AzTrials
AzTrials

Spinal and Bulbar Muscular AtrophyPhase 1/2NCT05517603

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

A Phase 1/2 study testing AJ201 in people with Spinal and Bulbar Muscular Atrophy and Kennedy's Disease, run by AnnJi Pharmaceutical Co., Ltd. at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA). Read more about this study

Who is it for?
Ages 18 years and older, men only
What is being tested?
AJ201
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
25 participants
Where
6 sitesUnited States
Study dates
28 Feb 2023to 8 Apr 2024
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • Men only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Able to give informed consent before any assessment is performed.
  • 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.
  • Able to complete 2MWT with or without the aid of an assisted device at screening.
  • SBMAFRS score ≥26 (subjects with moderate to high physical performance) at screening.
  • Willing to participate in all aspects of study design and assessments, including blood draw and muscle biopsies.
  • Male subjects and their female spouses/partners who are of childbearing potential must agree to use highly effective contraception consisting of 2 forms of birth control (at least 1 of which must be a barrier method) starting from the first dose of the study drug and continuing throughout the study period and for 90 days after the last dose of the study drug. Male subjects should also not donate sperm during the study and for 90 days after the final administration of the study drug.
  • Able to communicate well with the Investigator, to understand, and comply with the requirements of the study.

Who cannot join

  • Nonambulatory.
  • Contraindications to MRI such as a contraindicated nonremovable metal device (ie, pacemaker, defibrillator, insulin pump, metal clips, nonremovable jewelry) or claustrophobia.
  • Use of other investigational products within 30 days, or within 5 half-lives, whichever is longer, prior to the first dosing, or until the expected PD effect has returned to baseline, whichever is longer. Approved COVID-19 vaccines are not considered investigational treatments and are allowed prior to, during, and after the study.
  • 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.
  • Known history of allergic reactions to curcumin analogs or excipients in the study drug formulation.
  • Known history of clinically significant cardiovascular disease (including uncontrolled hypertension, ischemic heart disease [eg, myocardial infarction, angina, abnormal coronary arteriography or cardiac stress testing/imaging]), heart failure or left ventricle dysfunction of New York Heart Association Classification III-IV, or clinically significant cerebrovascular disease (stroke or transient ischemic attacks).
Show all 23Show fewer
  • An abnormal ECG at screening visit which is judged to be clinically relevant and represents an unacceptable risk for study participation by the Investigator. An example is Brugada-like ECG changes which have been reported in SBMA patients in Italy and Japan.
  • Any surgical or medical condition which may jeopardize the subject in case of participation in the study. The Investigator should make this determination in consideration of the subject's medical history and/or clinical or laboratory evidence of any of the following during screening:
  • Liver disease or liver injury as indicated by abnormal liver function tests such as AST, ALT, gamma-glutamyl transferase (GGT), alkaline phosphatase (ALP), or serum bilirubin in the presence of normal serum creatine kinase (CK).
  • Significant swallowing dysfunction, which may increase the risk of accidental choking and aspiration pneumonia.
  • Subjects with renal impairment defined as a creatinine clearance of <90 mL/min at screening. (Creatinine Clearance = [140 - age in years] *weight in kg]/[72*serum Cr(mg/dL)]).
  • History of active tuberculosis or exposure to endemic areas within 8 weeks prior to QuantiFERON®-TB testing performed at screening.
  • Positive QuantiFERON®-TB indicating possible tuberculosis infection.
  • History of immunodeficiency diseases, including a positive HIV test result at screening.
  • A positive hepatitis B surface antigen or hepatitis C test result at screening.
  • Subjects with known bleeding disorders, or who are under treatment with anticoagulants or with a platelet count <50,000 (due to the increased risk of bleeding during muscle biopsy procedure).
  • History of drug or alcohol abuse within the 12 months prior to the first dosing, or evidence of such abuse as indicated by the laboratory assays conducted during screening.
  • The Investigator should be guided by the following criteria during screening: a. Any single laboratory parameter may not exceed 3 times the upper limit of normal (ULN). A single parameter elevated up to and including 3 times the ULN should be re-checked once more as soon as possible, and in all cases, at least prior to enrollment/randomization, to rule out lab error. For abnormal liver function tests, in the presence of elevated serum CK levels, ALT, or AST, up to 5 times the ULN are acceptable if other liver tests are normal. b. If the total bilirubin concentration is increased above 1.5 times the ULN, total bilirubin should be differentiated into the direct and indirect reacting bilirubin. In any case, serum indirect bilirubin should not exceed the value of 1.6 mg/dL (27 mol/L).
  • Use of drugs that are inhibitors of CYP3A4, 2B6, or 2C19 within 2 weeks prior to the first dosing and during the study.
  • Use of drugs that are substrates of MATE1 or MATE2-K transporters within 2 weeks prior to the first dosing and during the study.
  • Use of turmeric or products containing curcumin within 2 weeks prior to the first dosing and during the study.
  • Use of aspirin or nonsteroidal anti-inflammatory agents within 3 days prior to the baseline visit (due to the increased risk of bleeding during muscle biopsy procedure).
  • Any reason that, in the opinion of the Investigator, would prevent the subject from participating in the study.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • National Institutes of Health

    Bethesda, Maryland, 20814

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • University of California, Irvine

    Orange, California, 92868

  • Stanford University

    Palo Alto, California, 94304

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Washington University in St. Louis

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, First-In-Patient Study Of AJ201 To Evaluate Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics In Adults With Spinal And Bulbar Muscular Atrophy (SBMA)

This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).

Being tested
AJ201, Placebo
Medical subject headings
Bulbo-Spinal Atrophy, X-Linked

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AJ201Drug

    Also called JM17

    Administered orally

  • PlaceboDrug

    Administered orally

Study groups in NCT05517603
Study groupRoleWhat they receive
Experimental: AJ201Active comparatorSubjects taking active drug AJ201 600mg/day for 12 weeks.
Receives: Drug: AJ201
Placebo ComparatorPlacebo comparatorSubjects taking placebo for 12 weeks.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and proportion of subjects with AEs including SAEs and TEAEs.

    Safety will be monitored throughout the study.

    Measured: 12 weeks

Secondary outcomes (3)

  1. Pharmacokinetics: Maximum Plasma Concentration (Cmax) will be assessed

    Blood sampling will be collected at Visit 3 and Visit 4.

    Measured: 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.

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

    Blood sampling will be collected at Visit 3 and Visit 4.

    Measured: 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.

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

    Samples will be collected at Visit 2 and Visit 5.

    Measured: Visit 2/Week 1 and Visit 5/Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Aug 2022
  2. Study start28 Feb 2023
  3. Primary completion8 Apr 2024
  4. Study completion8 Apr 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05517603
Sponsor's protocol ID
JM17-201-201
Status last verified
May 2024
First submitted
23 Aug 2022
Last updated
15 Aug 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AJ201 in people with Spinal and Bulbar Muscular Atrophy and Kennedy's Disease, run by AnnJi Pharmaceutical Co., Ltd. at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and proportion of subjects with AEs including SAEs and TEAEs. (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05517603.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in