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The purpose of the study is to show that the intake of L-citrulline improves muscle function in patients with Post-Polio Syndrome (PPS).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| L-citrulline | Experimental | 15 g L-citrulline p.o. per day (3x 5g) for 24 weeks |
|
| Placebo | Placebo Comparator | L-citrulline Placebo 3 times daily p.o. for 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 15g L-citrulline daily p.o. | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Mean change of 6 Minute Walking Distance(6MWD) | Baseline to week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean change of motor function measure (MFM) total score | Baseline to week 48 | |
| Change of quantitative muscle MRI (IDEAL-CPMG with Tâ‚‚ and lipid quantitation and dynamic 31P-MR spectroscopy) | Baseline to week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Change of disease severity measured by questionnaires | 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) | Baseline to week 48 |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Dirk Fischer, Prof. | University Children's Hospital Basel | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Basel, Children's Hospital | Basel | 4056 | Switzerland |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32676980 | Derived | 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. | |
| 28274276 |
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| ID | Term |
|---|---|
| D016262 | Postpoliomyelitis Syndrome |
| ID | Term |
|---|---|
| D011051 | Poliomyelitis |
| D009187 | Myelitis |
| D002494 | Central Nervous System Infections |
| D007239 | Infections |
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|
| Change of serum concentrations for markers of muscle necrosis | Baseline to week 48 |
| Change of serum concentrations for markers of oxidative stress | Baseline to week 48 |
| Change of serum concentrations for markers of nitrosative stress | Baseline to week 48 |
| Change of serum concentrations for markers of mitochondrial related genes | Baseline to week 48 |
| Changes in Spinal cord MRI from week 48 to week 96 |
| week 48 to week 96 |
| Changes in brain MRI from week 48 to week 96 |
| week 48 to week 96 |
| 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. |
| D004769 |
| Enterovirus Infections |
| D010850 | Picornaviridae Infections |
| D012327 | RNA Virus Infections |
| D014777 | Virus Diseases |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D019636 | Neurodegenerative Diseases |
| D000090862 | Neuroinflammatory Diseases |
| D009468 | Neuromuscular Diseases |