Biomarkers in Kennedy Disease The identification of biomarkers as clinical endpoints in drug efficacy trials in Kennedy disease; an international academic multicenter study in Utrecht and Leuven

Biomarkers in Kennedy Disease The identification of biomarkers as clinical endpoints in drug efficacy trials in Kennedy disease; an international academic multicenter study in Utrecht and Leuven - Biomarkers in Kennedy Disease

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON32727

Enrollment

Registered

2010-08-25

Start date

2011-01-25

Completion date

Unknown

Last updated

2024-05-06

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

Conditions

Kennedy Disease Spinal bulbar muscular atrophy

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1) genetically confirmed KD in patients with more than one of the following symptoms,: muscle weakness, muscle atrophy, bulbar palsy, fasciculations, tremor or mild sensory symptoms 2) male 3) Androgen receptor gene with a CAG repeat exceeding 38 copies 4) age >18 years 5) given written informed consent

Exclusion criteria

Exclusion criteria: 1) Androgen receptor gene with CAG repeat of less than 38 repeats 2) female carriers 3)

Design outcomes

Primary

Measure	Time frame
The main study parameters of this study are the correlations between possible biomarkers and the disease progression in patients. Disease status and progression will be measured with the ALS-FRS-R, Norris scale and ShortForm-36. Possible biomarkers are the manual muscle testing, 6minute-walking-test, circumference of limbs, bloodtesten (transaminases, creatine kinase, glucose, HbA1c, prolactine, testosterone, luteinizing hormone, follicle stimulating hormone and sex-hormone-binding-globuline) and genomic profiling of mRNA. With the discovery of (a) new biomarker(s) the sample size calculation for drug efficacy trials in KD can be made.	—

Measure

Time frame

The main study parameters of this study are the correlations between possible biomarkers and the disease progression in patients. Disease status and progression will be measured with the ALS-FRS-R, Norris scale and ShortForm-36. Possible biomarkers are the manual muscle testing, 6minute-walking-test, circumference of limbs, bloodtesten (transaminases, creatine kinase, glucose, HbA1c, prolactine, testosterone, luteinizing hormone, follicle stimulating hormone and sex-hormone-binding-globuline) and genomic profiling of mRNA. With the discovery of (a) new biomarker(s) the sample size calculation for drug efficacy trials in KD can be made.

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Countries

Netherlands

Outcome results