CystinuriaPhase 2/3NCT04137978

Study Evaluating Patients With Cystinuria

A Phase 2/3 study testing ADV7103 in people with Cystinuria, run by Advicenne Pharma at 13 sites in 2 countries.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial summary

This is an open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and acceptability of alkalising treatments at long-term in patients with cystinuria. Read more about this study

Who is it for?: Ages 6 months to 70 years
Conditions the study is for: Cystinuria
What is being tested?: ADV7103
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 13 sitesin 2 countries
Study dates: 1 May 2024to Aug 2025
Phase: Phase 2/3
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 6 months to 70 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

For ADV7103 cohort:
Patient who has participated to and completed the previous B12CS Study or B13CS Study.
Patient for whom the safety and tolerability of ADV7103 were satisfactory during B12CS Study or B13CS Study.
Female patient of childbearing potential (defined by the Clinical Trial Facilitation Group (CTFG) as woman fertile, following menarche until becoming post-menopausal unless permanently sterile*) using an acceptable effective birth control method and having a negative pregnancy test at the inclusion, or a woman postmenopausal* or a woman surgically sterilized*.
Patient and/or parents or legal representative(s) who is(are) willing and able to participate in the study, to understand and to comply with study procedures for the entire length of the study.
Patient or parents or legal representative(s) who has(have) provided a signed written informed consent.

Show all 17Show fewer

Patient of ≤17 Patient of ≤17 years of age for whom the assent has been collected or has been tried to be collected.
Patient who is affiliated to a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research.
For Standard of Care cohort:
Patient who has a diagnosis of cystinuria based on medical diagnosis (at least one previous or current episode of calculus of cystine, and/or one previous or current episode of cystine crystalluria) or on genetic diagnosis (only for patients of Subset 4).
Patient treated with an alkalising treatment at a well-adapted dose (defined as a daily dose deemed by the investigator aiming to maintain overtime urinary pH value ≥ 7.0 and/or compatible with an acceptable safety profile and/or patient's constraints or compliance).
Patient male or female, including child aged between 6 months and 17 years old and adult aged ≥ 18 years old up to 70 years old.
Female patient of childbearing potential (defined by the Clinical Trial Facilitation Group (CTFG) as woman fertile, following menarche until becoming post-menopausal unless permanently sterile*) using an acceptable effective birth control method and having a negative pregnancy test at the inclusion, or a woman postmenopausal* or a woman surgically sterilized*.
Patient and/or parents or legal representative(s) who is(are) willing and able to participate in the study, to understand and to comply with study procedures for the entire length of the study.
Patient or parents or legal representative(s) who has/have provided a signed written informed consent.
Patient of ≤17 years of age for whom the assent has been collected or has been tried to be collected.
Patient who is affiliated to a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research.

Who cannot join

For ADV7103 cohort:
Patient who has not participated to B12CS study or B13CS study
Patient for whom any safety issue could contraindicate her/his participation to the extension study
For Standard of Care cohort:
Patient that is receiving the second line therapy -- cystine chelating agents (sulfhydryl compounds).
Patient who presents kalaemia > 5.0 mmol/L.

Show all 18Show fewer

Patient who presents a moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 according to Schwartz formula for the children and both MDRDs and CKD-EPI for adults).
Patient who presents - barring the study disease - any previous or concurrent medical condition or any laboratory or clinical findings or any other condition that in the opinion of the investigator would be negatively affected by the study product or that would affect the study product or that precludes his participation, e.g. uncontrolled diabetes mellitus, adrenal insufficiency, cardiac impairment, repeated infections, metabolic alkalosis, chronic diarrhoea.
Female patient who is pregnant or breast-feeding.
Patient who cannot stop potassium sparing diuretics (e.g. antagonists of aldosterone as such spironolactone, canrenoate and eplerenone, amiloride, triamterene), angiotensin converting enzyme inhibitors, angiotensin II receptor antagonists, tacrolimus, potassium desodic salts.
Patient who received any medication that could interfere with the study treatment within 4 weeks before the inclusion in the study, including angiotensin converting enzyme inhibitors, angiotensin II receptor antagonists, tacrolimus, potassium desodic salts, antibiotics.
Patient who received potassium sparing diuretics 6 weeks before the inclusion in the study.
Patient who is admitted to hospital in emergency settings.
Patient who participated in a clinical trial within the last 3 months before enrolment.
Patient who is at risk of non-compliance in the judgment of the investigator.
Patient who could present any other condition, which in the opinion of the investigator, would preclude participation in the study.
Patient who cannot be contacted in case of emergency.
Patient under any administrative or legal supervision.

Trial locations and contacts

13 sites in 2 countries.

