AJ201 for Kennedy's Disease

Recruiting at 6 trial locations

Overseen By Tahseen Mozaffar, MD

Age: 18+

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: AnnJi Pharmaceutical Co., Ltd.

Must not be taking: CYP3A4 inhibitors, Muscle metabolism drugs

Disqualifiers: Nonambulatory, Cardiovascular disease, others

Compensation: Varies

Number of Visits: 2

Duration: 12 weeks

25 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new medicine called AJ201 to see if it is safe and how it behaves in people with Spinal and Bulbar Muscular Atrophy (SBMA).

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial. Specifically, you cannot use drugs that affect muscle metabolism, certain enzyme inhibitors, or turmeric products within a specified time before starting the study. It's best to discuss your current medications with the study team to see if any changes are needed.

How does the drug AJ201 for Kennedy's Disease differ from other treatments?

AJ201 is a novel treatment for Kennedy's Disease, a condition with limited existing therapies. Unlike other treatments, AJ201 is being specifically tested in a clinical trial setting, which may offer new insights into its effectiveness and safety compared to the placebo (dummy treatment). ^{1 2 3 4 5}

Eligibility Criteria

Adult males over 18 with Spinal and Bulbar Muscular Atrophy (SBMA) who can walk, have moderate physical performance, agree to use two forms of birth control, and can undergo study procedures like blood draws and muscle biopsies. Excluded are those nonambulatory, allergic to curcumin analogs or excipients in AJ201, with significant cardiovascular or cerebrovascular disease, recent use of investigational products or drugs affecting muscle metabolism.

Inclusion Criteria

Able to communicate well with the Investigator, to understand, and comply with the requirements of the study

I can walk with or without help during the test.

Your physical performance score is 26 or higher, which means you have moderate to high physical abilities.

Exclusion Criteria

I haven't taken any experimental drugs recently, except for approved COVID-19 vaccines.

I haven't taken any muscle-affecting drugs or high-dose steroids in the last month.

Your heart's electrical activity, as shown in the ECG test, is not normal and could be risky for you to participate in the study. For example, if your ECG shows changes similar to those seen in SBMA patients in Italy and Japan.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either AJ201 600mg/day or placebo for 12 weeks

12 weeks

Multiple visits including Visit 2 (Week 1), Visit 3 (Week 2), Visit 4 (Week 6), and Visit 5 (Week 12)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• AJ201
• Placebo

Trial Overview The trial is testing the safety and effects of a new drug called AJ201 compared to a placebo in men with SBMA. It's randomized and double-blind meaning neither the participants nor the researchers know who gets the real drug versus placebo until after results are collected.

Participant Groups

Group I: Experimental: AJ201 Active Control 1 Intervention

Subjects taking active drug AJ201 600mg/day for 12 weeks.

Group II: Placebo Comparator Placebo Group 1 Intervention

Subjects taking placebo for 12 weeks.

Who Is Running the Clinical Trial?

AnnJi Pharmaceutical Co., Ltd.

Lead Sponsor

Trials

3

Recruited

150+

Findings from Research

Multiple system atrophy (MSA) is a severe neurodegenerative disorder with a mean survival of only 9 years, characterized by autonomic failure and either parkinsonism or cerebellar ataxia, with limited treatment options available.

While pharmacological treatments for motor symptoms are largely ineffective, early identification and treatment of autonomic and urogenital symptoms can be beneficial, and ongoing multicenter trials are exploring potential neuroprotective therapies like riluzole and human recombinant growth hormone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multiple system atrophy: an update. Wenning, GK., Geser, F., Stampfer-Kountchev, M., et al.[2013]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Placebo response in degenerative cerebellar ataxias: a descriptive review of randomized, placebo-controlled trials. [2022]

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

Treatment of cerebellar ataxia with 5-HT1A agonist. [2018]

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

Planning Future Clinical Trials for Machado-Joseph Disease. [2018]

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

Clinical trials: past, current, and future for atypical Parkinsonian syndromes. [2021]

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

Multiple system atrophy: an update. [2013]