Pyruvate Dehydrogenase Complex Deficiency DiseasePhase 2PDH-RAVICTINCT06887777
Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)
A Phase 2 study testing Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] in people with Pyruvate Dehydrogenase Complex Deficiency Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
This is a phase II, multicenter, prospective, non-comparative clinical trial to assess the efficacy and safety of the treatment of pyruvate dehydrogenase deficiency (PDH) patients with glycerol phenylbutyrate (Ravicti®). Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Pyruvate Dehydrogenase Complex Deficiency Disease
- What is being tested?
- Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteFrance
- Study dates
- 1 Oct 2025to 1 Apr 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Child from 2 to 17 years of age Or
- Adult from 18 to 25 years of age
- With a PDH deficiency confirmed by molecular biology:
- a class 4 or 5- missense variant on the PDHA1 gene or
- one homozygous variant or two mixed heterozygous variants of class 4 or 5 that are missense variants on PDHB or DLAT genes or
- one homozygous variant or two mixed heterozygous variants of class 4 or 5 on PDHX genes (including non-sense and frameshift variants, and intragenic deletions
Show all 9Show fewer
- For females of childbearing potential, negative bHCG and effective method of contraception (sexual abstinence, hormonal contraception containing ethinylestradiol and levonorgestrel, intrauterine device or hormone-releasing system, cap, diaphragm or sponge with spermicide, condom) until 30 days after the end of study. For male, an effective method of contraception (sexual abstinence, condom) until 30 days after the end of study
- Signature of consent by the legal representative
- Beneficiary of a social security coverage (affiliated or entitled)
Who cannot join
- Patient with E3 deficiency due to pathogenic mutation in DLD gene
- Patient with non-sense mutation on PDHB or DLAT gene, and male patient with non-sense mutation or PDHA1 gene.
- Patient with planned hip or scoliosos surgery during the study timeframe.
- Patient whose parents / legal representative refuse flu vaccine.
- Treatment change during the last 3 months prior inclusion (ketogenic diet and/or B1 vitamin)
- Hypersensitivity to Glycerol Phenylbutyrate or to any of the excipients
Show all 9Show fewer
- No disease requiring Glycerol Phenylbutyrate (Hyperammonemia due to urea cycle disease or other aetiology)
- Pregnant or breastfeeding women
- Participation to another clinical trial on medicinal products for human use
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Universitaire Necker - Enfants Malades
Paris, France, 75015
Pascale De Lonlay, MD, 01 44 49 58 52 ext. +33, pascale.delonlay@aphp.fr
About this study
Official title: A Phase II, Multicentric, Prospective, Non-comparative Clinical Trial to Assess the Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency (PDH) Patients With Glycerol Phenybutyrate (RAVICTI®)
This is a phase II, multicenter, prospective, non-comparative clinical trial to assess the efficacy and safety of the treatment of pyruvate dehydrogenase deficiency (PDH) patients with glycerol phenylbutyrate (Ravicti®).
The trial will be conducted with three visits: 3 day hospitalizations including clinical consultations and paramedical procedures at Month 0 (M0), Month 3 (M3), Month 6 (M6).
Read more
During all the research, AE/SAE and treatment compliance will be recorded. Patients will keep their usual treatment during the study time: vitamin B1, ketogenic diet, possible anti-epileptic and/or dystonic treatment(s).
The efficacy on fatigue, polyhandicap, neurodevelopmental functioning, quality of life and seizure amount for epileptic patients will be evaluated at 0, 3 and 6 months. Biological balance will be assed with regular quantification of PDH deficiency markers, lactate concentration and amino acid plasma quantification.
Read the detailed description
PDH deficiencies are mainly characterized by primary lactic acidosis associated with neurological disorders. The diagnosis is suspected in the presence of an increase of pyruvate and lactate with a normal or low lactate/pyruvate ratio, especially in postprandial period, in the blood and/or cerebrospinal fluid.
Neurological disorders are explained by the energy deficit associated with the absence of aerobic oxidation of glucose, their preferential energy substrate, which cannot be compensated by the catabolism of fatty acids.
