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Rett Syndrome Clinical Trials Near Me

As of November 13, 2026, 13 Rett Syndrome clinical trials are recruiting now and 2 are not yet recruiting. In the US, 9 of them have at least 1 open site, across 21 states. Enter your ZIP code below to see which are closest to you.

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All 13 Rett Syndrome trials and their US sites

Enter your ZIP code above to sort them by how far their nearest open site is from you.

  1. Rett Syndrome Registry
    Most sites in California, Ohio and Texas
    RecruitingAges 0-99International Rett Syndrome Foundation
    19US sites
    15 states
  2. To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
    Most sites in California, Arizona and Colorado
    RecruitingPhase 3Ages 5-20Biomed Industries
    14US sites
    11 states
  3. A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
    Most sites in Florida, Texas and Arkansas
    RecruitingPhase 3Ages 5-35UCB BIOSCIENCES
    8US sites
    6 states
  4. Neurophysiologic Biomarkers in Rett Syndrome
    Most sites in California, Colorado and Massachusetts
    RecruitingAges 1-18Children's Hospital of Philadelphia
    6US sites
    6 states
  5. Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome
    Only in New York
    RecruitingAges 2+University of Rochester
    1US site
  6. Cognitive Function in Rett Syndrome During Trofinetide Treatment
    Only in Minnesota
    RecruitingAges 18-99University of Minnesota
    1US site
  7. Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders
    Only in Texas
    RecruitingAges 4-12Healing Hope International
    1US site
  8. The Rett Syndrome Global Registry
    Only in Connecticut
    RecruitingRett Syndrome Research Trust
    1US site
  9. A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
    RecruitingPhase 2Ages 2-21Holland Bloorview Kids Rehabilitation Hospital
    No US site yet
  10. Gene Editing as a Therapeutic Approach for Rett Syndrome
    RecruitingAges 6 Months+University of Siena
    No US site yet
  11. GROWing Up With Rare GENEtic Syndromes
    RecruitingAges 18+dr. Laura C. G. de Graaff-Herder
    No US site yet
  12. Repurposing Mirtazapine in Rett Syndrome
    RecruitingPhase 2Ages 5-40University of Trieste
    No US site yet
  13. Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
    RecruitingPhase 1Ages 6-21Unravel Biosciences
    No US site yet
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Who runs the Rett Syndrome trials in the US, and what stage they are in

Of the 9 Rett Syndrome trials with a US site, 2 are in Phase 3, the last stage before a company can ask the FDA for approval. The remaining 7 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 2 of them, and universities, hospitals, foundations and government groups run 7. The most recently listed one first appeared on ClinicalTrials.gov on July 28, 2026.

Rett Syndrome trials in the US, state by state

Of the 9 Rett Syndrome trials with a US site, 7 accept children under 18. Their open sites are in 21 states, most often Texas, Pennsylvania and California. A trial counts once in each state where it has an open site, and some trials accept both children and adults.

StateTrialsChildrenAdultsCities with the most
Texas554Houston (4), Dallas (1), Irving (1), plus 1 more
Colorado434Aurora (2), Denver (1), Fort Collins (1)
Pennsylvania444Philadelphia (4)
California333Los Angeles (3), La Jolla (1), Oakland (1), plus 3 more
Florida333Miami (2), Orlando (1), Tampa (1)
Massachusetts333Boston (2), Brookline (1)
Illinois222Chicago (2)
Maryland222Baltimore (2)
Minnesota212Minneapolis (1), Saint Paul (1)
New York222Rochester (1), The Bronx (1)
Show the other 11 states
Tennessee222Nashville (2)
Washington222Seattle (2)
Alabama111Birmingham (1)
Arizona111Phoenix (1)
Arkansas111Little Rock (1)
Connecticut111Trumbull (1)
Georgia111Atlanta (1)
Missouri111St Louis (1)
North Carolina111Chapel Hill (1)
Ohio111Cincinnati (1), Columbus (1)
South Carolina111Greenwood (1)

US hospitals running the most Rett Syndrome trials

Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Rett Syndrome.

  1. Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania2 trials
  2. Texas Children's HospitalHouston, Texas2 trials
  3. Ability and BeyondThe Woodlands, Texas1 trial
  4. Biomed Testing Facility # BIO-01-85012Phoenix, Arizona1 trial
  5. Biomed Testing Facility # BIO-02-94104San Francisco, California1 trial
  6. Biomed Testing Facility # BIO-03-95817Sacramento, California1 trial
  7. Biomed Testing Facility # BIO-04-90095Los Angeles, California1 trial
  8. Biomed Testing Facility # BIO-05-92093La Jolla, California1 trial
  9. Biomed Testing Facility #BIO-06-80042Aurora, Colorado1 trial
  10. Biomed Testing Facility #BIO-07-33606Tampa, Florida1 trial

Help getting to a Rett Syndrome trial far from home

Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.

These nonprofits also help patients reach care, and each one sets its own rules.

Mercy Medical Angels
Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
Air Charity Network
Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
Ronald McDonald House
Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
Healthcare Hospitality Network
Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.

Program details were checked on each program's website on October 24, 2026.

Questions about finding a Rett Syndrome trial near you

Are there Rett Syndrome clinical trials near me?

As of November 13, 2026, 13 Rett Syndrome clinical trials are recruiting now and 2 are not yet recruiting. In the US, 9 of them have at least 1 open site, across 21 states. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.

Which states have Rett Syndrome clinical trial sites?

Recruiting Rett Syndrome trials list open sites in 21 states. Texas, Colorado and Pennsylvania have the most, and the table on this page lists every state.

Are there Rett Syndrome trials for children near me?

Of the 9 Rett Syndrome trials with a US site, 7 accept children under 18. Their open sites are in 21 states, most often Texas, Pennsylvania and California. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.

Who pays for travel to a Rett Syndrome clinical trial?

Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.

What if there is no Rett Syndrome trial near me?

Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Rett Syndrome trial opens a new site or changes status.

How is the distance to a trial site measured?

Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.

FDA-approved for Rett Syndrome: Daybue (trofinetide).

Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.