AlkaptonuriaPhase 3SONIA 2NCT01916382
Suitability of Nitisinone in Alkaptonuria 2
A Phase 3 study testing Nitisinone in people with Alkaptonuria, run by University of Liverpool at 1 site in United Kingdom.
Trial summary
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical… Read more about this study
- Who is it for?
- Ages 25 years and older
- Conditions the study is for
- Alkaptonuria
- What is being tested?
- Nitisinone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 140 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Apr 2014to 2 Feb 2020
- 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 25 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A patient must fulfil the following criteria in order to be included in the study:
- 1. Diagnosis of AKU Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain.
- 3. Age ≥25 years. 4. Willing and able to visit the investigational site for study visits. 5. Signed written informed consent given.
- -
Who cannot join
- The presence of any of the following will exclude a patient from inclusion in the study:
- Currently pregnant or lactating.
- Female patient of child-bearing potential not using a reliable method of contraception.
- Known allergy to nitisinone or any of the constituents of the investigational product.
- Current malignancy.
- Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
Show all 20Show fewer
- Unstable cardiovascular disease.
- Serum potassium < 3.0 mmol/L.
- eGFR < 60 mL/min .
- ALT > 1.5 x upper limit of normal.
- Haemoglobin < 10.0 g/dL.
- Platelets < 100 x 109/L.
- Total white blood count < 3.0 x 109/L or neutrophil count < 1.5 x 109/L.
- History of alcohol or drug abuse.
- Participation in another clinical study within 3 months of randomization.
- Treatment with nitisinone within 60 days of randomization.
- Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
- Foreseeable inability to cooperate with given instructions or study procedures.
- Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
- -
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Royal Liverpool Hospital
Liverpool, Merseyside, L7 8XP
About this study
Official title: An International, Multicentre, Randomised, Evaluator-blind, No-treatment Controlled, Parallel-group Study to Assess the Efficacy and Safety of Once Daily Nitisinone in Patients With Alkaptonuria After 12 Months of Treatment, Followed by an Additional 36 Month Treatment Period.
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical development of nitisinone for AKU: a phase 3 clinical trial to prove efficacy. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of developing 200 new therapies by 2020.
- Conditions
- Alkaptonuria
- Being tested
- Nitisinone
- Keywords
- Alkaptonuria, nitisinone,Ochronosis, Homogentisic acid
- Medical subject headings
- Alkaptonuria, Ochronosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NitisinoneDrug
Also called Orfadin
drug
| Study group | Role | What they receive |
|---|---|---|
| Nitisinone | Experimental | Homogentisic acid lowering drug intervention Receives: Drug: Nitisinone |
| No treatment | No intervention | comparrator |
What is being measured
The primary outcome is the main question the study is designed to answer.
24 houre Urine Homogentisic acid
Measured: year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2013 (estimated)
- Study startApr 2014
- Primary completionFeb 2016
- Study completion2 Feb 2020 (estimated)
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 AlkaptonuriaStudies 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
- None (open label)
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Liverpool
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Professor Lakshminarayan Ranganath, University of Liverpool
- Investigators
- L Ranganath, Principal investigator, Liverpool University Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01916382
- Sponsor's protocol ID
- SONIA 2
- Acronym
- SONIA 2
- Status last verified
- May 2018
- First submitted
- 2 Aug 2013
- Last updated
- 31 May 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Imrich R, Sedlakova J, Ulehlova M, Gornall M, Jackson R, Olsson B, Rudebeck M, Gallagher J, Lukacova O, Mlynarikova V, Stancik R, Vrtikova E, Zanova E, Zatkova A, Arnoux JB, Rovensky J, Luangrath E, Bygott H, Khedr M, Ranganath LR. Radiological evolution of spinal disease in alkaptonuria and the effect of nitisinone. RMD Open. 2022 Oct;8(2):e002422. doi: 10.1136/rmdopen-2022-002422.
PubMed 36270742Ranganath LR, Psarelli EE, Arnoux JB, Braconi D, Briggs M, Broijersen A, Loftus N, Bygott H, Cox TF, Davison AS, Dillon JP, Fisher M, FitzGerald R, Genovese F, Glasova H, Hall AK, Hughes AT, Hughes JH, Imrich R, Jarvis JC, Khedr M, Laan D, Le Quan Sang KH, Luangrath E, Lukacova O, Milan AM, Mistry A, Mlynarikova V, Norman BP, Olsson B, Rhodes NP, Rovensky J, Rudebeck M, Santucci A, Shweihdi E, Scott C, Sedlakova J, Sireau N, Stancik R, Szamosi J, Taylor S, van Kan C, Vinjamuri S, Vrtikova E, Webb C, West E, Zanova E, Zatkova A, Gallagher JA. Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial. Lancet Diabetes Endocrinol. 2020 Sep;8(9):762-772. doi: 10.1016/S2213-8587(20)30228-X. Epub 2020 Aug 18.
