Access cutting-edge junctional epidermolysis bullosa treatment through this phase 1/2 clinical trial at a research site in Los Angeles. Study-provided care at no cost to qualified participants.
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Access junctional epidermolysis bullosa specialists at no cost
This study follows strict safety protocols and ethical guidelines
All study-related junctional epidermolysis bullosa treatment provided free
Check if you qualify for this junctional epidermolysis bullosa clinical trial in Los Angeles, CA
No-Cost Study Care
Local to Los Angeles
Convenient for CA 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.
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or LAMC2 genes, resulting in loss of laminin 332 and poor epidermal-dermal adherence. Eighty percent of H-JEB patients have LAMB3 mutations and about 95% of these are nonsense mutations. The investigators recently demonstrated that gentamicin readily induced nonsense mutation readthrough and produced full-length laminin beta3 in several nonsense mutations tested. Importantly, the gentamicin-induced laminin beta3 restored laminin 332 assembly, secretion, and deposition into the dermal-epidermal junction (DEJ). Newly induced laminin 332 reversed abnormal H-JEB cellular phenotypes. Herein, the investigators propose the first clinical trial of gentamicin (by topical and intravenous administration) in JEB patients with nonsense mutations. The milestones will include restored laminin 332 and hemidesmosomes at the DEJ, improved wound closure, and the absence of significant gentamicin side effects.
Sponsor: University of Southern California
Yes, this clinical trial (NCT03526159) has an active research site in Los Angeles, CA that is currently enrolling participants.
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.
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.
Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.
If you're searching for junctional epidermolysis bullosa treatment options in Los Angeles, CA, this clinical trial (NCT03526159) 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 Los Angeles research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced junctional epidermolysis bullosa 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.
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