Phenylbutyrate was therefore proposed to increase the enzymatic activity of PDH in PDH deficits, particularly in patient cells and mouse model: it has reduced the phosphorylated form in these models and thus increased the enzymatic activity of the PDH complex. Phenylbutyrate would be more active when the PDH deficit is linked to missense variants, and less effective in the presence of non-meaning variant, with the exception of variants on the PDHX gene which are mostly non-sense variants.
The study plan is to treat these patients with Glycerol Phenylbutyrate (Ravicti®) 1.1 g/mL oral fluid (or in enteral tube or gastrostomy). Sodium Phenylbutyrate and Glycerol Phenylbutyrate are commonly used in inherited metabolic diseases in urea cycle diseases, for chelating ammonia, in children and adults. The expectation is to obtain an improvement of patients' fatigue and neurodevelopmental disability for PDH patients. Phenylbutyrate prevents PDH kinase from phosphorylating the PDH complex, allowing the complex to remain active. It acts on different isoforms of PDH kinase.
- Being tested
- Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
- Keywords
- Pyruvate dehydrogenase (PDH), Enzyme, PDH deficiency, Glycerol Phenylbutyrate, Fatigue
- Medical subject headings
- Pyruvate Dehydrogenase Complex Deficiency Disease, Fatigue
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]Drug
The patients will orally take a dose of 200 mg/kg/day three times a day during meals: breakfast, lunch or afternoon snack and diner for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Glycerol phenylbutyrate treatment | Experimental | The patients will orally take a dose of 200 mg/kg/day three times a day during meals: breakfast, lunch or afternoon snack and diner for 6 months.
Questionnaires will be answered by parents or the patient's legal guardian.
An additional hospital visit à 3 months following treatment start will be conducted with 6 blood drawing Receives: Drug: Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy on fatigue at 6 months
The efficacy of Glycerol Phenylbutyrate treatment on fatigue at 6 months will be evaluated by the Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL MFS) at Month 0 and Month 6. Results range from 0 to 72.
Measured: 6 months
Secondary outcomes (12)
Efficacy on fatigue at 3 months
The efficacy of Glycerol Phenylbutyrate treatment on fatigue at 3 months will be evaluated by the Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL MFS) at Month 0 and Month 3. Results range from 0 to 72.
Measured: 3 months
Efficacy on polyhandicap
The efficacy of Glycerol Phenylbutyrate treatment on polyhandicap, will be evaluated by the Polyhandicap severity scale at Month 0 and Month 6, performed by a psychomotrician, a physiotherapist or a medical physician. Results range from 0 to 93.
Measured: 6 months
Efficacy on neurodevelopmental functioning
The efficacy of Glycerol Phenylbutyrate treatment on neurodevelopmental functions will be evaluated by a semi-structured interview with the Vineland Adaptive Behavior Scales second edition (VABS-II) 20 at Month 0 and Month 6. Results range from 0 to 160.
Measured: 6 months
Efficacy on epilepsy
The efficacy of Glycerol Phenylbutyrate treatment on the number of seizures during the last 3 months using patient diary, and electroencephalogram (EEG) for epileptic patients at Month 6 compared to Month 0.
Measured: 6 months
Efficacy on the biological balance
Evaluation of the efficacy of Glycerol Phenylbutyrate treatment on the biological balance by quantification of the decrease in pyruvate concentration, marker of PDH deficiency, and lactate concentration (points Redox performed before and after 3 meals), and the quantification of amino acids in plasma notably alanine and proline (markers of hyperlactatemia) (aminoacid chromatography) at 3 months.
Measured: 3 months
Efficacy on the biological balance
Evaluation of the efficacy of Glycerol Phenylbutyrate treatment on the biological balance by quantification of the decrease in pyruvate concentration, marker of PDH deficiency, and lactate concentration (points Redox performed before and after 3 meals), and the quantification of amino acids in plasma notably alanine and proline (markers of hyperlactatemia) (aminoacid chromatography) at 6 months.
