Trial document




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  DRKS00009032

Trial Description

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Title

Non-interventional study X-TRA

Safety and efficacy of safinamide (Xadago®) as add-on therapy to levodopa in mid- to late-stage Parkinson’s disease patients – a non-interventional observational study in daily practice of German neurologists

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Trial Acronym

X-TRA

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URL of the Trial

http:///

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Brief Summary in Lay Language

The signs and symptoms of Parkinson’s disease (PD), a chronic neurodegenerative disorder predominantly characterized by the loss of dopaminergic neurons in the substantia nigra pars compacta, are most effectively treated by levodopa (L-dopa).
However, nearly all PD patients experience a fluctuating response to L-dopa sooner or later within few years of therapy dependent on dosing of L-dopa. End-of-dose wearing off and dyskinesia are the most common motor complications associated with L-dopa treatment.
Once they appear, the management of motor complications is often challenging and patients require recurrent drug therapy adjustments to improve fluctuations of movement without exacerbating severe dyskinesia.
Safinamide (Xadago®) is a new treatment for adult patients with fluctuating mid-to-late-stage idiopathic PD as add-on therapy to a stable dose of L-dopa, alone or in combination with other PD compounds.
Safinamide improves motor symptoms, motor complications and quality of life in combination with other PD drugs such as dopamine agonists and L-dopa, reduces off time and extends on time without troublesome dyskinesia.
The objective of this non-interventional study is to gain insight into the efficacy and safety of Xadago® in a wide population of patients with idiopathic PD within the approved indication during routine clinical care. With the aid of commonly used diagnostic methods, particularly motor and non-motor symptoms, influence on quality of life, and adverse effects of the therapy shall be determined.

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Brief Summary in Scientific Language

The signs and symptoms of Parkinson’s disease (PD), a chronic neurodegenerative disorder predominantly characterized by the loss of dopaminergic neurons in the substantia nigra pars compacta, are most effectively treated by levodopa (L-dopa).
However, nearly all PD patients experience a fluctuating response to L-dopa sooner or later within few years of therapy dependent on dosing of L-dopa. End-of-dose wearing off and dyskinesia are the most common motor complications associated with L-dopa treatment.
Once they appear, the management of motor complications is often challenging and patients require recurrent drug therapy adjustments to improve fluctuations of movement without exacerbating severe dyskinesia.
Safinamide (Xadago®) is a new treatment for adult patients with fluctuating mid-to-late-stage idiopathic PD as add-on therapy to a stable dose of L-dopa, alone or in combination with other PD compounds.
Safinamide is a unique molecule with novel mechanisms of action, both dopaminergic and nondopaminergic ones, which include monoamine oxidase-B (MAO-B) inhibition, sodium channel blockade and calcium channel modulation, thus inhibiting the excessive glutamate release. Safinamide improves motor symptoms, motor complications and quality of life in combination with other PD drugs such as dopamine agonists and L-dopa, reduces off time and extends on time without troublesome dyskinesia.
The objective of this non-interventional study is to gain insight into the efficacy and safety of Xadago® in a wide population of patients with idiopathic PD within the approved indication during routine clinical care. With the aid of commonly used diagnostic methods, particularly motor and non-motor symptoms, influence on quality of life, and adverse effects of the therapy shall be determined.

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Organizational Data

  •   DRKS00009032
  •   2015/08/19
  •   [---]*
  •   no
  •   Approved
  •   F-2015-032, Ethik-Kommission bei der Landesärztekammer Baden-Württemberg
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Secondary IDs

  • [---]*
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Health Condition or Problem studied

  •   G20 -  Parkinson disease
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Interventions/Observational Groups

  •   Since this is a non-interventional study, the treatment of patients with Xadago® is carried out according to the current summary of product characteristics within the approved indication. Patients must give their consent to the pseudonymized transfer of their data.
    At baseline the investigator documents the [patient´s] demographic data, basic information about the Parkinson's disease and relevant concomitant diseases.
    Treatments with Xadago® and other Parkinson's add-on therapies as well as all adverse events occurring under treatment are continuously documented.
    At the end of the observation period, reasons for premature termination are documented where applicable and whether Xadago® therapy will be continued after the end of the study.
    Investigator and patient carry out a concluding assessment of the change of symptoms under treatment and the tolerability of Xadago®
    At specified dates the investigator evaluates the patient´s motor and non-motor symptoms as well as abnormal involuntary movements (dyskinesia) and asks the patient about his well-being using the standardized questionnaires MDS-UPDRS (Movement Disorder Society-Sponsored Revision of the Unified Parkinson’s Disease Rating Scale), NMSS (Non-Motor Symptoms Scale) und AIMS (Abnormal Involuntary Movement Scale). The patient or his/her legal representative will be asked to answer the patient´s questionnaire PDQ -8 (Parkinson’s Disease Quality of Life Questionnaire) at the beginning and the end of the observation period.
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Characteristics

