Back to resultsL-Citrulline in Patients With Post-Polio Syndrome
Primary Purpose
Post-Polio Syndrome
Status
Completed
Phase
Phase 3
Locations
Switzerland
Study Type
Interventional
Intervention
15g L-citrulline daily p.o.
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Post-Polio Syndrome focused on measuring Post-Polio Syndrome, PPS
Eligibility Criteria
Inclusion Criteria:
- Prior paralytic poliomyelitis with evidence of motor neuron loss
- A period of partial or complete functional recovery after acute paralytic Poliomyelitis
- Slowly progressive and persistent new muscle weakness or decreased endurance, with or without generalized fatigue, muscle atrophy, or muscle and joint pain. Symptoms that persist for at least a year
- Exclusion of other neuromuscular, medical and skeletal abnormalities as causes of symptoms
- Patients older than 18 years at time of Screening
- ambulant
- ability to walk 150m in the 6 min. Walking distance (6MWD)
- females of childbearing potential willing to use contraceptive during the study
Exclusion Criteria:
- Previous (3 months or less) or concomitant participation in any other therapeutic trial
- Use of L-citrulline or L-arginine within the last 3 months
- Known individual hypersensitivity to L-citrulline
- Known or suspected malignancy
- Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
- Pregnant or breast-feeding women
- Severe renal failure (calculated glomerular filtration rate (GFR) < 30 ml/min)
Sites / Locations
- University of Basel, Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
L-citrulline
Placebo
Arm Description
15 g L-citrulline p.o. per day (3x 5g) for 24 weeks
L-citrulline Placebo 3 times daily p.o. for 24 weeks
Outcomes
Primary Outcome Measures
Mean change of 6 Minute Walking Distance(6MWD)
Secondary Outcome Measures
Mean change of motor function measure (MFM) total score
Change of quantitative muscle MRI (IDEAL-CPMG with T₂ and lipid quantitation and dynamic 31P-MR spectroscopy)
Change of serum concentrations for markers of muscle necrosis
Change of serum concentrations for markers of oxidative stress
Change of serum concentrations for markers of nitrosative stress
Change of serum concentrations for markers of mitochondrial related genes
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02801071
Brief Title
L-Citrulline in Patients With Post-Polio Syndrome
Official Title
"Treatment With L-citrulline in Patients With Post-polio Syndrome - a Randomized Double Blind Placebo Controlled Study"
Study Type
Interventional
2. Study Status
Record Verification Date
January 2019
Overall Recruitment Status
Completed
Study Start Date
June 14, 2016 (Actual)
Primary Completion Date
August 15, 2017 (Actual)
Study Completion Date
October 5, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Dirk Fischer
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of the study is to show that the intake of L-citrulline improves muscle function in patients with Post-Polio Syndrome (PPS).
Detailed Description
This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline, respectively placebo given orally; 5 g L-citrulline or placebo will be given three times daily. The duration of the study is 48 weeks and comprehends one screening and four study visits.
Additionally, in order to investigate whether PPS is associated with an atrophy of spinal cord gray and potentially also white matter as well as motor cortex atrophy, it is planned to offer all trial participants an additional optional spinal cord/brain MRI investigation and a MRI at week 48 and a follow-up one year later together with a clinical and physiotherapy assessment during extension phase. We don't expect Citrulline to affect the spinal cord gray or white and gray matter.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Post-Polio Syndrome
Keywords
Post-Polio Syndrome, PPS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
L-citrulline
Arm Type
Experimental
Arm Description
15 g L-citrulline p.o. per day (3x 5g) for 24 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
L-citrulline Placebo 3 times daily p.o. for 24 weeks
Intervention Type
Drug
Intervention Name(s)
15g L-citrulline daily p.o.