Measured: 6 months
Efficacy on quality of life
Evaluation of the efficacy of Glycerol Phenylbutyrate treatment on quality of life of persons with polyhandicap at 3 months assessed by using the PolyQol questionnaire 22 at Month 0 and Month 3. Results range from 20 to 100.
Measured: 3 months
Efficacy on quality of life
Evaluation of the efficacy of Glycerol Phenylbutyrate treatment on quality of life of persons with polyhandicap at 6 months assessed by using the PolyQol questionnaire at Month 0 and Month 6. Results range from 20 to 100.
Measured: 6 months
Treatment tolerance
Glycerol Phenylbutyrate treatment tolerance assessment at 3 months [allergy, side effects, amino acid chromatography (glutamine and other amino acid levels) in plasma].
Measured: 3 months
Treatment tolerance
Glycerol Phenylbutyrate treatment tolerance assessment at 6 months [allergy, side effects, amino acid chromatography (glutamine and other amino acid levels) in plasma].
Measured: 6 months
Treatment compliance
Glycerol Phenylbutyrate treatment compliance assessment at 3 months.
Measured: 3 months
Treatment compliance
Glycerol Phenylbutyrate treatment compliance assessment at 6 months.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2025
- Study start1 Oct 2025
- Primary completion1 Apr 2026 (estimated)
- Study completion1 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Pyruvate Dehydrogenase Complex Deficiency DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06887777
- Sponsor's protocol ID
- APHP230834
- Other IDs
- 2024-516410-38-00
- Acronym
- PDH-RAVICTI
- Status last verified
- Mar 2026
- First submitted
- 10 Mar 2025
- Last updated
- 31 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kolobova E, Tuganova A, Boulatnikov I, Popov KM. Regulation of pyruvate dehydrogenase activity through phosphorylation at multiple sites. Biochem J. 2001 Aug 15;358(Pt 1):69-77. doi: 10.1042/0264-6021:3580069.
PubMed 11485553Strumilo S. Short-term regulation of the alpha-ketoglutarate dehydrogenase complex by energy-linked and some other effectors. Biochemistry (Mosc). 2005 Jul;70(7):726-9. doi: 10.1007/s10541-005-0177-1.
PubMed 16097935Strumilo S. Short-term regulation of the mammalian pyruvate dehydrogenase complex. Acta Biochim Pol. 2005;52(4):759-64. Epub 2005 Jul 11.
PubMed 16025163Ferriero R, Brunetti-Pierri N. Phenylbutyrate increases activity of pyruvate dehydrogenase complex. Oncotarget. 2013 Jun;4(6):804-5. doi: 10.18632/oncotarget.1000. No abstract available.
PubMed 23868807Ferriero R, Boutron A, Brivet M, Kerr D, Morava E, Rodenburg RJ, Bonafe L, Baumgartner MR, Anikster Y, Braverman NE, Brunetti-Pierri N. Phenylbutyrate increases pyruvate dehydrogenase complex activity in cells harboring a variety of defects. Ann Clin Transl Neurol. 2014 Jul;1(7):462-70. doi: 10.1002/acn3.73. Epub 2014 Jun 19.
PubMed 25356417Ferriero R, Iannuzzi C, Manco G, Brunetti-Pierri N. Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate. J Inherit Metab Dis. 2015 Sep;38(5):895-904. doi: 10.1007/s10545-014-9808-2. Epub 2015 Jan 20.
PubMed 25601413Ferriero R, Manco G, Lamantea E, Nusco E, Ferrante MI, Sordino P, Stacpoole PW, Lee B, Zeviani M, Brunetti-Pierri N. Phenylbutyrate therapy for pyruvate dehydrogenase complex deficiency and lactic acidosis. Sci Transl Med. 2013 Mar 6;5(175):175ra31. doi: 10.1126/scitranslmed.3004986.
