ncbi.nlm.nih.gov

Lamotrigine in Rett syndrome: treatment experience from a pilot study. [2019]

7. England pubmed.ncbi.nlm.nih.gov

Fingolimod in children with Rett syndrome: the FINGORETT study. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome. [2020]

9. England pubmed.ncbi.nlm.nih.gov

Antiepileptic drugs in Rett Syndrome. [2018]