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Mean change of 6 Minute Walking Distance(6MWD)
Time Frame
Baseline to week 48
Secondary Outcome Measure Information:
Title
Mean change of motor function measure (MFM) total score
Time Frame
Baseline to week 48
Title
Change of quantitative muscle MRI (IDEAL-CPMG with T₂ and lipid quantitation and dynamic 31P-MR spectroscopy)
Time Frame
Baseline to week 48
Title
Change of serum concentrations for markers of muscle necrosis
Time Frame
Baseline to week 48
Title
Change of serum concentrations for markers of oxidative stress
Time Frame
Baseline to week 48
Title
Change of serum concentrations for markers of nitrosative stress
Time Frame
Baseline to week 48
Title
Change of serum concentrations for markers of mitochondrial related genes
Time Frame
Baseline to week 48
Other Pre-specified Outcome Measures:
Title
Change of disease severity measured by questionnaires
Description
Mean change of disease severity as assessed by a combination of the Self-Reported Impairments in Persons With Late Effects of Polio Rating Scale (SIPP-RS), the Inclusion Body Myositis Functional Rating Scale (IBM-FRS) and the World Health Organization Quality Of Life (WHOQOL-BREF)
Time Frame
Baseline to week 48
Title
Changes in Spinal cord MRI from week 48 to week 96
Description
Determination of the degree of spinal cord gray and white matter atrophy in PPS patients in comparison to healthy controls and its relationship to physical disability (QMT).
Determination of the degree of motor cortex atrophy in PPS patients compared to healthy controls and its association with spinal cord gray and white matter atrophy in PPS patients.
Determination of the association between spinal cord gray matter atrophy and microstructural alterations as assessed by diffusion tensor imaging.
Test the suitability of the applied, optimized imaging and post-processing methods for longitudinal data.
Time Frame
week 48 to week 96
Title
Changes in brain MRI from week 48 to week 96
Description
Determination of the degree of spinal cord gray and white matter atrophy in PPS patients in comparison to healthy controls and its relationship to physical disability (QMT).
Determination of the degree of motor cortex atrophy in PPS patients compared to healthy controls and its association with spinal cord gray and white matter atrophy in PPS patients.
Determination of the association between spinal cord gray matter atrophy and microstructural alterations as assessed by diffusion tensor imaging.
Test the suitability of the applied, optimized imaging and post-processing methods for longitudinal data.
Time Frame
week 48 to week 96
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Prior paralytic poliomyelitis with evidence of motor neuron loss
A period of partial or complete functional recovery after acute paralytic Poliomyelitis
Slowly progressive and persistent new muscle weakness or decreased endurance, with or without generalized fatigue, muscle atrophy, or muscle and joint pain. Symptoms that persist for at least a year
Exclusion of other neuromuscular, medical and skeletal abnormalities as causes of symptoms
Patients older than 18 years at time of Screening
ambulant
ability to walk 150m in the 6 min. Walking distance (6MWD)
females of childbearing potential willing to use contraceptive during the study
Exclusion Criteria:
Previous (3 months or less) or concomitant participation in any other therapeutic trial
Use of L-citrulline or L-arginine within the last 3 months
Known individual hypersensitivity to L-citrulline
Known or suspected malignancy
Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
Pregnant or breast-feeding women
Severe renal failure (calculated glomerular filtration rate (GFR) < 30 ml/min)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dirk Fischer, Prof.
Organizational Affiliation
University Children's Hospital Basel
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Basel, Children's Hospital
City
Basel
ZIP/Postal Code
4056
Country
Switzerland
12. IPD Sharing Statement
Citations:
PubMed Identifier
32676980
Citation
Gocheva V, Hafner P, Orsini AL, Schmidt S, Schaedelin S, Rueedi N, Rubino-Nacht D, Weber P, Fischer D. Health-related quality of life, self-reported impairments and activities of daily living in relation to muscle function in post-polio syndrome. J Patient Rep Outcomes. 2020 Jul 16;4(1):59. doi: 10.1186/s41687-020-00226-5.
Results Reference
derived
PubMed Identifier
28274276
Citation
Schmidt S, Gocheva V, Zumbrunn T, Rubino-Nacht D, Bonati U, Fischer D, Hafner P. Treatment with L-citrulline in patients with post-polio syndrome: study protocol for a single-center, randomised, placebo-controlled, double-blind trial. Trials. 2017 Mar 9;18(1):116. doi: 10.1186/s13063-017-1829-3.
Results Reference
derived
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L-Citrulline in Patients With Post-Polio Syndrome
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