Country

City, postcode or hospital

13 sites in 2 countries

France11 sites

CHU de Bordeaux - Hôpital Pellegrin
Bordeaux, 33076
Centre Hospitalier Universitaire de Lyon
Bron, 69500
CHU Grenoble
Grenoble, 38043
CHRU Lille
Lille, 59000
CHU Pitié-Salpétrière
Paris, 15013
Hôpital Necker AP-HP
Paris, 75015
Hôpital Necker Enfants Malades
Paris, 75015
Hôpital Ténon - Explorations fonctionnelles Mutlidisciplinaires et INSERM UMR S 1155
Paris, 75020
Hôpital Américain CHU de Reims
Reims, 51092
CHU Reims
Reims, 51100
CHU Purpan
Toulouse, 31059

Other trials in France

Belgium2 sites

Cliniques Universitaires Saint-Luc
Brussels
UZ Leuven, Gasthuisberg Hospital
Leuven

Other trials in Belgium

About this study

Official title: Open Label, Multicentre Study, Evaluating the Safety, Tolerability, Efficacy, Compliance and Acceptability of Alkalising Treatments at Long-term in Patients With Cystinuria

This is an open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and acceptability of alkalising treatments at long-term in patients with cystinuria.

Read the detailed description

B14CS study is an extension study which follows the B12CS-B13CS study. Patients who have participated in B12CS-B13CS Study will have the possibility to switch to B14CS Study for a long-term treatment period (2 year-period).

The B14CS Study is an open-label long-term study and including 2 types of cohorts.

ADV7103 Cohort: Cohort of patients who will have completed either B12CS or B13CS Study. Following an optional titration period (for B12CS Study subjects), all patients will be maintained at their ADV7103 optimal dose for a 2-year-period.
SoC Cohort: Cohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period. This cohort is designed in order to contextualize the safety, tolerability and efficacy of the long-term ADV7103 treatment.

The B14CS Study will be composed of 3 periods (more detailed hereafter):

Lead-in Period: Period of 3 weeks, during which the treatment (ADV7103 or SoC) will be taken at the optimal dose as previously defined (Maintenance Phase). The Lead-in Period can include a Titration Phase, for ADV7103 B12CS Cohort only, in order to define the individual optimal dose of ADV7103 for the patients of this cohort;
Assessment Period: Period of 7 days to evaluate the effect of study products (ADV7103 and SoC) at the optimal dose;
Follow-up Period: Period of 23 months to evaluate the effects of the study products (ADV7103 and SoC) at long-term.

Conditions: Cystinuria
Being tested: ADV7103, Standard of Care
Medical subject headings: Cystinuria

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ADV7103Drug
Also called Potassium Citrate and Potassium Bicarbonate
Patients receive ADV7103 twice a day at optimal dose.
Standard of CareDrug
Cohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period.

Study groups in NCT04137978
Study group	Role	What they receive
ADV7103	Active comparator	Patients receive ADV7103 twice a day at optimal dose. Each dose of ADV7103 contains a fixed ratio of 1/3 of ADV7103-CK (potassium citrate) and 2/3 of ADV7103-BK (potassium bicarbonate) based on the mass of active substances. Other Names: • Potassium Citrate and Potassium Bicarbonate Receives: Drug: ADV7103
Standard of care comparator	Active comparator	Alkalinising treatment (SoC) taken at the usual dose and frequency Receives: Drug: Standard of Care

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage of urinary pH values ≥ 7.0 during 24h on Day 7 (after ADV7103 treatment period)
To evaluate the safety and the tolerability of ADV7103 and standard of care (SoC) after a long-term treatment.
Measured: 7 Days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 May 2024Aug 2025

First posted24 Oct 2019
Study start1 May 2024 (estimated)
Primary completionJun 2025 (estimated)
Study completionAug 2025 (estimated)

Why it stopped: Unspecified business decision/strategic reason

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for CystinuriaStudies 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 2/3
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04137978
Sponsor's protocol ID: B14CS
Status last verified: Mar 2024
First submitted: 18 Oct 2019
Last updated: 12 Mar 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cystinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Open Label, Multicentre Study, Evaluating the Safety, Tolerability, Efficacy, Compliance and Acceptability of Alkalising Treatments at Long-term in Patients With Cystinuria
ClinicalTrials.gov ID: NCT04137978
Acronym / sponsor ID: B14CS
Conditions: Cystinuria
Phase: Phase 2/3
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Unspecified business decision/strategic reason
Enrolment: 0 participants (actual)
Lead sponsor: Advicenne PharmaIndustry (pharma, biotech, devices)
Study start: 1 May 2024 (estimated)
Primary completion: Jun 2025 (estimated)
Study completion: Aug 2025 (estimated)
First posted: 24 Oct 2019 (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 an open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and acceptability of alkalising treatments at long-term in patients with cystinuria.

