Pittsburgh, PANCT06773026Now EnrollingIRB Ready

Medium-chain Acyl-CoA Dehydrogenase Deficiency Clinical Trial in Pittsburgh, PA

Access cutting-edge medium-chain acyl-coa dehydrogenase deficiency treatment through this clinical trial at a research site in Pittsburgh. Study-provided care at no cost to qualified participants.

Sponsored by Jerry Vockley, MD, PhD

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Expert Care in Pittsburgh

Access medium-chain acyl-coa dehydrogenase deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related medium-chain acyl-coa dehydrogenase deficiency treatment provided free

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Check if you qualify for this medium-chain acyl-coa dehydrogenase deficiency clinical trial in Pittsburgh, PA

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Why Participate?

  • No-Cost Study Care

  • Local to Pittsburgh

    Convenient for PA 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 Pittsburgh site if eligible
  4. 4Begin participation

About This Medium-chain Acyl-CoA Dehydrogenase Deficiency Study in Pittsburgh

This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.

Sponsor: Jerry Vockley, MD, PhD

Who Can Participate

Inclusion Criteria

A diagnosis of MCADD and molecular confirmation of at least one copy of the common c.985A\>G mutation.
≥4 years of age
Able to perform and comply with study activities placement of a continuous glucose monitor, IV catheter, and all blood draws.
Negative pregnancy test for all female subjects of childbearing age.
Signed informed consent by the subject or parent/guardian of minors.
All females of childbearing age and all sexually active males must agree to use an acceptable method of contraception throughout the study. Appropriate contraceptive methods include hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier methods. Abstinence is an acceptable form of birth control, though appropriate contraception must be used if the subject becomes sexually active.
Willing and able to adhere to requirements for maintaining continuous glucose monitoring.

Exclusion Criteria

Use of any investigational drug within 30 days of Day 1.
Active infection (viral or bacterial) or any other intercurrent condition as reported by the subject or noted on physical exam at screening.
Any clinical or laboratory abnormality of Grade 3 or greater severity according to the CTCAE v5.0, or Grade 3 elevations in liver enzymes, defined as levels 5-20 times ULN in alanine aminotransferase (ALT/SGPT), or aspartate aminotransferase (AST/SGOT) in a clinically stable subject.
Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at increased risk by participating in this study.
Use of any medication known to significantly affect renal clearance (e.g., probenecid) or to increase protein catabolism (e.g., corticosteroids), or other medication known to increase ammonia levels (e.g., valproic acid or haloperidol), within the 48 hours prior to Day 1 and throughout the study.
Subjects with renal insufficiency will be excluded from the study. Cutoff eGFR \<60 mL/min/1.73m2 (GFR categories G3a-G5) will be used as measure of renal insufficiency.
Use of sodium benzoate within one week of Day 1.
Known hypersensitivity to PAA or PBA.
Breastfeeding or lactating females.
Subjects at risk of hypokalemia due to pre-existing diagnosis or on medications that can cause hypokalemia.
Subjects with type 1 or type 2 diabetes, or who take medications as part of their routine care that can cause hypoglycemia
A positive urine drug screen at screening for drugs without a prescription
Subjects who are taking medications in the antimetabolite drug class (e.g., hydroxyurea, 5-fluorouracil (5-FU), methotrexate) will be excluded; these medications can interfere with the DEXCOM sensor and cause inaccurate glucose readings

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 Pittsburgh?

Yes, this clinical trial (NCT06773026) has an active research site in Pittsburgh, PA 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.

Medium-chain Acyl-CoA Dehydrogenase Deficiency Treatment Options in Pittsburgh, PA

If you're searching for medium-chain acyl-coa dehydrogenase deficiency treatment options in Pittsburgh, PA, this clinical trial (NCT06773026) 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 Pittsburgh research site is actively enrolling participants for this clinical trial. You'll receive care from experienced medium-chain acyl-coa dehydrogenase deficiency 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 medium-chain acyl-coa dehydrogenase deficiency clinical trials near you to find additional studies recruiting in your area.

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