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An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome
Plain-language guide to NCT02023424: phase 1 interventional trial of Glatiramer Acetate, 10 participants. Who took part, design and what was measured.
Key Facts
- Registry ID
- NCT02023424
- Phase
- phase 1
- Status
- status unknown
- Start
- 2014-01
- Completion (planned or actual)
- 2014-09
- Participants
- 10 (estimated)
- Design
- na, open label (everyone knows who gets what), treatment
- Conditions studied
- Rett Syndrome
- Results posted
- not yet
What This Study Did, in Plain Language
What Was Tested
This registered phase 1 trial tested Glatiramer Acetate.
Primary Safety Objective:To evaluate the safety and tolerability of 6 months treatment with GA in these patients.
It had 1 arm (groups): Copaxone (experimental).
The registry classes the interventions as drug.
Who Took Part
10 planned participants were the target.
It was open to women only aged from 6 years up to 15 years.
Healthy volunteers were not accepted; participants needed the condition being studied.
Examples of who could join: Females, age 6-15 years (inclusive). Patients whose parents or legal custodians have provided written informed consent to participate in the study. A diagnosis of RTT (classical or variant), defined according to the internationally agreed 2010 RetSearch criteria \[4\]. Evidence of a genetically defined pathological change in the MECP2 gene (point mutation or deletion). Patients with known epileptiform activity as recorded on EEG.
The full record lists 9 inclusion and 7 exclusion criteria; among the exclusions: intractable seizures that started during the last 6 months prior to beginning of the study; known hypersensitivity to glatiramer or mannitol; participation in another clinical study.
How the Study Was Run
It was run as a single-group and open-label study.
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 2014-01 with completion listed for 2014-09.
It ran at 1 site in Israel.
Its stated primary purpose is treatment.
What Was Measured
It also tracked 5 secondary outcomes, for example Change in height and weight.
What This Means in Plain Language
Put simply, this is a phase 1 trial of Glatiramer Acetate in 10 people.
Its size is small, so on its own it can only give an early look.
Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.
More context: the Glatiramer Acetate compound page, all human trials.
Our Notes on the Compound
Glatiramer Acetate: A mix of synthetic amino-acid chains (Copaxone) used in relapsing multiple sclerosis.
Related Pages
Source: ClinicalTrials.gov record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.