Mucopolysaccharidosis VIPhase 2iMProveSNCT03370653
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
A Phase 2 study testing Odiparcil in people with Mucopolysaccharidosis VI, run by Inventiva Pharma at 4 sites in 4 countries.
Trial summary
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Mucopolysaccharidosis VI
- What is being tested?
- Odiparcil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 30 Dec 2017to 22 Oct 2019
- Phase
- Phase 2
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Patients with MPS Type VI receiving enzyme replacement therapy (Naglazyme) for at least 6 months on the licensed dosage or as per local guidelines.
- Patients with MPS Type VI not receiving enzyme replacement therapy for the following reasons:
- Patients previously treated with ERT but have discontinued for more than 3 months either due to medical decision or personal choice
- Patients allergic to ERT therapy
- Patients that have had a previous hematopoietic stem cell transplant (HSCT)
- Patients not treated with ERT i.e. treatment naïve
Who cannot join
- Use of any investigational product or investigational medical device within 30 days prior to screening. This will include product bought over the counter specifically compounds like genistein and pentosane polysulphate which may not be considered as investigational products by patients and some health care professionals.
- Concurrent disease or condition that would interfere with study participation or pose a safety concern for example patient with: severe cardiac insufficiency as define NYHA class > II, and severe restrictive chronic respiratory insufficiency as reflected by serum [HCO3-] ≥28 mEq/L.
- Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
- Patient with spinal cord compression requiring surgical intervention.
- Subjects with the following liver test anomalies: any ALT, AST > 3xULN or bilirubin >1.5xULN (except if Gilbert syndrome) at screening visit.
- Evidence of an immunosuppressive state, including known HIV infection, agammaglubilinemias, T-Cell deficiencies.
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- Subjects with history of chronic infections, including but not limited to subjects with past history of viral hepatitis C, or B, with recent history of serious or life-threatening infection or any current signs or symptoms that may indicate infection at visit V-1 of study as per investigators clinical judgement.
- History of malignant cancer except of cervical carcinoma in situ, basal cell carcinoma, dermatological squamous cell carcinoma.
- Subjects with significant haematologic abnormalities, such as haemoglobin <8 g/dL, or WBC<2000 /mm3 or absolute neutrophil count <1300 /mm3, or platelet <30.000 /mm3.
- International Normalized Ratio (INR), activated partial thromboplastin time (aPTT) or thrombin time (TT) values above the laboratory reference range at screening. For patients on anti-coagulants, they should be within their target effect on INR and be stable.
- Any history of bleeding diathesis
- Patient with coexistence of corneal pathologies other than corneal clouding (e.g. exposure keratopathy)
- An unwillingness on the part of male patients to abstain from sexual intercourse with pregnant or lactating women; or an unwillingness to use highly effective form of birth control if engaging in sexual intercourse with a woman who could become pregnant from the time of the first dose of study medication until completion of follow-up procedures.
- An unwillingness on the part of female patients to use highly effective form of birth control2 if engaging in sexual intercourse and to have a monthly pregnancy test during treatment and until completion of follow-up procedures.
- Pregnant or lactating women.
- Have a known hypersensitivity to any of the ingredients or excipients of the IMP including: Microcrystalline Cellulose, Povidone, Sodium starch glycolate (type A), Magnesium stearate, Opadry™ II 85F18422
- 1. Previous hematopoietic stem cell transplant (HSCT)
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). Medical need for treatment of MPS is still very high due to the poor penetration of the recombinant enzymes into the blood brain barrier as well as the ocular barriers and into tissues that are poorly vascularized, such as cartilages and bones. Odiparcil is an orally active compound that allows the synthesis of soluble glycosaminoglycans (GAGs), mainly chondroitin sulfate (CS) and dermatane sulfate (DS). The neosynthesized solubles GAGs are then excreted in urine. By diverting endogenous GAG synthesis to the synthesis of soluble odiparcil linked GAGs, odiparcil should decrease the intracellular pool of GAGs and consequently decrease the lysosomal GAG accumulation.
The primary objective of the study is to assess the safety and efficacy of two doses of odiparcil in MPS VI patients and to provide evidence to enable the selection of the relevant dose of odiparcil for phase III study. The secondary objective of this study is to characterize the dose response, PK and PD of odiparcil.
