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Study of the Efficacy of Topiramate in Patients With Prader Willi Syndrome Over 8 Weeks

NCT02810483 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 69

Last updated 2018-04-11

No results posted yet for this study

Summary

There is no specific treatment for core symptoms of PWS. Regarding behavioral and psychiatric symptoms (hyperphagia, imulsivity and self-mutilations), one of the only drug options consists in antipsychotics, that are not efficient and might be responsible for a worsening of the weight gain (major issue in PWS). An alternative therapeutic approach for behavioral disturbances has been suggested by some authors with topiramate (Epitomax®), an antiepileptic drug that can be used as a mood stabilizer and anti-impulsive. In addition, topiramate is used as a treatment for eating disorders because it induces loss of weight and appetite. This last effect might be useful in the case of SPW.

Except for some clinical case reports, the investigators only found one open study for topiramate in SPW 8 patientssuggesting promising effects. There si however no placebo controlled study..

Objective:

To evaluate the efficacy of topiramate (200 mg / d) on Eating disorders (E), self Mutilations (M), irritability and Impulsivity (I), metabolic status, and tolerance among of PWS patients.

Methodology:

This is a multicenter (out-patients in Toulouse, Reims, Nantes and Paris and in-patients in Hendaye) 8 weeks double-blind placebo controlled study .

Subjects (n = 125 for 112 analyzable) all having PWS, aged 12 years-old and more should have any of the following symptoms: E, M and U (see above). All subjects will be randomly allocated into two groups one taking a placebo, the other taking topiramate (50mg / day initially, increasing up to 50mg per week 200mg / day).

The population of analyzable patients in and out patient will be of equal size (n = 56). The inclusion period is two years..

Are excluded subjects with antipsychotic or mood stabilizer medication or topiramate.

The primary endpoint will be the rate of responders, with response defined by obtaining a score of 1 or 2 on the CGI improvement after 8 weeks of treatment

Other assessments, secondary endpoints :

* Clinic: Weight / Size / Self-injury behavior (french Echelle des Conduites Auto et Hétéro Aggressives, ECAHA))
* Psychometric: C-SHARP and A-SHARP / Conners (Impulsivity) / Dickens (Eating behavior for PWS)
* Organic: NFS, serum electrolytes, creatinine, ammonia plasma, serum bicarbonate, AST / ALT / GGT, ghrelin, fasting glucose, lipid profile and insulin, leptin, TG and HbA1c.
* Side effects of topiramate: SAPS / SANS and BPRS (hallucinations), anxiety scales and laboratory tests.

Conditions

Interventions

DRUG

Topiramate

Increase phase. The starting dose is 50 mg / day with gradual increase in dose to 50 mg / week until the end of the 3rd week. Dose of study: 200mg for 5 weeks. Decrease phase: The 9th week, 100mg for 4 days then 50mg for 3 days.

DRUG

Placebo Comparator

ncrease phase. The starting dose is 50 mg / day with gradual increase in dose to 50 mg / week until the end of the 3rd week. Dose of study: 200mg for 5 weeks. Decrease phase: The 9th week, 100mg for 4 days then 50mg for 3 days.

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Olivier BONNOT, PhD, MD · Assistance Publique - Hôpitaux de Paris

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-12-31
Primary Completion
2015-11-30
Completion
2016-06-30

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02810483 on ClinicalTrials.gov