  •   Non-interventional
  •   Observational study
  •   Single arm study
  •   Open (masking not used)
  •   [---]*
  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   N/A
  •   No
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Primary Outcome

The objective of this non-interventional study is to gain insight into the efficacy and safety of Xadago® directly after market launch in a wide population of patients with idiopathic PD within the approved indication during routine clinical care. With the aid of commonly used diagnostic methods, particularly motor and non-motor symptoms, influence on quality of life, and adverse effects of the therapy shall be determined.
The following questionnaires are used:
- at start of observation: Assessment of motor symptoms via Physician questionnaire MDS-UPDRS, Part III; Assessment of non-motor symptoms via Physician questionnaire NMSS; Assessment of dyskinesia via Physician questionnaire AIMS; Assessment of quality of life via Patient questionnaire PDQ-8
- approx. 3 months after start of observation: Assessment of non-motor symptoms via Physician questionnaire NMSS; Assessment of dyskinesia via Physician questionnaire AIMS
- End of observation (approx. 6 months after start of observation resp. at premature termination): Assessment of motor symptoms via Physician questionnaire MDS-UPDRS, Part III; Assessment of non-motor symptoms via Physician questionnaire NMSS; Assessment of dyskinesia via Physician questionnaire AIMS; Assessment of quality of life via Patient questionnaire PDQ 8.
Treatment with Xadago® and other Parkinson's add-on therapies as well as all adverse events occurring under treatment are continuously documented.

Abbreviations:
MDS-UPDRS: : Movement Disorder Society-Sponsored Revision of the Unified Parkinson’s Disease Rating Scale
NMSS: Non-Motor Symptoms Scale
AIMS: Abnormal Involuntary Movement Scale
PDQ-8: Parkinson's Disease Quality of Life Questionnaire

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Secondary Outcome

[---]*

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • other 
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Recruitment

  •   Actual
  •   2015/09/03
  •   2500
  •   Multicenter trial
  •   National
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Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   no maximum age
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Additional Inclusion Criteria

• The patient is at least 18 years old at time point of enrollment into the study
• Diagnosis of mid- to late-stage fluctuating PD
• The patient has not yet been treated with Xadago®
• The patient receives a stable dose of L-Dopa
• The decision to initiate the treatment with Xadago® has been made by the treating physician considering the indications of the SmPC
• The patient has been informed about the study in detail by the physician and the written informed consent of the patient (resp. his/her statutory representative) to the participation in the study has been obtained

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Exclusion Criteria

• Hypersensitivity to the active substance or to any of the excipients
• Concomitant treatment with other monoamine oxidase (MAO) inhibitors
• Concomitant treatment with pethidine
• severe hepatic impairment
• albinism, retinal degeneration, uveitis, inherited retinopathy or severe progressive diabetic retinopathy
Also, following patients will not be included in the study according to the SmPC:
• Women of childbearing potential without adequate contraception
• Pregnant women
• Breast-feeding women

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Addresses

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    • Zambon GmbH
    • Fraunhoferstr. 18b
    • 82152  Planegg
    • Germany
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    • Parkinson-Klinik Wolfach GmbH & Co KG
    • Mr.  Prof. Dr. med.  Wolfgang  Jost 
    • Kreuzbergstr. 12-24
    • 77709  Wolfach
    • Germany
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    • Zambon GmbH
    • Mr.  Dr.  Dietrich  Bosse 
    • Kurfürstendamm 103/104
    • 10711  Berlin
    • Germany
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Sources of Monetary or Material Support

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    • Zambon GmbH
    • Fraunhoferstr. 18b
    • 82152  Planegg
    • Germany
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Status

  •   Recruiting complete, follow-up complete
  •   2016/12/09
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Trial Publications, Results and other Documents

  • [---]*
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* This entry means the parameter is not applicable or has not been set.