Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

What Was Tested

The trial (phase 2) is built around Glatiramer Acetate.

The drug, Copaxone (generic name - Glatiramer acetate) is medication FDA approved for the treatment of multiple sclerosis.

Copaxone's high safety profile has been documented in large cohorts of patients for more than 12 years.

It had 1 arm (groups): Copaxone (experimental).

The registry classes the interventions as drug.

More on the Plan

The MeCP2 gene plays a major role in transcriptional regulation of other genes, one of which is the gene encoding brain-derived neurotrophic factor (BDNF).

The disease progression and severity of symptoms is directly affected by the level of BDNF expression.

Copaxone treatment by subcutaneous injection caused elevation of BDNF levels.

Who Took Part

Enrollment: 10 actual participants took part.

It was open to women only aged from 10 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Ambulatory (with their without support).

The full record lists 3 inclusion and 5 exclusion criteria; among the exclusions: prolonged qtc (obtained within 30 days prior to enrollment); presence of co morbid non-rett related disease; presence of immunodeficiency requiring intravenous immunoglobulin 3 (ivig 3) months prior to enrollment.

How the Study Was Run

Design: single-group and open-label.

Nothing was hidden in this open-label Glatiramer Acetate study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2013-08 with completion listed for 2014-08.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Gait Velocity as Measured by GAITRite System (time frame: Baseline and Final week of treatment (week 32)).

It also tracked 5 secondary outcomes, for example Breath Hold Index (Number of Breath Holds Per Hour; Assessed in the Sleep Monitoring Lab), Breath Hold Time (Assessed in the Sleep Monitoring Lab), Visual Memory Novelty Score as Assessed by TX300 Tobii Computer, Visual Attention (Number of Fixations) Assessed by Eye-tracking TX300 Tobii Computer and Visual Attention (Fixation Length) Assessed by Eye-tracking TX300 Tobii Computer.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Glatiramer Acetate in 10 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Glatiramer Acetate compound page, all human trials.