Is NCT06270316 recruiting in Birmingham, AL?

Yes, NCT06270316 is currently recruiting participants at its Birmingham, AL location. This fabry disease clinical trial is actively enrolling qualified participants.

How do I join the fabry disease clinical trial in Birmingham?

To join this clinical trial in Birmingham, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Birmingham clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Fabry Disease Clinical Trial in Birmingham, AL

Who Can Participate

Inclusion Criteria

Male of age ≥ 18 years and ≤50 years

Confirmed clinical diagnosis of classic Fabry disease (FD) defined as:

Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR

α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy \[ERT\] levels).

eGFR ≥ 40 mL/min/1.73 m2

Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment. Suboptimal response is defined as plasma lyso-Gb3 ≥ 2.3 nanograms per milliliter (ng/mL) at Screening and one or both of the following:

Persistent moderate or severe neuropathic pain (intermittent or continuous) over a period of at least 3 months prior to consent

Presence of gastrointestinal symptoms (abdominal cramping, constipation, or diarrhea), reported by the Participant as moderate or severe and that are either persistent or occurring two or more times over the 12 weeks prior to consent

Weight ≤ 120 kilograms (kg) Key

Exclusion Criteria

Any allergic hypersensitivity reaction to ERT or infusion reaction in the 12 months prior to consent that was of severity grade 3 or above based on Common Terminology Criteria for Adverse Events (CTCAE v5.0) and required emergency intervention for hypertension/hypotension to stabilize blood pressure or hypoxia OR any other life-threatening complication.

Proteinuria, with random urine protein/creatinine ratio (rUPCR) ≥1 mg/mg at Screening

Current use of chaperone therapy such as migalastat (Galafold®)

Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin

Presence of chronic, active, or latent infection with hepatitis B or C, human immunodeficiency virus (HIV), or tuberculosis (TB) as assessed at the screening visit

Active or ongoing infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, GI, endocrine (such as diabetes mellitus with poor glycemic control), pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder that could, in the opinion of the Investigator, risk the safety of the Participant, or interfere with adherence to the protocol procedures or interpretation of results

Evidence of any liver disease, including hepatitis, fibrosis, cirrhosis of the liver, neoplastic lesion, or any known medical condition that could impact the intended transduction of the vector and/or expression and activity of the protein

History of kidney transplantation or currently on hemodialysis or peritoneal dialysis

Uncontrolled hypertension, defined as systolic blood pressure \>140 millimeters of mercury (mmHg) (inclusive) and/or diastolic blood pressure outside the range of 60 to 85 mmHg (inclusive) at Screening, confirmed on at least 2 repeated measurements

Patients taking blood pressure medication to control blood pressure or proteinuria (eg, angiotensin-converting enzyme \[ACE\] inhibitors and angiotensin II receptor blockers \[ARBs\]) and have been titrated to a stable dose for at least 3 months prior to Screening are allowed in the study.

Glycated hemoglobin (HbA1c) at Screening ≥7%

Contraindication to systemic corticosteroid therapy or immunosuppressive therapy

Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the 12 months prior to Screening

Screening laboratory values for renal and liver function that meet or exceed any of the following:

Alanine transaminase (ALT) \> 2 x upper limit of normal for the testing laboratory (ULN)

Aspartate aminotransferase (AST) \> 2 x ULN

Total Bilirubin \> 2 x ULN (except if this is caused by Gilbert disease)

Alkaline phosphatase (ALP) \> 2 x ULN

Creatinine \> 2 x ULN

Screening laboratory values for hematologic and coagulation function that meet any of the following:

Hemoglobin \< lower limit of normal (LLN) (as per reference laboratory ranges)

Platelet count \< 150 x1000/μl

International normalized ratio (INR) \>1.1

Soluble terminal complement complex (sC5b-9)\>ULN

Significant anatomical abnormalities on renal ultrasound such as the presence of only 1 kidney, significant differences in kidney sizes between the right and left kidneys \>1.5 centimeters (about 0.59 inch), or presence of kidney cysts

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 Birmingham?

Yes, this clinical trial (NCT06270316) has an active research site in Birmingham, AL 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.

Fabry Disease Treatment Options in Birmingham, AL

If you're searching for fabry disease treatment options in Birmingham, AL, this clinical trial (NCT06270316) 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 Birmingham research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced fabry disease 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 fabry disease clinical trials near you to find additional studies recruiting in your area.

Fabry Disease Clinical Trial in Birmingham, AL

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About This Fabry Disease Study in Birmingham