Rett Syndrome: Clinical Trials

14 trials for Rett Syndrome, newest first

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14 completed. 14 Phase 2, 2 Phase 1, 2 Phase 3.

By phase and status

PhaseTrialsStatus
Phase 214Completed 14
Phase 12Completed 2
Phase 32Completed 2

Follow Rett Syndrome: new trials by email Literature Review of Rett Syndrome trials Research Report on Rett Syndrome

Trials (filtered) newest first · largest first

All 14 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
Drug: Bionetide, Placebo - Rett Syndrome
Phase 2 / Phase 3CompletedBiomed Industries, Inc.1872024-03-152025-02-27
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
NTI164 - Rett Syndrome
Phase 1 / Phase 2CompletedFenix Innovation Group142023-09-202026-09-09
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
ANAVEX2-73 oral liquid, Placebo oral liquid - Rett Syndrome
Phase 2 / Phase 3CompletedAnavex Life Sciences Corp.922020-07-012023-08-21
A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
Ketamine - Rett Syndrome
Phase 2CompletedRett Syndrome Research Trust232019-03-122024-05-28
Study of ANAVEX2-73 in Patients With Rett Syndrome
ANAVEX2-73, Placebo - Rett Syndrome
Phase 2CompletedAnavex Life Sciences Corp.312019-02-282021-01-12
A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
NNZ-2566, Placebo - Rett Syndrome
Phase 2CompletedNeuren Pharmaceuticals Limited822016-03-012020-08-14
Pharmacological Treatment of Rett Syndrome With Statins
Lovastatin - Rett Syndrome
Phase 2CompletedMontefiore Medical Center202015-07-012019-07-12
Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)
Glatiramer Acetate - Rett Syndrome
Phase 2CompletedMontefiore Medical Center102013-08-012018-11-05
Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome
fingolimod (FTY720) - Rett's Syndrome
Phase 1 / Phase 2CompletedUniversity Hospital, Basel, Switzerland62013-08-012018-06-15
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
NNZ-2566, Placebo - Rett Syndrome
Phase 2CompletedNeuren Pharmaceuticals Limited672013-03-012018-02-05
Treatment of Rett Syndrome With Recombinant Human IGF-1
Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo - Rett Syndrome
Phase 2CompletedBoston Children's Hospital302013-01-012018-03-26
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
EPI-743, Placebo - Rett Syndrome
Phase 2CompletedEdison Pharmaceuticals Inc242013-01-012018-07-26
Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
dextromethorphan, placebo - Rett Syndrome
Phase 2CompletedHugo W. Moser Research Institute at Kennedy Krieger, Inc.572012-03-012018-12-04
Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome
Administration of a high dose of desipramine, Administration of a low dose of desipramine, Administration of a placebo - Rett Syndrome
Phase 2CompletedAssistance Publique Hopitaux De Marseille362009-02-172018-07-26

Narrower conditions

Ataxia (2)  ·  Autistic Disorder (2)  ·  Autism Spectrum Disorder (1)  ·  Genetic Diseases, X-Linked (1)  ·  X-Linked Intellectual Disability (1)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (14)  ·  Genetic Diseases, Inborn (14)  ·  Heredodegenerative Disorders, Nervous System (14)  ·  Intellectual Disability (14)  ·  Nervous System Diseases (14)  ·  Neurobehavioral Manifestations (14)  ·  Neurologic Manifestations (14)  ·  Genetic Diseases, X-Linked (13)  ·  X-Linked Intellectual Disability (13)  ·  Child Development Disorders, Pervasive (3)  ·  Mental Disorders (3)  ·  Neurodevelopmental Disorders (3)  ·  Autism Spectrum Disorder (2)  ·  Dyskinesias (2)  ·  Pathological Conditions, Signs and Symptoms (2)

Interventions

Injections (2)  ·  tetrahydro-N, N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (2)  ·  trofinetide (2)  ·  DNA, Ribosomal (1)  ·  Desipramine (1)  ·  Dextromethorphan (1)  ·  Fingolimod Hydrochloride (1)  ·  Glatiramer Acetate (1)  ·  Ketamine (1)  ·  Lovastatin (1)  ·  Sodium Chloride (1)  ·  Water (1)  ·  alpha-tocotrienol quinone (1)  ·  mecasermin (1)

Sponsors

Anavex Life Sciences Corp. (2)  ·  Montefiore Medical Center (2)  ·  Neuren Pharmaceuticals Limited (2)  ·  Assistance Publique Hopitaux De Marseille (1)  ·  Biomed Industries, Inc. (1)  ·  Boston Children's Hospital (1)  ·  Edison Pharmaceuticals Inc (1)  ·  Fenix Innovation Group (1)  ·  Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1)  ·  Rett Syndrome Research Trust (1)  ·  University Hospital, Basel, Switzerland (1)

Countries

United States (9)  ·  Australia (3)  ·  Canada (1)  ·  France (1)  ·  Italy (1)  ·  Switzerland (1)  ·  United Kingdom (1)

Top investigators

Alan Percy, MD (2)  ·  Timothy Feyma, MD (2)  ·  Aleksandra Djukic, MD PhD (1)  ·  Arthur Beisang, MD (1)  ·  Daniel G Glaze, M.D. (1)  ·  Daniel Glaze, MD (1)  ·  David Nguyen, PhD (1)  ·  Eric Marsh, MD (1)  ·  Jana von Hehn, PhD (1)  ·  Jeff Neul, MD (1)  ·  Jeffrey L Neul, M.D., Ph.D. (1)  ·  Jeffrey Neul, MD, PhD (1)  ·  Josette Mancini (1)  ·  Lloyd L. Tran, PhD (1)  ·  Ludwig Kappos, Prof. (1)

Results publications

PMID 37460385, PMID 38363467, PMID 27363291, PMID 29468173, PMID 33395098, PMID 33407685, PMID 39856538