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A Pilot Phase II Study for the evaluation of efficacy and tolerability of Gammaidroxybutyrate (ALCOVER) administered by oral route as add on treatmant in patients affected by DYT 11 Myoclonic Dystonia. - ND

A Pilot Phase II Study for the evaluation of efficacy and tolerability of Gammaidroxybutyrate (ALCOVER) administered by oral route as add on treatmant in patients affected by DYT 11 Myoclonic Dystonia. - ND

Status
Active, not recruiting
Phases
Unknown
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2006-001039-23-IT
Enrollment
Unknown
Registered
2007-09-10
Start date
2006-04-10
Completion date
Unknown
Last updated
2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

DYT 11 Myoclonic Dystonia. MedDRA version: 9.1 Level: SOC Classification code 10029205 Term: Nervous system disorders

Interventions

Trade Name: ALCOVER*OS SOLUZ 140ML 17,5% Pharmaceutical Form: Oral solution INN or Proposed INN: SODIO OXIBATO CAS Number: 591-81-1 Concentration unit: % percent Concentration type: equal Concentrati

Sponsors

ISTITUTO NEUROLOGICO "CARLO BESTA"
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: Male and females > 17 years. Diagnosis of myoclonic dystonia genetically confirmed (mutation of epsilon sarcoglycan gene, 7q21-q31; DYT 11). Refractory to treatment with conventional medications. No contraindications to GHB. Written informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Serious medical and/or psychiatric diseases. More than two concomitant medications for myoclonic dystonia therapy and/or modifications of concomitant therapy in the last month prior to basal visit. Alcohol or drug abuse (last 2 years). Pregnant or lactating females. Subjects who received an experimantal drug or have participated in a clinical trial within 3 months prior to screening.

Design outcomes

Primary

MeasureTime frame
Main Objective: To get preliminary data on the efficacy and tolerability of GHB in treating the movement disorder of the DYT 11 myoclonic dystonia patients and to determine the optimal therapeutic dose.;Secondary Objective: To evaluate the modification of various neurophysiological parameters following the GHB therapy to assess the possible relation between these variations and the clinical course of the movement disorder.;Primary end point(s): Rate of responders (decrease of at least 25% of total UMRS score. Total and single item score at basal visit will be compared with scores at final visit.—

Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026