What side effects are associated with this type of intervention?

The most common side effects of Botox (botulinum toxin type A) treatments include injection site pain, stiffness, cramping, hematoma, and paresthesias. For treatments targeting limb tremor, dose-dependent hand weakness is frequently observed. In the case of head tremor, neck weakness and post-injection pain are common. When used for chronic migraines, the most reported adverse effects are neck pain and musculoskeletal pain.

What prior FDA approvals exist?

Botox (botulinum toxin type A) has received FDA approval for several treatments, including chronic migraine prevention, spasticity in cerebral palsy, and primary axillary hyperhidrosis. Its mechanism of action involves blocking acetylcholine release at the neuromuscular junction, leading to temporary muscle paralysis or reduced gland activity. These approvals highlight its efficacy in managing conditions characterized by excessive muscle activity or glandular secretion.

What other types of research exist?

Promising novel alternatives to Botox for patients considering treatment in 2024 include: 1) Bevacizumab, which has shown efficacy in reducing vestibular schwannomas and improving hearing in NF2-related conditions. 2) Antisense oligonucleotide therapies, such as Tofersen, which are being developed for neurodegenerative diseases like ALS. 3) Levodopa, which has shown potential in improving vision in albinism. These alternatives offer new mechanisms of action and potential benefits for conditions traditionally managed with Botox.

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Other

G03-52-01 Safety and Efficacy Study for Healthy Subjects

Phase 2

Waitlist Available

Research Sponsored by Resilience Government Services, Inc.

Eligibility Criteria Checklist

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

Must have

Able and willing to comply and be available for all protocol procedures and follow-up for the duration of the study.

Assessed by the Investigator to be a healthy male or healthy, non-pregnant, non-lactating female between the ages of 18 and 65 inclusive on the day of dosing.

Must not have

Any previous injection or any planned injection within 4 months after enrollment of botulinum toxin for cosmetic reasons, spastic dysphonia, torticollis, or any other reason

Female subjects that are pregnant or breastfeeding or intending to become pregnant within the projected duration of the trial starting from the Screening visit until last visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 240 days

Summary

This trial is testing a medication called G03-52-01 given as an injection into the muscle. It involves adult participants and will assess the effects of the medication over time.

Who is the study for?

Healthy adults aged 18-65 with a BMI of 18.5 to 35 kg/m2 can join this trial. Women must not be pregnant or breastfeeding and should use effective contraception if of childbearing potential. Participants must have normal lab results, no drug abuse, and agree to avoid vigorous activity around dosing days.

What is being tested?

The study is testing G03-52-01 against a placebo in healthy volunteers. It's a Phase 2 trial where participants are randomly assigned to receive either the test drug or an inactive substance (placebo), but neither they nor the researchers know who gets which one.

What are the potential side effects?

While specific side effects for G03-52-01 aren't listed, common reactions in trials may include injection site discomfort, headache, fatigue, or flu-like symptoms. Placebos typically have no active ingredients but can cause similar mild reactions due to psychological factors.

Eligibility Criteria

Inclusion Criteria

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

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I can attend all study visits and follow the study plan.

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I am a healthy adult between 18 and 65 years old and not pregnant or breastfeeding.

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I am a healthy adult between 18 and 65 years old and not pregnant or breastfeeding if female.

Exclusion Criteria

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

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I have not had, nor plan to have, any botulinum toxin injections for any reason in the 4 months around joining.

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I am not pregnant, breastfeeding, nor planning to become pregnant during the trial.

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I have not received any antibody treatments or blood transfusions in the last 6 months.

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I haven't taken any forbidden medications in the last 28 days and won't during the study.

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I have been exposed to or treated with botulinum toxin or related antibodies.

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I have not had monoclonal antibody treatment in the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 240 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~240 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 240 days for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Occurrence of AEs following administration of G03-52-01 to the final visit (Cohort 4)

Occurrence of AEs following administration of G03-52-01 to the final visit (Cohorts 1-3)

Occurrence of SAEs following administration of G03-52-01 to the final visit (Cohort 4)

+10 more

Secondary study objectives

Descriptive statistics of selected ADA at all timepoints tested (Cohorts 1-3)

Descriptive statistics of selected ADA at all timepoints tested (Cohorts 4)

Descriptive statistics of selected PD at all timepoints tested (Cohort 4)

+4 more

Other study objectives

Descriptive statistics of selected PK parameters at all time points tested of the two lots

Side effects data

From 2023 Phase 1 trial • 40 Patients • NCT04171115

25%

Headache

13%

Diastolic hypertension

13%

Light headedness

13%

Gastroenteritis

13%

Dysuria

13%

Limbs stiffness

13%

Injection site bruising

100%

80%

60%

40%

20%

Study treatment Arm

100 mg of G03-52-01

10mg of G03-52-01

25mg of G03-52-01

Pooled Placebo

50 mg of G03-52-01

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: 50 mg dose of G03-52-01Experimental Treatment1 Intervention

150 subjects randomized to 50 mg of G03-52-01

Group II: 100 mg dose of G03-52-01 or PlaceboExperimental Treatment2 Interventions

250 subjects randomized to 100 mg of G03-52-01 or placebo

Group III: 100 mg dose of G03-52-01Experimental Treatment1 Intervention

150 subjects randomized to 100 mg of G03-52-01

Group IV: PlaceboPlacebo Group1 Intervention

75 subjects randomized to placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

G03-52-01

2020

Completed Phase 1

~40

0 / 9Eligibility 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

Botox (botulinum toxin type A) works by blocking the release of acetylcholine at the neuromuscular junction, preventing muscle contraction. This mechanism helps alleviate symptoms in conditions like myofascial pain, essential tremor, dystonia, and chronic migraines by reducing muscle activity. For patients, understanding this mechanism is important as it explains the potential benefits and side effects, such as muscle weakness or injection site pain, associated with Botox treatments.

Local treatment of dystonia and spasticity with injections of botulinum-A toxin.Botulinum toxin treatment of myofascial pain: a critical review of the literature.