AJ201 for Kennedy's Disease

No longer recruiting at 7 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new drug, AJ201, for treating Spinal and Bulbar Muscular Atrophy (SBMA), a condition that causes muscle weakness. Participants will receive either AJ201 or a placebo (a non-active substance) to help researchers understand the drug's impact. The study seeks adult males diagnosed with SBMA who experience muscle weakness but can still walk a certain distance, even with assistance. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that AJ201 is undergoing safety testing in people with spinal and bulbar muscular atrophy (SBMA). This drug targets a specific muscle issue caused by the condition. Although AJ201 is still in early trials, its progression to this phase indicates some initial safety in humans.

While detailed safety data remains unavailable, ongoing research examines how well participants tolerate the drug and identifies any side effects. The trial includes a placebo, a substance with no active drug, to compare results and better understand AJ201's effects. The study is double-blind, so neither participants nor researchers know who receives the drug or the placebo, ensuring unbiased results. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Kennedy's Disease?

Unlike current treatments for Kennedy's Disease, which mainly focus on managing symptoms, AJ201 is an experimental drug designed to directly target the disease's underlying mechanisms. Researchers are excited about AJ201 because it works by modulating specific cellular pathways that could potentially slow disease progression, offering a more targeted approach than existing therapies. This could mean more effective treatment with fewer side effects, providing new hope for those affected by this condition.

What evidence suggests that AJ201 might be an effective treatment for Kennedy's Disease?

Research has shown that AJ201, which participants in this trial may receive, may help treat Spinal and Bulbar Muscular Atrophy (SBMA). In earlier studies, patients taking AJ201 walked an average of 17.6 meters farther in a 6-minute walk test. They also showed a 0.8-point improvement on a scale measuring muscle function. Noticeable improvements in physical and muscle function appeared after just 12 weeks of treatment. Additionally, 53% of patients taking AJ201 experienced a significant drop in levels of a harmful protein, compared to only 17% of those taking a placebo. These findings suggest that AJ201 could effectively improve SBMA symptoms. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. clinicaltrialsarena.com clinicaltrialsarena.com/news/annji-outcomes-trial/

AnnJi reports outcomes from spinal bulbar MA therapy trial This included an average gain of 17.6m in the 6 Minute Walk Test (6MWT) and a 0.8-point increase observed in the SBMA Functional Rating Scale.

2. ajpharm.com ajpharm.com/2025/05/21/annji-pharmaceutical-company-announces-positive-phase-1-2a-resultsfor-aj201-in-spinal-and-bulbar-muscular-atrophy-sbma-patients/

AnnJi Pharmaceutical News After 12 weeks of oral treatment, AJ201 recipients showed clinically meaningful improvements in physical and muscle function compared to placebo.

3. kennedysdisease.org kennedysdisease.org/research/overview.html/article/2025/05/21/first-in-patient-trial-of-a-drug-to-treat-kennedy-s-disease

First-In-Patient Trial of a Drug to Treat Kennedy's Disease AnnJi Pharmaceutical Company Announces Positive Phase 1/2a Results for AJ201 in Spinal and Bulbar Muscular Atrophy (SBMA) Patients.

4. practicalneurology.com practicalneurology.com/news/fast-track-designation-granted-to-investigational-treatment-for-kennedy-disease/2484232/

Fast Track Designation Granted to Investigational Treatment ... Additionally, 53% of participants treated with AJ201 vs 17% of those treated with placebo showed a >50% reduction in mutant AR protein levels ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05517603

NCT05517603 | A Study to Evaluate Safety, Tolerability ... This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in ...

6. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/protocoldetails.aspx?id=001505-N&&query=

NIH Clinical Center: Search the Studies A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, First In-Patient Study of AJ201 to Evaluate Safety, Tolerability, Pharmacokinetics, and ...

7. ajpharm.com ajpharm.com/aj201/

AJ201 AJ201 is a new chemical entity being developed for the treatment of spinal and bulbar muscular atrophy (SBMA), a neurodegenerative disease.

8. ir.avenuetx.com ir.avenuetx.com/news-events/press-releases/detail/92/avenue-therapeutics-announces-last-patient-last-visit-in

Press Releases AJ201 is a novel, first-in-class asset in development for the treatment of spinal and bulbar muscular atrophy. It was designed to modify SBMA ...