PubMed 23467562
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicentric, Prospective, Non-comparative Clinical Trial to Assess the Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency (PDH) Patients With Glycerol Phenybutyrate (RAVICTI®)
- ClinicalTrials.gov ID
- NCT06887777
- Acronym / sponsor ID
- PDH-RAVICTI / APHP230834
- Keywords
- Pyruvate dehydrogenase (PDH), Enzyme, PDH deficiency, Glycerol Phenylbutyrate, Fatigue
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 1 Oct 2025 (actual)
- Primary completion
- 1 Apr 2026 (estimated)
- Study completion
- 1 Apr 2027 (estimated)
- First posted
- 20 Mar 2025 (actual)
- 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.
This is a phase II, multicenter, prospective, non-comparative clinical trial to assess the efficacy and safety of the treatment of pyruvate dehydrogenase deficiency (PDH) patients with glycerol phenylbutyrate (Ravicti®).
The trial will be conducted with three visits: 3 day hospitalizations including clinical consultations and paramedical procedures at Month 0 (M0), Month 3 (M3), Month 6 (M6).
During all the research, AE/SAE and treatment compliance will be recorded. Patients will keep their usual treatment during the study time: vitamin B1, ketogenic diet, possible anti-epileptic and/or dystonic treatment(s).
The efficacy on fatigue, polyhandicap, neurodevelopmental functioning, quality of life and seizure amount for epileptic patients will be evaluated at 0, 3 and 6 months. Biological balance will be assed with regular quantification of PDH deficiency markers, lactate concentration and amino acid plasma quantification.
Detailed description
PDH deficiencies are mainly characterized by primary lactic acidosis associated with neurological disorders. The diagnosis is suspected in the presence of an increase of pyruvate and lactate with a normal or low lactate/pyruvate ratio, especially in postprandial period, in the blood and/or cerebrospinal fluid.
Neurological disorders are explained by the energy deficit associated with the absence of aerobic oxidation of glucose, their preferential energy substrate, which cannot be compensated by the catabolism of fatty acids.
Phenylbutyrate was therefore proposed to increase the enzymatic activity of PDH in PDH deficits, particularly in patient cells and mouse model: it has reduced the phosphorylated form in these models and thus increased the enzymatic activity of the PDH complex. Phenylbutyrate would be more active when the PDH deficit is linked to missense variants, and less effective in the presence of non-meaning variant, with the exception of variants on the PDHX gene which are mostly non-sense variants.
The study plan is to treat these patients with Glycerol Phenylbutyrate (Ravicti®) 1.1 g/mL oral fluid (or in enteral tube or gastrostomy). Sodium Phenylbutyrate and Glycerol Phenylbutyrate are commonly used in inherited metabolic diseases in urea cycle diseases, for chelating ammonia, in children and adults. The expectation is to obtain an improvement of patients' fatigue and neurodevelopmental disability for PDH patients. Phenylbutyrate prevents PDH kinase from phosphorylating the PDH complex, allowing the complex to remain active. It acts on different isoforms of PDH kinase.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Glycerol phenylbutyrate treatment | Experimental | Drug: Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]The patients will orally take a dose of 200 mg/kg/day three times a day during meals: breakfast, lunch or afternoon snack and diner for 6 months. Questionnaires will be answered by parents or the patient's legal guardian. An additional hospital visit à 3 months following treatment start will be conducted with 6 blood drawing |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Child from 2 to 17 years of age Or
- Adult from 18 to 25 years of age
- With a PDH deficiency confirmed by molecular biology:
- For females of childbearing potential, negative bHCG and effective method of contraception (sexual abstinence, hormonal contraception containing ethinylestradiol and levonorgestrel, intrauterine device or hormone-releasing system, cap, diaphragm or sponge with spermicide, condom) until 30 days after the end of study. For male, an effective method of contraception (sexual abstinence, condom) until 30 days after the end of study
- Signature of consent by the legal representative
Key exclusion criteria (9 listed)
- Patient with E3 deficiency due to pathogenic mutation in DLD gene
- Patient with non-sense mutation on PDHB or DLAT gene, and male patient with non-sense mutation or PDHA1 gene.
- Patient with planned hip or scoliosos surgery during the study timeframe.
