Bethesda, MDNCT04944940Now EnrollingIRB Ready

Spinal and Bulbar Muscular Atrophy Clinical Trial in Bethesda, MD

Access cutting-edge spinal and bulbar muscular atrophy treatment through this clinical trial at a research site in Bethesda. Study-provided care at no cost to qualified participants.

Sponsored by National Institute of Neurological Disorders and Stroke (NINDS)

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Expert Care in Bethesda

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IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related spinal and bulbar muscular atrophy treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Bethesda

    Convenient for MD residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Bethesda site if eligible
  4. 4Begin participation

About This Spinal and Bulbar Muscular Atrophy Study in Bethesda

Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors. Researchers want to learn more about the effects of SBMA. Objective: To identify measurements that change over time in SBMA, including tests of muscle strength and function, as well as measurements of muscle and fat size. Eligibility: Men over the age of 18 both with and without a history of SBMA. Design: Participants will have a medical history, physical exam, and blood and urine tests. They will have neuromuscular ultrasound. They will have a lumbar puncture to obtain spinal fluid. For this, a needle will be inserted into the spinal canal in the lower back. Participants will have muscle strength and function tests. These tests may include pushing, pulling, rising from a chair and sitting back down, and/or walking. During these tests, they may wear an accelerometer (activity tracker) on their wrist. Participants will get an activity tracker to wear on their wrist for 10 days at home every 3 months. Participants with SBMA will also have lower limb magnetic resonance imaging (MRI) and optional whole-body MRI. They will have lung function tests. They will have speech and swallow tests. They will complete questionnaires. They may have optional body scans to measure bone density and lean body mass. They may have optional muscle biopsies. For biopsies, a needle will be used to take a small piece of muscle from the leg. Participants with SBMA will have 5 study visits over 2 years (every 6 months). Participants without SBMA will have 1 study visit.

Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)

Who Can Participate

Inclusion Criteria

Some restrictions are placed on participation in the study because we aim to identify disease biomarkers specific to those with early to intermediate stages of disease who would be potential candidates for future therapeutic studies. In order to be eligible to participate in the SBMA cohort, an individual must meet all of the following criteria:
Stated willingness to comply with all study procedures and availability for the duration of the study
Male, above the age of 18 years
Genetically confirmed SBMA
Ability of subject to understand and the willingness to sign a written informed consent document
Ability of subject to travel to the NIH Clinical Center. Note: an SBMA patient who meets both of the additional following criteria will be offered an optional whole body MRI at subsequent follow-up visits:
Spinal bulbar muscular atrophy functional rating of \< 50 (and \> 35).
On initial whole body MRI, subject has evidence of muscle fat replacement such that the total volume of disease affected muscles (i.e., muscles with at least 10% muscle fat infiltration and no more than 50% muscle fat fraction) is at least:
500ml if only 1 muscle is eligible or
250ml if more than one muscle meets the criteria In order to be eligible to participate in this study in the Healthy Control cohort, an individual must meet all of the following criteria:
Stated willingness to comply with all study procedures and availability to travel to the NIH for the duration of the study
Male, above the age of 18 years
No history of SBMA or other neuromuscular disorder
No history of facial palsy
Ability of subject to understand and the willingness to sign a written informed consent document
Ability of subject to travel to the NIH Clinical Center.

Exclusion Criteria

SBMA is a disease that affects males and manifests in adulthood. Thus, woman and children are not included in this study. This study will not include individuals who lack consent capacity. An SBMA patient who meets any of the following criteria will be excluded from participation in this study:
Contraindications to MRI such as a contraindicated non-removable metal device (i.e., pacemaker, defibrillator, insulin pump, metal clips, non-removable jewelry) or claustrophobia.
Non ambulatory
Use of androgen reducing agents within the past two years Note: An SBMA patient who meets any of the following criteria will be excluded from the lumbar puncture procedure:
PT/PTT values that are prolonged greater than or equal to 3 seconds from the upper limit of normal (including treatment with oral and parenteral anticoagulants)
INR greater than or equal to 1.5, thrombocytopenia (\<70,000), or abnormal bleeding time or platelet dysfunction
History of a bleeding disorder
Use of anticoagulants Note: An SBMA patient who meets any of the following criteria will be excluded from the muscle biopsy procedure:
Advanced wasting of tibialis anterior that precludes needle muscle biopsy (in order to ensure that a sample taken would be of muscle and not just fat and fascia)
Use of aspirin or non-steroidal anti-inflammatory agents 3 days prior to the procedure Note: An SBMA patient who meets any of the following criteria will be excluded from the whole body MRI:
Patient has a history of prior treatment with androgen reducing agents including LHRH agonists or antagonists, androgen receptor antagonists and selective androgen receptor modifiers.
Patient is unable to complete the study assessments of QMT or timed walk tests.
Patient anticipates making major lifestyle changes during the observation period relating to diet and exercise. A Healthy Control participant who meets any of the following criteria will be excluded from the study:
PT/PTT values that are prolonged greater than or equal to 3 seconds from the upper limit of normal (including treatment with oral and parenteral anticoagulants)
INR greater than or equal to 1.5, thrombocytopenia (\<70,000), or abnormal bleeding time or platelet dysfunction
History of a bleeding disorder
Use of anticoagulants

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Bethesda?

Yes, this clinical trial (NCT04944940) has an active research site in Bethesda, MD that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Spinal and Bulbar Muscular Atrophy Treatment Options in Bethesda, MD

If you're searching for spinal and bulbar muscular atrophy treatment options in Bethesda, MD, this clinical trial (NCT04944940) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Bethesda research site is actively enrolling participants for this clinical trial. You'll receive care from experienced spinal and bulbar muscular atrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all spinal and bulbar muscular atrophy clinical trials near you to find additional studies recruiting in your area.

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