What side effects are associated with this type of intervention?

Common treatments for Cerebral Palsy, such as botulinum toxin (BoNT) injections and various oral agents for spasticity and dystonia, have known side effects. BoNT injections can lead to transient, mild complications such as pain at the injection site, muscle weakness, and flu-like symptoms. Long-term use of BoNT does not appear to prevent contractures or reduce the need for orthopedic surgery and may result in a gradual deterioration in range of motion. Oral agents for dystonia often have a narrow therapeutic window, with modest efficacy and frequent side effects that limit clinical benefit. These side effects can include sedation, dry mouth, and gastrointestinal disturbances.

What prior FDA approvals exist?

FDA-approved treatments for Cerebral Palsy primarily focus on managing spasticity and include oral antispasticity medications such as baclofen and benzodiazepines, as well as botulinum toxin injections for localized spasticity. Intrathecal baclofen pumps are also used for severe cases. These treatments have been evaluated for their safety, tolerability, and pharmacokinetic properties, similar to the ongoing studies for MTR-601. However, there is no specific mention of MTR-601 or similar drugs being FDA-approved for Cerebral Palsy at this time.

What other types of research exist?

Based on the available information, promising novel alternatives to MTR-601 for Cerebral Palsy patients could include therapies that have shown efficacy in related neurological conditions. For example, the use of positive allosteric modulators (PAMs) of mGluR4, such as VU0652957 (Valiglurax), which have been optimized for better pharmacokinetic and pharmacodynamic profiles, could be considered. Additionally, therapies targeting the PI3K/Akt/mTOR signaling pathway, which have shown potential in improving neurological outcomes, might also be promising. However, it is essential to consult with a healthcare provider to evaluate the suitability and safety of these alternatives for individual patients.

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MTR-601 for Muscle Spasticity

Q: What is the mechanism of action of this treatment?

The most common treatments for Cerebral Palsy (CP) target spasticity and include oral antispasticity medications like baclofen and benzodiazepines, which work by enhancing inhibitory neurotransmission to reduce muscle stiffness. Botulinum toxin injections block acetylcholine release at the neuromuscular junction, leading to muscle relaxation. Alcohol blocks and selective dorsal rhizotomy disrupt nerve signals to reduce spasticity. Intrathecal baclofen pumps deliver medication directly to the spinal fluid, providing targeted relief with fewer systemic side effects. Understanding the pharmacokinetics and pharmacodynamics of these treatments is crucial for optimizing their efficacy and safety, similar to the goals of the MTR-601 trial, which evaluates these parameters to ensure effective and tolerable treatment options for CP patients.

Phase 1

Recruiting

Led By Alan Hand, MD

Research Sponsored by Motric Bio

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Weight ≥50 kg and BMI <33 kg/m2

Aged 18-45 years and in good physical health based on medical history, physical examination, vital signs, laboratory, ECG, Echocardiography, muscle strength, and spirometry test values

Must not have

Use of medications/products known to alter drug absorption, metabolism, or elimination processes within 30 days prior to dosing

Active neoplastic disease or history of any neoplastic disease within 5 years of screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to day 18.

Summary

This trial tests a new drug, MTR-601, in healthy volunteers to see if it is safe. Researchers will measure how the drug behaves in the body and its effects on muscle strength.

Who is the study for?

Healthy non-smokers aged 18-45 with a BMI <33 kg/m2 can join this trial. They must not have used tobacco for 3 months, be in good health based on various medical tests, and agree to use contraception if applicable. People with cardiovascular diseases, positive drug/alcohol screens, recent surgeries that affect drug absorption or metabolism, or those who've been hospitalized recently cannot participate.

What is being tested?

The study is testing MTR-601's safety and how the body processes it under different conditions (with food or without). It will also look at how MTR-601 affects muscle strength and its accumulation in muscles through biopsies and other markers.

What are the potential side effects?

While specific side effects are not listed here, common ones may include reactions at the injection site, fatigue, headache, nausea. The trial aims to identify any potential side effects from single or multiple doses of MTR-601.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I weigh at least 50 kg and my BMI is less than 33.

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I am between 18-45 years old and in good health.

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My vital signs are normal and my lung function is good.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't taken any medications that affect drug processing in the last 30 days.

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I have not had cancer or been cancer-free for less than 5 years.

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I have not had a serious infection in the last 6 weeks.

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I have a history of heart, blood vessel, or metabolic diseases.

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I haven't used tobacco or nicotine products in the last 3 months.

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I haven't been hospitalized recently, don't have a primary muscle disorder, and my IPSS score is less than 6.

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I have had surgery on my stomach or intestines that could affect how drugs work in my body.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to day 18.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to day 18.

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to day 18. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence, severity and relatedness of Treatment-emergent adverse events (TEAEs). TEAE severity will be measured as mild, moderate or severe, and relatedness will be either related or not related.

Secondary study objectives

Maximum plasma concentration (Cmax) of MTR-601

Pharmacodynamic of MTR-601-Forearm extension and flexion strength by dynamometry

Pharmacodynamic of MTR-601-Grip strength by dynamometry

+6 more

Other study objectives

Exploratory biomarkers of MTR-601 activity.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MTR-601Experimental Treatment1 Intervention

Safety and tolerability of oral MTR-601, a highly selective fast twitch myosin 2 ATPase inhibitor in normal healthy volunteers

Group II: PlaceboPlacebo Group1 Intervention

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Cerebral Palsy (CP) target spasticity and include oral antispasticity medications like baclofen and benzodiazepines, which work by enhancing inhibitory neurotransmission to reduce muscle stiffness. Botulinum toxin injections block acetylcholine release at the neuromuscular junction, leading to muscle relaxation. Alcohol blocks and selective dorsal rhizotomy disrupt nerve signals to reduce spasticity. Intrathecal baclofen pumps deliver medication directly to the spinal fluid, providing targeted relief with fewer systemic side effects. Understanding the pharmacokinetics and pharmacodynamics of these treatments is crucial for optimizing their efficacy and safety, similar to the goals of the MTR-601 trial, which evaluates these parameters to ensure effective and tolerable treatment options for CP patients.

Functional and surgical treatments in patients with spinal muscular atrophy (SMA).