Detailed description

The B14CS Study is an open-label long-term study and including 2 types of cohorts.

ADV7103 Cohort: Cohort of patients who will have completed either B12CS or B13CS Study. Following an optional titration period (for B12CS Study subjects), all patients will be maintained at their ADV7103 optimal dose for a 2-year-period.
SoC Cohort: Cohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period. This cohort is designed in order to contextualize the safety, tolerability and efficacy of the long-term ADV7103 treatment.

The B14CS Study will be composed of 3 periods (more detailed hereafter):

Lead-in Period: Period of 3 weeks, during which the treatment (ADV7103 or SoC) will be taken at the optimal dose as previously defined (Maintenance Phase). The Lead-in Period can include a Titration Phase, for ADV7103 B12CS Cohort only, in order to define the individual optimal dose of ADV7103 for the patients of this cohort;
Assessment Period: Period of 7 days to evaluate the effect of study products (ADV7103 and SoC) at the optimal dose;
Follow-up Period: Period of 23 months to evaluate the effects of the study products (ADV7103 and SoC) at long-term.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
ADV7103	Active comparator	Drug: ADV7103Patients receive ADV7103 twice a day at optimal dose. Each dose of ADV7103 contains a fixed ratio of 1/3 of ADV7103-CK (potassium citrate) and 2/3 of ADV7103-BK (potassium bicarbonate) based on the mass of active substances. Other Names: • Potassium Citrate and Potassium Bicarbonate
Standard of care comparator	Active comparator	Drug: Standard of CareAlkalinising treatment (SoC) taken at the usual dose and frequency

Who may qualify

Ages: 6 months to 70 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (17 listed)

For ADV7103 cohort:
Patient who has a diagnosis of cystinuria based on medical diagnosis (at least one previous or current episode of calculus of cystine, and/or one previous or current episode of cystine crystalluria) or on genetic diagnosis (only for patients of Subset 4).
Patient treated with an alkalising treatment at a well-adapted dose (defined as a daily dose deemed by the investigator aiming to maintain overtime urinary pH value ≥ 7.0 and/or compatible with an acceptable safety profile and/or patient's constraints or compliance).
Patient male or female, including child aged between 6 months and 17 years old and adult aged ≥ 18 years old up to 70 years old.
Female patient of childbearing potential (defined by the Clinical Trial Facilitation Group (CTFG) as woman fertile, following menarche until becoming post-menopausal unless permanently sterile*) using an acceptable effective birth control method and having a negative pregnancy test at the inclusion, or a woman postmenopausal* or a woman surgically sterilized*.

Key exclusion criteria (18 listed)

For ADV7103 cohort:
Patient that is receiving the second line therapy -- cystine chelating agents (sulfhydryl compounds).
Patient who presents kalaemia > 5.0 mmol/L.
Patient who presents a moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 according to Schwartz formula for the children and both MDRDs and CKD-EPI for adults).
Patient who presents - barring the study disease - any previous or concurrent medical condition or any laboratory or clinical findings or any other condition that in the opinion of the investigator would be negatively affected by the study product or that would affect the study product or that precludes his participation, e.g. uncontrolled diabetes mellitus, adrenal insufficiency, cardiac impairment, repeated infections, metabolic alkalosis, chronic diarrhoea.

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
ADV7103Also: Potassium Citrate and Potassium BicarbonatePatients receive ADV7103 twice a day at optimal dose.	Drug	ADV7103
Standard of CareCohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period.	Drug	Standard of care comparator

Primary outcome

Percentage of urinary pH values ≥ 7.0 during 24h on Day 7 (after ADV7103 treatment period)
Time frame: 7 Days
To evaluate the safety and the tolerability of ADV7103 and standard of care (SoC) after a long-term treatment.

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
Company websiteLink provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Cystinuria

RecruitingObservational study
Prospective Research Rare Kidney Stones (ProRKS)
Hyperoxaluria, Cystinuria
RecruitingObservational study
Rare Kidney Stone Consortium Biobank
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Rare Kidney Stone Consortium Patient Registry
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Monogenic Kidney Stone - Genetic Testing
Rare Kidney Stone Diseases
Active, not recruiting
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Nephrocalcinosis, Renal Calculi

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.

Luc-André Granier, M.D. study director

Advicenne Pharma

13 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing ADV7103 in people with Cystinuria, run by Advicenne Pharma at 13 sites in 2 countries.

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 17 inclusion and 18 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: withdrawn, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in France, Belgium. 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 combined 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.

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 was withdrawn before enrolling participants, so there are no results.

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?

Percentage of urinary pH values ≥ 7.0 during 24h on Day 7 (after ADV7103 treatment period) (time frame: 7 Days). 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 NCT04137978.

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 12 Mar 2024.