Sacramento, CANCT03373968Now EnrollingIRB Ready

Duchenne Muscular Dystrophy Clinical Trial in Sacramento, CA

Access cutting-edge duchenne muscular dystrophy treatment through this clinical trial at a research site in Sacramento. Study-provided care at no cost to qualified participants.

Sponsored by Italfarmaco

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Expert Care in Sacramento

Access duchenne muscular dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related duchenne muscular dystrophy treatment provided free

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Check if you qualify for this duchenne muscular dystrophy clinical trial in Sacramento, CA

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Why Participate?

  • No-Cost Study Care

  • Local to Sacramento

    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.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Sacramento site if eligible
  4. 4Begin participation

About This Duchenne Muscular Dystrophy Study in Sacramento

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

Sponsor: Italfarmaco

Who Can Participate

Inclusion Criteria

Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met:
all the inclusion criteria and none of the

Exclusion Criteria

had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e. included in"off-target" group,
never been randomized because, the enrollment in the off target group was completed.
Aged ≥6 years old;
Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
Subjects must be willing to use adequate contraception:
Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:
True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject.
Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral,
transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly. Exclusion Criteria:
Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
Use of any current investigational drug other than Givinostat;
Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
Have platelets count, White Blood Cell and Hemoglobin at screening \< Lower Limit of Normal (LLN)\
(for abnormal screening laboratory test results (\<LLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still \<LLN, then exclusionary);
Have Triglycerides \> 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit\
(for abnormal screening laboratory test results (\>300 mg/dL), the triglycerides will be repeated once; if the repeat test result is still \>300 mg/dL, then exclusionary);
Have inadequate renal function, as defined by serum Cystatin C \>2 x the upper limit of normal (ULN) at screening visit\*. If the value is \>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 x ULN, the subject should be excluded);
Have heart failure (New York Heart Association Class III or IV)
Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\
(i.e. \> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
Have a baseline QTcF \>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures.
Have any hypersensitivity to the components of study medication;
Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.
the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the laboratory results done in screening/baseline (Visit 1) visit they will check the GIVINOSTAT dose and modify it as per protocol safety rules and/or dosage modifications rules.

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 Sacramento?

Yes, this clinical trial (NCT03373968) has an active research site in Sacramento, CA 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 Sacramento, CA

If you're searching for duchenne muscular dystrophy treatment options in Sacramento, CA, this clinical trial (NCT03373968) 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 Sacramento 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.

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