What side effects are associated with this type of intervention?

Common treatments for Kennedy's Disease (SBMA) include androgen receptor antagonists and supportive therapies. Androgen receptor antagonists can cause side effects such as gynecomastia, hot flashes, decreased libido, and liver function abnormalities. Supportive therapies, which may include physical therapy and medications for symptom management, generally have fewer side effects but can include fatigue, gastrointestinal issues, and muscle cramps. The investigational drug AJ201, being studied for its potential to modify disease progression, may have its own unique side effects that are currently being evaluated in clinical trials.

What prior FDA approvals exist?

As of now, there are no FDA-approved drugs specifically for the treatment of Kennedy's Disease (Spinal and Bulbar Muscular Atrophy, SBMA). Current management primarily focuses on symptomatic relief and supportive care. Investigational drugs like AJ201 are being studied to potentially modify disease progression in SBMA, but no similar treatments have received FDA approval to date.

What other types of research exist?

Promising alternatives to AJ201 for Kennedy's Disease patients include investigational drugs that target androgen receptor pathways, gene therapy approaches, and other small molecule inhibitors that aim to modify disease progression. Specific examples include selective androgen receptor modulators (SARMs), antisense oligonucleotides targeting the androgen receptor gene, and potential gene editing techniques such as CRISPR/Cas9. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on the latest clinical trial data and individual health status.

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AJ201 for Kennedy's Disease

Phase 1 & 2

Waitlist Available

Research Sponsored by AnnJi Pharmaceutical Co., Ltd.

Eligibility Criteria Checklist

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

Must have

Able to complete 2MWT with or without the aid of an assisted device at screening

Adult males aged 18 or greater with a confirmed genetic diagnosis (confirmed CAG repeat expansion in the AR gene of at least 36 repeat) of SBMA and clinical diagnosis of symptomatic muscle weakness

Must not have

Known history of allergic reactions to curcumin analogs or excipients in the study drug formulation

Use of drugs known to affect muscle metabolism within the previous 1 month prior to the first dosing, including (but not limited to) systemic corticosteroids (>10 mg/day prednisone or equivalent), androgens, or androgen reducing agents, systemic beta agonists or beta blockers, and relevant herbal, or nutraceutical products. For subjects using systemic corticosteroids (≤10 mg/day or equivalent), they should be on stable dose for the previous 3 months prior to first dosing

Timeline

Screening 3 weeks

Treatment Varies

Follow Up visit 3/week 2 and visit 4/week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose.

Summary

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).

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Since this is an early-phase trial for AJ201, specific side effects aren't listed but generally could include reactions at injection sites, gastrointestinal issues, fatigue or allergic responses. Safety monitoring will identify side effects during the trial.

Eligibility Criteria

Inclusion Criteria

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

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I can walk with or without help during the test.

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I am a man over 18 with SBMA, confirmed by a genetic test showing at least 36 CAG repeats in the AR gene, and I have muscle weakness.

Exclusion Criteria

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

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I am allergic to curcumin or ingredients in the study medication.

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

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I have a serious heart condition or have had a stroke.

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I cannot walk by myself.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ visit 3/week 2 and visit 4/week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~visit 3/week 2 and visit 4/week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose.

This trial's timeline: 3 weeks for screening, Varies for treatment, and visit 3/week 2 and visit 4/week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose. 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 and proportion of subjects with AEs including SAEs and TEAEs.

Secondary study objectives

Pharmacodynamics: Change from baseline in mutant androgen receptor protein levels in skeletal muscle in treatment vs placebo group.

Pharmacokinetics: Area Under the Curve (AUC) will be assessed

Pharmacokinetics: Maximum Plasma Concentration (Cmax) will be assessed

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: Experimental: AJ201Active Control1 Intervention

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

Group II: Placebo ComparatorPlacebo Group1 Intervention

Subjects taking placebo for 12 weeks.

0 / 6Eligibility 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

Kennedy's Disease treatments, such as the investigational drug AJ201, often focus on modulating androgen receptor activity, reducing toxic protein aggregates, and enhancing cellular stress responses. These mechanisms are crucial as they aim to slow disease progression, improve muscle function, and enhance the quality of life for patients with Spinal and Bulbar Muscular Atrophy (SBMA).

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