L-Citrulline in Patients With Post-Polio Syndrome

Post-Polio Syndrome Clinical Trial

Official title:

"Treatment With L-citrulline in Patients With Post-polio Syndrome - a Randomized Double Blind Placebo Controlled Study"

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02801071
• Other study ID #: PPS
• Status: Completed
• Phase: Phase 3
• Start date: June 14, 2016
• Est. completion date: October 5, 2018

Study information

• Verified date: January 2019
• Source: University Hospital, Basel, Switzerland
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: October 5, 2018
• Est. primary completion date: August 15, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Poliomyelitis
• Post-Polio Syndrome
• Postpoliomyelitis Syndrome
• Syndrome

Intervention

Drug:
• 15g L-citrulline daily p.o.

• Placebo

Locations

Country	Name	City	State
Switzerland	University of Basel, Children's Hospital	Basel

Sponsors (1)

Lead Sponsor	Collaborator
Dirk Fischer

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	Changes in Spinal cord MRI from week 48 to week 96	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.	week 48 to week 96
Other	Changes in brain MRI from week 48 to week 96	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.	week 48 to week 96
Primary	Mean change of 6 Minute Walking Distance(6MWD)		Baseline to week 48
Secondary	Mean change of motor function measure (MFM) total score		Baseline to week 48
Secondary	Change of quantitative muscle MRI (IDEAL-CPMG with T2 and lipid quantitation and dynamic 31P-MR spectroscopy)		Baseline to week 48
Secondary	Change of serum concentrations for markers of muscle necrosis		Baseline to week 48
Secondary	Change of serum concentrations for markers of oxidative stress		Baseline to week 48
Secondary	Change of serum concentrations for markers of nitrosative stress		Baseline to week 48
Secondary	Change of serum concentrations for markers of mitochondrial related genes		Baseline to week 48