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Rett Syndrome clinical trials

The registry lists 96 studies for Rett Syndrome across more than 10 countries. 19 are recruiting or about to start. 86 of them are mainly about Rett Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-02.

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Breast cancer · Recruiting: 2 came in, 3 changed

What changed in your saved searches, Oct 31 – Nov 6:

A study of drug A after surgery for early breast cancer

Came in · now Recruiting

Drug B with hormone therapy in advanced breast cancer

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· Sites: 4 added, including Seoul and Busan

· Enrollment: 300 → 450 participants

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Status

Completed
48
Recruiting
14
Active, not recruiting
11
Terminated
8
Status unknown
8
Enrolling by invitation
3

Phase

Phase 2
26
Phase 3
18
Phase 1
8
Early Phase 1
2

Where

United States
55
Italy
11
Australia
9
United Kingdom
8
Canada
6
France
6

Who runs them

Rett Syndrome Research Trust (5) · ACADIA Pharmaceuticals Inc. (4) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (4) · University of Alabama at Birmingham (3) · National Taiwan University Hospital (3) · Taysha Gene Therapies, Inc. (3) · Edison Pharmaceuticals Inc (3) · Anavex Life Sciences Corp. (3)

What is being tested here

Drugs under test in Rett Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
NTI164112
Lamivudine101
Fenfluramine111
Vorinostat101
Trofinetide046
Blarcamesine023
Dextromethorphan013
Glatiramer002
Alpha-tocotrienol quinone002
Mecasermin002

Coming up

6phase 3 studies under way
12studies finish main data collection within a year1 of them phase 3

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The 10 nearest, phase 3 first

Who these studies take

Of the 14 Rett Syndrome studies recruiting now, 4 (29%) set no upper age limit and 7 (50%) stop at 75 or younger. 12 (86%) take children under 18. 6 (43%) are for women only and 2 (14%) for men only; 3 (21%) take healthy volunteers.

Recruiting studies that take each age, of 14
AgeWomenMen
1010 (71%)6 (43%)
1812 (86%)8 (57%)
307 (50%)8 (57%)
406 (43%)7 (50%)
506 (43%)7 (50%)
656 (43%)7 (50%)
756 (43%)6 (43%)
856 (43%)6 (43%)

Across all 96 studies, recruiting or not: 66% stop at 75 or younger and 66% at 65; 90% take children; 57% are for women only and 3% for men only; 10% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 50 of 96. Browse all 96 → or search them.