PubMed 32822600
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International, Multicentre, Randomised, Evaluator-blind, No-treatment Controlled, Parallel-group Study to Assess the Efficacy and Safety of Once Daily Nitisinone in Patients With Alkaptonuria After 12 Months of Treatment, Followed by an Additional 36 Month Treatment Period.
- ClinicalTrials.gov ID
- NCT01916382
- Acronym / sponsor ID
- SONIA 2 / SONIA 2
- Conditions
- Alkaptonuria
- Keywords
- Alkaptonuria, nitisinone,Ochronosis, Homogentisic acid
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- University of LiverpoolUniversity, hospital or other
- Study start
- Apr 2014 (actual)
- Primary completion
- Feb 2016 (actual)
- Study completion
- 2 Feb 2020 (estimated)
- First posted
- 5 Aug 2013 (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.
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical development of nitisinone for AKU: a phase 3 clinical trial to prove efficacy. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of developing 200 new therapies by 2020.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nitisinone | Experimental | Drug: NitisinoneHomogentisic acid lowering drug intervention |
| No treatment | No intervention | –comparrator |
Who may qualify
- Ages
- 25 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 1. Diagnosis of AKU Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain.
- 3. Age ≥25 years. 4. Willing and able to visit the investigational site for study visits. 5. Signed written informed consent given.
- -
Key exclusion criteria (20 listed)
- Currently pregnant or lactating.
- Female patient of child-bearing potential not using a reliable method of contraception.
- Known allergy to nitisinone or any of the constituents of the investigational product.
- Current malignancy.
- Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
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 |
|---|---|---|
| NitisinoneAlso: Orfadindrug | Drug | Nitisinone |
Primary outcome
24 houre Urine Homogentisic acid
Time frame: year 1
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2018.
- Derived from this study: PubMed 36270742Imrich R, Sedlakova J, Ulehlova M, Gornall M, Jackson R, Olsson B, Rudebeck M, Gallagher J, Lukacova O, Mlynarikova V, Stancik R, Vrtikova E, Zanova E, Zatkova A, Arnoux JB, Rovensky J, Luangrath E, Bygott H, Khedr M, Ranganath LR. Radiological evolution of spinal disease in alkaptonuria and the effect of nitisinone. RMD Open. 2022 Oct;8(2):e002422. doi: 10.1136/rmdopen-2022-002422.
- Derived from this study: PubMed 32822600Ranganath LR, Psarelli EE, Arnoux JB, Braconi D, Briggs M, Broijersen A, Loftus N, Bygott H, Cox TF, Davison AS, Dillon JP, Fisher M, FitzGerald R, Genovese F, Glasova H, Hall AK, Hughes AT, Hughes JH, Imrich R, Jarvis JC, Khedr M, Laan D, Le Quan Sang KH, Luangrath E, Lukacova O, Milan AM, Mistry A, Mlynarikova V, Norman BP, Olsson B, Rhodes NP, Rovensky J, Rudebeck M, Santucci A, Shweihdi E, Scott C, Sedlakova J, Sireau N, Stancik R, Szamosi J, Taylor S, van Kan C, Vinjamuri S, Vrtikova E, Webb C, West E, Zanova E, Zatkova A, Gallagher JA. Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial. Lancet Diabetes Endocrinol. 2020 Sep;8(9):762-772. doi: 10.1016/S2213-8587(20)30228-X. Epub 2020 Aug 18.
Related trials for Alkaptonuria
RecruitingObservational study
Study of Alkaptonuria
Alkaptonuria
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.
L Ranganath principal investigator
Liverpool University Hospitals NHS Foundation Trust
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 Nitisinone in people with Alkaptonuria, run by University of Liverpool at 1 site in United Kingdom.
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 4 inclusion and 20 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 31 May 2018. 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 United Kingdom. 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 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?
Results are not available in the source record.
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?
24 houre Urine Homogentisic acid (time frame: year 1). 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 NCT01916382, 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 31 May 2018.