Madera, CANCT05861999Now EnrollingIRB Ready

Spinal Muscular Atrophy Clinical Trial in Madera, CA

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

Sponsored by Hoffmann-La Roche

Quick Self-Assessment

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Expert Care in Madera

Access spinal muscular atrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related spinal muscular atrophy treatment provided free

Apply for This Madera Location

Check if you qualify for this spinal muscular atrophy clinical trial in Madera, CA

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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 Madera

    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 Madera site if eligible
  4. 4Begin participation

About This Spinal Muscular Atrophy Study in Madera

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

Sponsor: Hoffmann-La Roche

Who Can Participate

Inclusion Criteria

\<2 years of age at the time of informed consent
Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene
Confirmed presence of two SMN2 gene copies as documented through laboratory testing
Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment
If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration.
In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function/abilit

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 Madera?

Yes, this clinical trial (NCT05861999) has an active research site in Madera, 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.

Spinal Muscular Atrophy Treatment Options in Madera, CA

If you're searching for spinal muscular atrophy treatment options in Madera, CA, this clinical trial (NCT05861999) 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 Madera research site is actively enrolling participants for this clinical trial. You'll receive care from experienced spinal muscular atrophy 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 spinal muscular atrophy clinical trials near you to find additional studies recruiting in your area.

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