DrepanocytoseNCT02874170
Study of the Function and Muscle Metabolism in Drepanocytose Affected Patient During a Moderate Intensity Exercise
A study testing Spectroscopy magnetic resonance of phosphorus 31 in people with Drepanocytose, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
Drepanocytose disease is the first genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS), which in its deoxygenated form, polymerizes and leads to structural changes of red blood cells (RBC) which then take the shape of a sickle, become more fragile, more rigid and less deformable. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Drepanocytose
- What is being tested?
- Spectroscopy magnetic resonance of phosphorus 31
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteFrance
- Study dates
- Jul 2015to Jul 2019
- Phase
- Interventional
- 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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Patient affected by sickel cells and healthy volonteers presenting no contre indication to spectrometry exam.
Who cannot join
- Patient affected by hemoglobinopathies other than drepanocytose
Trial locations and contacts
1 site in 1 country.
Jean-Pierre MATTEI, MD contact
david BENDAHAN, PhD contact
France1 site, 1 recruiting
- Recruiting
Assistance Publique Hôpitaux de Marseille
Marseille, 13354
David BENDAHAN, PhD, davide.bendahan@univ-amu.fr
About this study
Drepanocytose disease is the first genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS), which in its deoxygenated form, polymerizes and leads to structural changes of red blood cells (RBC) which then take the shape of a sickle, become more fragile, more rigid and less deformable. The fragility of GR sickle causes their mass destruction, leading to chronic anemia (i.e. low levels of GR in the blood) associated with low tissue oxygenation. More rigid and less deformable, sickle GR tend to hang in the microvessels, leading to vaso-occlusive crises (CVO) particularly painful, can cause the failure of certain organs (spleen, kidneys, brain, lung, heart, liver , bone ...) and to life-threatening patients. Preliminary studies conducted on patients with drepanocytose disease (HbSS) have demonstrated changes of muscle tissue indicating a possible failure in the supply and use of oxygen. To date, the translation of this metabolic remodeling in the muscle work is not known.
This project's main objective is to evaluate muscle function in drepanocytose attempted patients. We hypothesized that muscle remodeling associated with sickle cell disease have a functional impact on strength and muscle metabolism. The main objective is to characterize the maximal voluntary plantar flexor muscles. The criteria for these targets will be based on the comparison between healthy subjects, carriers of the sickle cell trait (HbAS) and sickle cell patients (HbSS) oxygen saturation.
The strictly non-invasive approach proposed in this project will study the functional parameters of the muscle of sickle cell disease and the possible link with the clinical manifestations of the disease, including vaso-occlusive crisis, in which tissue oxygenation and pH plays a major role.
- Conditions
- Drepanocytose
- Being tested
- Spectroscopy magnetic resonance of phosphorus 31
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Spectroscopy magnetic resonance of phosphorus 31Other
| Study group | Role | What they receive |
|---|---|---|
| drepanocytose affected patient | Experimental | Not described Receives: Other: Spectroscopy magnetic resonance of phosphorus 31 |
| Healthy volunteers | Other | Not described Receives: Other: Spectroscopy magnetic resonance of phosphorus 31 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of phosphorus 31released during muscle exercise assesses by magnetic resonance spectroscopy
Measured: 2 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2016 (estimated)
- Study startJul 2015
- Primary completionJul 2018 (estimated)
- Study completionJul 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DrepanocytoseStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Urielle DESALBRES, Study director, Assistance Publique Hôpitaux de MarseilleJean-Pierre MATTEI, Md, Principal investigator, Assistance Publique Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02874170
- Sponsor's protocol ID
- 2014-39
- Other IDs
- 2014-A01486-41
- Status last verified
- Aug 2016
- First submitted
- 17 Aug 2016
- Last updated
- 22 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of the Function and Muscle Metabolism in Drepanocytose Affected Patient During a Moderate Intensity Exercise
- ClinicalTrials.gov ID
- NCT02874170
- Acronym / sponsor ID
- 2014-39
- Conditions
- Drepanocytose
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- Jul 2015
- Primary completion
- Jul 2018 (estimated)
- Study completion
- Jul 2019 (estimated)
- First posted
- 22 Aug 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.
Drepanocytose disease is the first genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS), which in its deoxygenated form, polymerizes and leads to structural changes of red blood cells (RBC) which then take the shape of a sickle, become more fragile, more rigid and less deformable. The fragility of GR sickle causes their mass destruction, leading to chronic anemia (i.e. low levels of GR in the blood) associated with low tissue oxygenation. More rigid and less deformable, sickle GR tend to hang in the microvessels, leading to vaso-occlusive crises (CVO) particularly painful, can cause the failure of certain organs (spleen, kidneys, brain, lung, heart, liver , bone ...) and to life-threatening patients. Preliminary studies conducted on patients with drepanocytose disease (HbSS) have demonstrated changes of muscle tissue indicating a possible failure in the supply and use of oxygen. To date, the translation of this metabolic remodeling in the muscle work is not known.
This project's main objective is to evaluate muscle function in drepanocytose attempted patients. We hypothesized that muscle remodeling associated with sickle cell disease have a functional impact on strength and muscle metabolism. The main objective is to characterize the maximal voluntary plantar flexor muscles. The criteria for these targets will be based on the comparison between healthy subjects, carriers of the sickle cell trait (HbAS) and sickle cell patients (HbSS) oxygen saturation.
The strictly non-invasive approach proposed in this project will study the functional parameters of the muscle of sickle cell disease and the possible link with the clinical manifestations of the disease, including vaso-occlusive crisis, in which tissue oxygenation and pH plays a major role.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| drepanocytose affected patient | Experimental | Other: Spectroscopy magnetic resonance of phosphorus 31 |
| Healthy volunteers | Other | Other: Spectroscopy magnetic resonance of phosphorus 31 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patient affected by sickel cells and healthy volonteers presenting no contre indication to spectrometry exam.
Key exclusion criteria (1 listed)
- Patient affected by hemoglobinopathies other than drepanocytose
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 |
|---|---|---|
| Spectroscopy magnetic resonance of phosphorus 31 | Other | Healthy volunteers, drepanocytose affected patient |
Primary outcome
Percentage of phosphorus 31released during muscle exercise assesses by magnetic resonance spectroscopy
Time frame: 2 hours
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2016.
- Individual participant dataSharing planned: No
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.
Jean-Pierre MATTEI, MD contact
david BENDAHAN, PhD contact
Urielle DESALBRES study director
Assistance Publique Hôpitaux de Marseille
Jean-Pierre MATTEI, Md principal investigator
Assistance Publique Hôpitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Spectroscopy magnetic resonance of phosphorus 31 in people with Drepanocytose, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 22 Aug 2016. 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 France. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jean-Pierre MATTEI, MD (jean-pierre.mattei@ap-hm.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of phosphorus 31released during muscle exercise assesses by magnetic resonance spectroscopy (time frame: 2 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Aug 2016.