Post-Polio SyndromePhase 3NCT02801071
L-Citrulline in Patients With Post-Polio Syndrome
A Phase 3 study testing 15g L-citrulline daily p.o. in people with Post-Polio Syndrome, run by Dirk Fischer at 1 site in Switzerland.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Post-Polio Syndrome
- What is being tested?
- 15g L-citrulline daily p.o.
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteSwitzerland
- Study dates
- 14 Jun 2016to 5 Oct 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: "Treatment With L-citrulline in Patients With Post-polio Syndrome - a Randomized Double Blind Placebo Controlled Study"
The purpose of the study is to show that the intake of L-citrulline improves muscle function in patients with Post-Polio Syndrome (PPS).
Read the 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.
- Conditions
- Post-Polio Syndrome
- Being tested
- 15g L-citrulline daily p.o., Placebo
- Keywords
- Post-Polio Syndrome, PPS
- Medical subject headings
- Postpoliomyelitis Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
15g L-citrulline daily p.o.Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| L-citrulline | Experimental | 15 g L-citrulline p.o. per day (3x 5g) for 24 weeks Receives: Drug: 15g L-citrulline daily p.o. |
| Placebo | Placebo comparator | L-citrulline Placebo 3 times daily p.o. for 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change of 6 Minute Walking Distance(6MWD)
Measured: Baseline to week 48
Secondary outcomes (6)
Mean change of motor function measure (MFM) total score
Measured: Baseline to week 48
Change of quantitative muscle MRI (IDEAL-CPMG with T₂ and lipid quantitation and dynamic 31P-MR spectroscopy)
Measured: Baseline to week 48
Change of serum concentrations for markers of muscle necrosis
Measured: Baseline to week 48
Change of serum concentrations for markers of oxidative stress
Measured: Baseline to week 48
Change of serum concentrations for markers of nitrosative stress
Measured: Baseline to week 48
Change of serum concentrations for markers of mitochondrial related genes
Measured: Baseline to week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2016 (estimated)
- Study start14 Jun 2016
- Primary completion15 Aug 2017
- Study completion5 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Post-Polio SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dirk Fischer
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Dirk Fischer, University Hospital, Basel, Switzerland
- Investigators
- Dirk Fischer, Prof., Principal investigator, University Children's Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02801071
- Sponsor's protocol ID
- PPS
- Status last verified
- Jan 2019
- First submitted
- 6 Jun 2016
- Last updated
- 23 Jan 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 32676980Schmidt 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.
PubMed 28274276
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- "Treatment With L-citrulline in Patients With Post-polio Syndrome - a Randomized Double Blind Placebo Controlled Study"
- ClinicalTrials.gov ID
- NCT02801071
- Acronym / sponsor ID
- PPS
- Conditions
- Post-Polio Syndrome
- Keywords
- Post-Polio Syndrome, PPS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Dirk FischerUniversity, hospital or other
- Study start
- 14 Jun 2016 (actual)
- Primary completion
- 15 Aug 2017 (actual)
- Study completion
- 5 Oct 2018 (actual)
- First posted
- 15 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| L-citrulline | Experimental | Drug: 15g L-citrulline daily p.o.15 g L-citrulline p.o. per day (3x 5g) for 24 weeks |
| Placebo | Placebo comparator | Drug: PlaceboL-citrulline Placebo 3 times daily p.o. for 24 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 15g L-citrulline daily p.o. | Drug | L-citrulline |
| Placebo | Drug | Placebo |
Primary outcome
Mean change of 6 Minute Walking Distance(6MWD)
Time frame: Baseline to week 48
6 secondary outcomes
Mean change of motor function measure (MFM) total score
Time frame: Baseline to week 48
Change of quantitative muscle MRI (IDEAL-CPMG with T₂ and lipid quantitation and dynamic 31P-MR spectroscopy)
Time frame: Baseline to week 48
Change of serum concentrations for markers of muscle necrosis
Time frame: Baseline to week 48
Change of serum concentrations for markers of oxidative stress
Time frame: Baseline to week 48
Change of serum concentrations for markers of nitrosative stress
Time frame: Baseline to week 48
Change of serum concentrations for markers of mitochondrial related genes
Time frame: Baseline to week 48
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2019.
- Derived from this study: PubMed 32676980Gocheva 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.
- Derived from this study: PubMed 28274276Schmidt 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dirk Fischer, Prof. principal investigator
University Children's Hospital Basel
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing 15g L-citrulline daily p.o. in people with Post-Polio Syndrome, run by Dirk Fischer at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change of 6 Minute Walking Distance(6MWD) (time frame: Baseline to week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02801071, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2019.