10 Participants Needed

SPG601 for Fragile X Syndrome

HS
CE
SR
Overseen BySarah Richter
Age: 18 - 65
Sex: Male
Trial Phase: Phase 2
Sponsor: Spinogenix
Must be taking: Psychotropic drugs
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.

Do I have to stop taking my current medications?

The trial requires that you have stable dosing of psychotropic drugs for at least 4 weeks before participating. Other medications are not specifically mentioned, so the protocol does not specify if you need to stop taking them.

What data supports the idea that SPG601 for Fragile X Syndrome is an effective drug?

The available research does not provide specific data on SPG601 for Fragile X Syndrome. However, it mentions other treatments like eFT508, which improved various symptoms in mice, and STX209, which corrected synaptic issues in a mouse model. Mavoglurant did not show significant improvement in human trials. Without direct data on SPG601, we cannot confirm its effectiveness compared to these treatments.12345

What safety data is available for SPG601 treatment?

The provided research does not contain specific safety data for SPG601 or its other names. The studies focus on different compounds, such as sodium 2,2 dimethylbutyrate (SDMB) and HQK-1001, which are not identified as SPG601. Therefore, no relevant safety data for SPG601 is available in the provided research.678910

Is the drug SPG601 a promising treatment for Fragile X Syndrome?

SPG601 is a promising drug for Fragile X Syndrome because it targets the GABA(B) receptor, which helps correct synaptic abnormalities. This could potentially treat core symptoms of the condition, making it a hopeful option for patients.1231112

Research Team

CE

Craig Erickson, MD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Eligibility Criteria

Adult men aged 18-45 with Fragile X Syndrome can join this trial if they're in good health, have stable use of psychotropic drugs for at least a month, and are using contraception. They need a caregiver and confirmed diagnosis via genetic testing. Those with uncontrolled seizures, recent substance abuse, or conditions affecting study completion cannot participate.

Inclusion Criteria

I am a man aged between 18 and 45.
Able and willing to provide written informed consent
I am in good health with no major medical issues.
See 5 more

Exclusion Criteria

Any physical or psychological condition that prohibits study completion
Auditory or visual impairments that cannot be corrected
History of suicidal behavior or suicidal ideation
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single oral dose of SPG601 or placebo at the first visit and cross over to receive the other product at the second visit

2 weeks
2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week

Treatment Details

Interventions

  • SPG601
Trial Overview The trial is testing SPG601's safety and effectiveness compared to a placebo in treating adult men with Fragile X Syndrome. It will also look into how the body processes the drug and its impact on the body's functions.
Participant Groups
2Treatment groups
Active Control
Placebo Group
Group I: Experimental: Active SPG601 to be administered to participants with Fragile X SyndromeActive Control1 Intervention
Participants with Fragile X Syndrome will be randomized to receive SPG601 or placebo (8 capsules of 100 mg) one time on days 1 and 8 of the study.
Group II: Placebo Comparator: Placebo comparator to be administered to participants with Fragile X SyndromePlacebo Group1 Intervention
Participants with Fragile X Syndrome will be randomized to receive SPG601 or placebo (8 capsules of 100 mg) one time on days 1 and 8 of the study.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Spinogenix

Lead Sponsor

Trials
6
Recruited
220+

Avance Clinical Pty Ltd.

Industry Sponsor

Trials
23
Recruited
3,700+

Findings from Research

eFT508, a specific MNK inhibitor, shows promise in treating Fragile X syndrome (FXS) by reducing the phosphorylation of eIF4E, which is linked to cognitive and behavioral deficits in FXS.
In mouse models, eFT508 successfully alleviated various FXS-related issues, including behavioral deficits and physical abnormalities, suggesting it could be a viable therapeutic option for reversing symptoms of FXS.
A Highly Selective MNK Inhibitor Rescues Deficits Associated with Fragile X Syndrome in Mice.Shukla, T., de la Peña, JB., Perish, JM., et al.[2023]
In two phase 2b clinical trials involving 175 adults and 139 adolescents with Fragile X syndrome, the mGluR5 antagonist mavoglurant did not show significant improvement in behavioral symptoms after 12 weeks of treatment, failing to confirm previous findings.
The safety profile of mavoglurant was consistent with earlier studies, showing few adverse events, but the trials indicated that the methylation status of the FMR1 gene did not predict treatment efficacy, suggesting a need for further research in younger populations and with different assessment markers.
Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials.Berry-Kravis, E., Des Portes, V., Hagerman, R., et al.[2016]
Pharmacologic activation of the GABA(B) receptor using STX209 (arbaclofen) successfully corrected elevated protein synthesis and synaptic abnormalities in a mouse model of Fragile X syndrome, indicating its potential as a therapeutic intervention.
Chronic administration of STX209 in juvenile Fmr1-knockout mice reduced excessive dendritic spine density without affecting normal spine density in wild-type mice, suggesting a targeted effect on the pathological features of Fragile X syndrome.
Reversal of disease-related pathologies in the fragile X mouse model by selective activation of GABAB receptors with arbaclofen.Henderson, C., Wijetunge, L., Kinoshita, MN., et al.[2022]

References

A Highly Selective MNK Inhibitor Rescues Deficits Associated with Fragile X Syndrome in Mice. [2023]
Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. [2016]
Reversal of disease-related pathologies in the fragile X mouse model by selective activation of GABAB receptors with arbaclofen. [2022]
NNZ-2566, a novel analog of (1-3) IGF-1, as a potential therapeutic agent for fragile X syndrome. [2018]
Pharmacological rescue of Ras signaling, GluA1-dependent synaptic plasticity, and learning deficits in a fragile X model. [2021]
Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteers. [2021]
Induction of fetal hemoglobin synthesis in children with sickle cell anemia on low-dose oral sodium phenylbutyrate therapy. [2019]
A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. [2022]
Short-term toxicity study of ST-20 (NSC-741804) by oral gavage in Sprague-Dawley rats. [2017]
10.United Statespubmed.ncbi.nlm.nih.gov
Efficacy of low-dose dextromethorphan in the treatment of nonketotic hyperglycinemia. [2013]
Fragile X syndrome: Are signaling lipids the missing culprits? [2017]
[Clinical practice guidelines for Fragile X syndrome]. [2022]
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