Tampa, FLNCT06844214Now EnrollingIRB Ready

Myotonic Dystrophy Clinical Trial in Tampa, FL

Access cutting-edge myotonic dystrophy treatment through this phase 1/2 clinical trial at a research site in Tampa. Study-provided care at no cost to qualified participants.

Sponsored by Sanofi

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Expert Care in Tampa

Access myotonic dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related myotonic dystrophy treatment provided free

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Check if you qualify for this myotonic dystrophy clinical trial in Tampa, FL

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Tampa

    Convenient for FL 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 Tampa site if eligible
  4. 4Begin participation

About This Myotonic Dystrophy Study in Tampa

This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) adm

Sponsor: Sanofi

Who Can Participate

Inclusion Criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
For Part A, participants must be 18 to 55 years of age inclusive, at the time of signing the informed consent.
For Part B, participants must be as follows:
10 to 17 years of age inclusive, at the time of signing the informed consent or,
18 to 55 years of age inclusive, at the time of signing the informed consent.
Participants with non-congenital onset DM1
Participants presenting with signs of DM1 including myotonia and muscle weakness, as diagnosed previously by a clinician based on medical history.
Participants with genetic diagnosis of DM1 \[cytosine-thymine-guanine (CTG) repeat length ≥50 in one allele from medical history\]
Participants who can walk independently for at least 10 meters at screening (orthoses and ankle braces allowed).

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:
Participants with neutralizing antibodies against the AAV.SAN011 capsid
Participants with left ventricular ejection fraction \<50%
Participants with liver or biliary disease defined as having at least one of the following:
ALT \>3 x ULN and AST \>3 x ULN
Alkaline phosphatase \>3 x ULN
Total bilirubin \>1.5 x ULN (unless has a genetically confirmed diagnosis of Gilbert's syndrome)
Direct bilirubin ≥1.5 x ULN
Participants with International normalized ratio \>1.5
Participants with renal disease defined as: • Serum creatinine \>1.5 x ULN and/or estimated glomerular filtration rate \<60 mL/min/1.73 m2 as determined by Chronic Kidney Disease Epidemiology Collaboration (2021) for those age ≥18 years and Bedside Schwartz Equation for those \<18 years
Participants with chronic respiratory insufficiency and on long term/hull-time ventilatory assistance requiring at least 6 hours per day for at least 21 consecutive days.
Participants with contraindication to corticosteroid or with conditions that could worsen in the presence of corticosteroids, as determined by the Investigator.
Participants with active hepatitis B or C infection; HBsAg (+), or HCV RNA (+), or current antiviral therapy for either.
Participants with HBcAb (+) who are not amenable for prophylactic anti-HBV therapy or pre-emptive therapy guided by serial HBV DNA monitoring during the corticosteroids therapy.
Participants at high risk for tuberculosis reactivation during the corticosteroids therapy as determined by the Investigator.
Participants with a known HIV infection
Participants with serious intercurrent illness that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
Participants with recent history of or current drug or alcohol abuse in the past 12 months prior to screening.
Participants with history of tibialis anterior biopsy within 12 weeks from Day 1 or planning to undergo tibialis anterior biopsies during the duration of this clinical trial.
Participants with significant developmental delay, intellectual disability, or behavioral neuropsychiatric manifestations as determined by the Investigator.
Participants with previous systemic corticosteroids treatment at doses of \>5 mg/day within 15 days of Day 1
Participants with previous treatment with anti-myotonic medication within 15 days of Day 1
Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
Participants who have been classified as severe cardiac risk by the Investigator. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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 Tampa?

Yes, this clinical trial (NCT06844214) has an active research site in Tampa, FL 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.

Myotonic Dystrophy Treatment Options in Tampa, FL

If you're searching for myotonic dystrophy treatment options in Tampa, FL, this clinical trial (NCT06844214) 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 Tampa research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced myotonic 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 myotonic dystrophy clinical trials near you to find additional studies recruiting in your area.

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