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ClinicalTrials.gov 11 recruiting now full pipeline small index

Rett Syndrome: full-pipeline trial registry

11 of 19 indexed Rett Syndrome trials are currently recruiting.

Corpus placement: #902 of 28,707 conditions by registered trial count.

19 US clinical trials · 11 currently recruiting · #902 of 28,707 conditions by registered trial count

Rett Syndrome: 19 US clinical trials tracked, 11 recruiting.

Rett Syndrome is the subject of 19 registered US clinical trials on ClinicalTrials.gov, 11 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 4 in Phase 1-2 (earlier-stage). The most active sponsor is Rett Syndrome Research Trust, running 3 of these trials.

Enrollment posture brief

Registry condition rett-syndrome

OPEN 11 | RECR 58% | LATE 3 | EARLY 4 | RANK #902 | SPON 3

Nearest open-share peer: Burnout (58% recruiting · 19 trials)

Rett Syndrome (this)58%Amyotrophic Lateral Sclerosi52.6%Anesthesia, Local42.1%Alcohol Consumption36.8%Attention Deficit Hyperactiv26.3%
Open-enrollment share among volume-matched conditions near Rett Syndrome

Rett Syndrome lists 11 open slots (58% of 19 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Rett Syndrome on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Rett Syndrome is recruiting above its size-band peers.

    11 of 19 indexed trials (57.9%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. One sponsor concentrates Rett Syndrome research.

    Rett Syndrome Research Trust accounts for 3 of 19 trials (15.8%). 14 distinct sponsors appear on at least one study for this condition label.

  3. Most Rett Syndrome trials plan to enrol fewer than 100 people.

    Median target enrolment is 50 among the 19 trials that report a genuine target (sentinel values excluded). 13 of those (68.4%) plan for fewer than 100 participants.

  4. Rett Syndrome splits between interventional and observational designs.

    10 interventional and 9 observational studies make up this index (52.6% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 12Phase 22Phase 33

Open and recent trials

19 total, page 1 of 1

RECRUITING 5,000 participants

The Rett Syndrome Global Registry

Rett Syndrome Research Trust

NCT04900493

RECRUITING 3,000 participants

Rett Syndrome Registry

International Rett Syndrome Foundation

NCT05432349

RECRUITING Phase 3 210 participants

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Biomed Industries

NCT06840496

RECRUITING 202 participants

Neurophysiologic Biomarkers in Rett Syndrome

Children's Hospital of Philadelphia

NCT05932589

RECRUITING NA 92 participants

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

Rush University Medical Center

NCT06139172

RECRUITING 80 participants

The Diagnostic Experience of Male Rett Syndrome

Children's Hospital Colorado

NCT06346106

RECRUITING Phase 3 33 participants

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Neurogene

NCT05898620

RECRUITING 30 participants

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Rett Syndrome Research Trust

NCT06338267

RECRUITING 20 participants

Cognitive Function in Rett Syndrome During Trofinetide Treatment

University of Minnesota

NCT06705816

RECRUITING Phase 3 15 participants

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Taysha Gene Therapies

NCT05606614

RECRUITING NA 10 participants

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Georgetown University

NCT05012475

ACTIVE NOT RECRUITING 50 participants

Obi Medical Robot: Evaluating Effectiveness Related to Usability

Desin

NCT07151950

ACTIVE NOT RECRUITING NA 14 participants

Technology-supported Motor Rehabilitation for People With Rett Syndrome

Georgetown University

NCT07418905

ACTIVE NOT RECRUITING Phase 1 6 participants

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Taysha Gene Therapies

NCT06152237

COMPLETED 426 participants

The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome

National Human Genome Research Institute (NHGRI)

NCT00891956

COMPLETED Phase 2 187 participants

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

Biomed Industries

NCT06849973

COMPLETED Phase 2 57 participants

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Hugo W. Moser Research Institute at Kennedy Krieger

NCT01520363

COMPLETED 21 participants

ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

Rett Syndrome Research Trust

NCT04514549

COMPLETED Phase 1 12 participants

Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)

Boston Children's Hospital

NCT01253317

Phase Distribution

PhaseTrial count
Phase 1 2
Phase 2 2
Phase 3 3

Named interventions

Most-linked intervention names on Rett Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

TSHA-102 Genetic
2 trials
Placebo Drug
2 trials
Emerald Device
1 trials
Drug: Bionetide Drug
1 trials
rhIGF-1 Drug
1 trials
dextromethorphan Drug
1 trials
computer gaming Behavioral
1 trials
One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils Device
1 trials

US sites by state

Facility locations filed on Rett Syndrome trials. A trial with sites in several states counts in each.

Massachusetts 9 trials
Texas 8 trials
Illinois 8 trials
Maryland 7 trials
Colorado 6 trials
California 6 trials
Tennessee 4 trials
Pennsylvania 4 trials

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

What to do with this Rett Syndrome page

19 registered trials, 11 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Rett Syndrome condition card totals 19 registered trials; most-linked intervention TSHA-102; top US site state Massachusetts. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.