Is NCT04906460 recruiting in Atlanta, GA?

Yes, NCT04906460 is currently recruiting participants at its Atlanta, GA location. This duchenne muscular dystrophy clinical trial is actively enrolling qualified participants.

How do I join the duchenne muscular dystrophy clinical trial in Atlanta?

To join this clinical trial in Atlanta, 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 Atlanta 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.

Duchenne Muscular Dystrophy Clinical Trial in Atlanta, GA

About This Duchenne Muscular Dystrophy Study in Atlanta

This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.

Sponsor: Wave Life Sciences USA, Inc.

Who Can Participate

Inclusion Criteria

Part A and Part B:

Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B

Diagnosis of DMD based on clinical phenotype.

Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention

Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ).

Ambulatory or non-ambulatory male

Stable pulmonary and cardiac function, as measured by the following: (Part B): 1\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \>55% in patients \<10 years of age and \>45% in patients ≥10 years of age, as measured (and documented) by echocardiogram (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study. 7.Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid. 8\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit (Part B ). Part C

New patients to be screened for Part C.

Diagnosis of DMD based on clinical phenotype.

Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention

Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) .

Ambulatory male

Stable pulmonary and cardiac function, as measured by the following: 1\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \>55% in patients as measured (and documented) by echocardiogram (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study. 7\. Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid. 8\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit .

Exclusion Criteria

Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.

Part B and Part C: Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study.

Part B: Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit

Part C: Any recreational substance use (including prescribed cannabinoids), with the exception of nicotine, irrespective of legality, within 2 months prior to Screening and/or unwilling to refrain from such use for the duration of the study.

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 Atlanta?

Yes, this clinical trial (NCT04906460) has an active research site in Atlanta, GA 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.

Duchenne Muscular Dystrophy Treatment Options in Atlanta, GA

If you're searching for duchenne muscular dystrophy treatment options in Atlanta, GA, this clinical trial (NCT04906460) 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 Atlanta research site is actively enrolling participants for this clinical trial. You'll receive care from experienced duchenne muscular dystrophy 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 duchenne muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

Duchenne Muscular Dystrophy Clinical Trial in Atlanta, GA

Expert Care in Atlanta

IRB Approved

No-Cost Care

Apply for This Atlanta Location

Why Participate?

Simple Process