- Patient whose parents / legal representative refuse flu vaccine.
- Treatment change during the last 3 months prior inclusion (ketogenic diet and/or B1 vitamin)
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 |
|---|---|---|
| Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]The patients will orally take a dose of 200 mg/kg/day three times a day during meals: breakfast, lunch or afternoon snack and diner for 6 months. | Drug | Glycerol phenylbutyrate treatment |
Primary outcome
Efficacy on fatigue at 6 months
Time frame: 6 months
The efficacy of Glycerol Phenylbutyrate treatment on fatigue at 6 months will be evaluated by the Pediatric Quality of Life Multidimensional Fatigue Scale (PedsQL MFS) at Month 0 and Month 6. Results range from 0 to 72.
12 secondary outcomes
Efficacy on fatigue at 3 months
Time frame: 3 months
Efficacy on polyhandicap
Time frame: 6 months
Efficacy on neurodevelopmental functioning
Time frame: 6 months
Efficacy on epilepsy
Time frame: 6 months
Efficacy on the biological balance
Time frame: 3 months
Efficacy on the biological balance
Time frame: 6 months
Efficacy on quality of life
Time frame: 3 months
Efficacy on quality of life
Time frame: 6 months
Treatment tolerance
Time frame: 3 months
Treatment tolerance
Time frame: 6 months
Treatment compliance
Time frame: 3 months
Treatment compliance
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Background: PubMed 11485553Kolobova E, Tuganova A, Boulatnikov I, Popov KM. Regulation of pyruvate dehydrogenase activity through phosphorylation at multiple sites. Biochem J. 2001 Aug 15;358(Pt 1):69-77. doi: 10.1042/0264-6021:3580069.
- Background: PubMed 16097935Strumilo S. Short-term regulation of the alpha-ketoglutarate dehydrogenase complex by energy-linked and some other effectors. Biochemistry (Mosc). 2005 Jul;70(7):726-9. doi: 10.1007/s10541-005-0177-1.
- Background: PubMed 16025163Strumilo S. Short-term regulation of the mammalian pyruvate dehydrogenase complex. Acta Biochim Pol. 2005;52(4):759-64. Epub 2005 Jul 11.
- Background: PubMed 23868807Ferriero R, Brunetti-Pierri N. Phenylbutyrate increases activity of pyruvate dehydrogenase complex. Oncotarget. 2013 Jun;4(6):804-5. doi: 10.18632/oncotarget.1000. No abstract available.
- Background: PubMed 25356417Ferriero R, Boutron A, Brivet M, Kerr D, Morava E, Rodenburg RJ, Bonafe L, Baumgartner MR, Anikster Y, Braverman NE, Brunetti-Pierri N. Phenylbutyrate increases pyruvate dehydrogenase complex activity in cells harboring a variety of defects. Ann Clin Transl Neurol. 2014 Jul;1(7):462-70. doi: 10.1002/acn3.73. Epub 2014 Jun 19.
- Background: PubMed 25601413Ferriero R, Iannuzzi C, Manco G, Brunetti-Pierri N. Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate. J Inherit Metab Dis. 2015 Sep;38(5):895-904. doi: 10.1007/s10545-014-9808-2. Epub 2015 Jan 20.
- Background: PubMed 23467562Ferriero R, Manco G, Lamantea E, Nusco E, Ferrante MI, Sordino P, Stacpoole PW, Lee B, Zeviani M, Brunetti-Pierri N. Phenylbutyrate therapy for pyruvate dehydrogenase complex deficiency and lactic acidosis. Sci Transl Med. 2013 Mar 6;5(175):175ra31. doi: 10.1126/scitranslmed.3004986.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] in people with Pyruvate Dehydrogenase Complex Deficiency Disease, run by Assistance Publique - Hôpitaux de Paris 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 9 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 31 Mar 2026. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pascale De Lonlay, MD, PhD (01 44 49 58 52 ext. +33, pascale.delonlay@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy on fatigue at 6 months (time frame: 6 months). 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 NCT06887777, which also lists 7 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 31 Mar 2026.