Patients With PH1 Treated With Lumasiran in FranceDAILY-LUMANCT06225882
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
An observational study of Patients With PH1 Treated With Lumasiran in France, run by Hospices Civils de Lyon at 7 sites in France.
Trial summary
Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutation in the AGXT gene encoding the hepatic peroxisomal enzyme AGT. Reduced AGT activity results in increased glyoxylate and oxalate production, causing the formation of kidney stones, nephrocalcinosis and renal failure. Read more about this study
- Who is it for?
- Ages 0 to 99 years
- Conditions the study is for
- Patients With PH1 Treated With Lumasiran in France
- What is being tested?
- Oxaluria evolution.
- How many people
- 100 participants
- Where
- 7 sitesFrance
- Study dates
- 1 Jan 2023to 31 Dec 2026
- Phase
- Observational
- 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 0 to 99 years
- All sexes
- Child, Adult, Older adult
Study population: Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.
Who may be able to join
- Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing.
Who cannot join
- Opposition of the patient or his legal representatives for minors.
- Not covered by social security.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
France7 sites, 7 recruiting
- Recruiting
CHU de Besançon
Besançon, 25030
Francois NOBILI, MD,PhD, 03 81 21 88 15 ext. +33, fnobili@chu-besancon.fr
Francois NOBILI, MD,PhD
- Recruiting
Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
Bron, 69500
Justine BACCHETTA, MD, justine.bacchetta@chu-lyon.fr
Justine BACCHETTA, MD
- Recruiting
Hopital Edouard Herriot
Lyon, 69003
Sandrine LEMOINE, PU-PH, 04 72 11 02 44, sandrine.lemoine01@chu-lyon.fr
Sandrine LEMOINE, PU-PH
- Recruiting
AP-HM - Timone Enfants
Marseille, 13385
Caroline ROUSSET-ROUVIERE, MD PHD, 04 91 38 80 50 ext. +33, caroline.rousset-rouviere@ap-hm.fr
Caroline ROUSSET-ROUVIERE, MD PHD
- Recruiting
Hôpital Européen G. Pompidou
Paris, 75015
Alexandre KARRAS, Pr, 1 56 09 37 60 ext. +33, alexandre.karras@egp.aphp.fr
Alexandre KARRAS, Pr
- Recruiting
CHU Paris - Hôpital Necker-Enfants Malades
Paris, 75743
Aude SERVAIS, PU PH, 01 44 49 54 16 ext. +33, aude.servais@aphp.fr
Olivia BOYER, MD PHD, 01 44 49 54 16 ext. +33, olivia.boyer@aphp.fr
- Recruiting
Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
Paris, Île-de-France Region, 75015
Bertrand KNEBELMANN, Pr, (0)144 495 241 ext. +33, bertrand.knebelmann@nck.aphp.fr
About this study
Official title: Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA
Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutation in the AGXT gene encoding the hepatic peroxisomal enzyme AGT. Reduced AGT activity results in increased glyoxylate and oxalate production, causing the formation of kidney stones, nephrocalcinosis and renal failure. Clinical trials of Lumasiran have provided information on the efficacy and safety of Lumasiran in the treatment of primary hyperoxaluria type 1. However, they do not provide data on long-term efficacy, safety and patient management. As part of the post-marketing follow-up of Lumasiran, in agreement with the authorities, this study proposes a retrospective and prospective follow-up over 5 years of pediatrics and adults patients treated in France with a standardized clinical, biological and radiological follow-up. The main objective is to monitor the evolution of PH1 parameters and particularly oxaluria before and after treatment.
- Being tested
- Oxaluria evolution.
- Keywords
- lumarisan, Primary hyperoxaluria type 1 (PH1), retrospective and prospective data.
- Medical subject headings
- Primary hyperoxaluria type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oxaluria evolution.Drug
To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment.
| Study group | Role | What they receive |
|---|---|---|
| Lumasiran | Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing. Receives: Drug: Oxaluria evolution. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evolution of oxaluria.
The evolution of oxaluria is followed by urinary biological analysis.
Measured: At baseline, At 1 month from the baseline, At 2 months from baseline, At 3 months from baseline, At 6 months from baseline, At 9 months from baseline, At 12 months from baseline, At 18 months from baseline, And 2 times a year until 5 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2024
- Study start1 Jan 2023
- Primary completion1 Jan 2024
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Patients With PH1 Treated With Lumasiran in FranceStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06225882
- Sponsor's protocol ID
- 69HCL22_0535
- Acronym
- DAILY-LUMA
- Status last verified
- Jan 2024
- First submitted
- 17 Jan 2024
- Last updated
- 26 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA
- ClinicalTrials.gov ID
- NCT06225882
- Acronym / sponsor ID
- DAILY-LUMA / 69HCL22_0535
- Keywords
- lumarisan, Primary hyperoxaluria type 1 (PH1), retrospective and prospective data.
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Jan 2023 (actual)
- Primary completion
- 1 Jan 2024 (actual)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 26 Jan 2024 (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.
Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutation in the AGXT gene encoding the hepatic peroxisomal enzyme AGT. Reduced AGT activity results in increased glyoxylate and oxalate production, causing the formation of kidney stones, nephrocalcinosis and renal failure. Clinical trials of Lumasiran have provided information on the efficacy and safety of Lumasiran in the treatment of primary hyperoxaluria type 1. However, they do not provide data on long-term efficacy, safety and patient management. As part of the post-marketing follow-up of Lumasiran, in agreement with the authorities, this study proposes a retrospective and prospective follow-up over 5 years of pediatrics and adults patients treated in France with a standardized clinical, biological and radiological follow-up. The main objective is to monitor the evolution of PH1 parameters and particularly oxaluria before and after treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lumasiran | – | Drug: Oxaluria evolution.Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing. |
Who may qualify
- Ages
- 0 to 99 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.
Key inclusion criteria (1 listed)
- Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing.
Key exclusion criteria (2 listed)
- Opposition of the patient or his legal representatives for minors.
- Not covered by social security.
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 |
|---|---|---|
| Oxaluria evolution.To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment. | Drug | Lumasiran |
Primary outcome
Evolution of oxaluria.
Time frame: At baseline, At 1 month from the baseline, At 2 months from baseline, At 3 months from baseline, At 6 months from baseline, At 9 months from baseline, At 12 months from baseline, At 18 months from baseline, And 2 times a year until 5 year
The evolution of oxaluria is followed by urinary biological analysis.
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 26 Jan 2024.
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.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Patients With PH1 Treated With Lumasiran in France, run by Hospices Civils de Lyon at 7 sites 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 2 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 26 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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?
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 Mélissa CLOAREC, Clinical Research Associate (04 27 85 51 54, melissa.cloarec@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evolution of oxaluria. (time frame: At baseline, At 1 month from the baseline, At 2 months from baseline, At 3 months from baseline, At 6 months from baseline, At 9 months from baseline, At 12 months from baseline, At 18 months from baseline, And 2 times a year until 5 year). 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 26 Jan 2024.