Read the detailed description
Study design: This phase IIa study consists of 2 parts performed sequentially: a preliminary safety assessment followed by the core study with a double-blind, randomized, dose-ranged cohort of patients receiving Enzyme Replacement Therapy (ERT) and an open-label cohort of patients not receiving ERT.
Preliminary safety assessment (N=2): open-label, escalating dose (2 doses) study. If acceptable safety profile is achieved, patients will be then included in the open-label arm of the core study.
Core study
Core study will be conducted on 2 populations in parallel:
- A first cohort (N=18): MPS VI patients receiving ERT assigned in 3 arms:
- Placebo (N=6)
- Odiparcil 500 mg per day (250 mg BID) (N=6)
- Odiparcil 1000 mg per day (500 mg BID) (N=6).
- A second cohort (N=6): MPS VI patient not receiving ERT (odiparcil 1000 mg per day (500 mg BID)).
Study duration: The overall study duration will be 20 months, including the 10-month enrolment period.
For each patient, the study duration will be:
- Preliminary safety assessment: 6 weeks including a 4-week run-in period followed by 2-week treatment period. Then, patients will go on treatment period in core study.
- Core study: 34 weeks including a 4-week run-in period followed by 26-week treatment period and 4-week of follow-up.
- Conditions
- Mucopolysaccharidosis VI
- Being tested
- Odiparcil, Placebo
- Keywords
- MPS VI, Lysosomal disease, Glycoaminoglycans (GAG), Odiparcil
- Medical subject headings
- Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OdiparcilDrug
Investigational product: odiparcil 250 mg tablets
PlaceboOther
Comparator: placebo tablets similar to odiparcil 250 mg tablets
| Study group | Role | What they receive |
|---|---|---|
| Double-blind - odiparcil 1000 mg per day | Experimental | 2 tablets of odiparcil 250 mg per os, twice daily (BID) Receives: Drug: Odiparcil |
| Double-blind - odiparcil 500 mg per day | Experimental | 1 tablet of placebo and 1 tablet of odiparcil 250 mg per os, twice daily (BID) Receives: Drug: Odiparcil; Other: Placebo |
| Double-blind - placebo | Placebo comparator | 2 tablets of placebo per os, twice daily (BID) Receives: Other: Placebo |
| Open Label - odiparcil 1000 mg per day | Experimental | 2 tablets of odiparcil 250 mg per os, twice daily (BID) Receives: Drug: Odiparcil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with modified clinical signs
Changes in physical examination and vital signs
Measured: 26 weeks
Number of patients with modified biological values
Change from baseline in laboratory safety tests (coagulation, liver enzymes and crystalluria) 12-lead-ECG and bone biomarkers.
Measured: 26 weeks
Incidence of AEs/SAEs
Incidence of AEs/SAEs, patient withdrawals from study due to AEs/SAEs,
Measured: 26 weeks
12-lead ECG
Change from Baseline in ECG
Measured: 26 weeks
Secondary outcomes (25)
Mobility: 6-minute walk test
Change from baseline in 6-minute walk test
Measured: 26 weeks
Mobility: 9-hole PEG test
Change from baseline in 9-hole PEG test
Measured: 26 weeks
Mobility: range of motion of the shoulder
Change from baseline in range of motion of the shoulder
Measured: 26 weeks
Pain assessment
Change from Baseline in Brief Pain Inventory (BPI)
Measured: 26 weeks
Respiratory function
Change from Baseline in FEV1
Measured: 26 weeks
Respiratory function
Change from Baseline in FVC
Measured: 26 weeks
Respiratory function
Change from Baseline in MVV
Measured: 26 weeks
Cardiac and vascular function
Change from Baseline in echocardiogram
Measured: 26 weeks
Cardiac and vascular function
Change from Baseline in carotid intima media thickness Odiparcil concentration remaining in patient plasma 12 hours following the last intake of investigational product at visits V4 and V7. An identification of odiparcil metabolites in plasma at visit V2.
Measured: 26 weeks
Audiology assessments
Change from Baseline in pure tone audiometry
Measured: 26 weeks
Audiology assessments
Change from Baseline in whisper voice test
Measured: 26 weeks
Ophthalmology assessments
Change from Baseline in corneal opacification
Measured: 26 weeks
Ophthalmology assessments
Change from Baseline in level of retinopathy
Measured: 26 weeks
Ophthalmology assessments
Change from Baseline in optic nerve involvement,
Measured: 26 weeks
Ophthalmology assessments
Change from Baseline in intra-ocular pressure
Measured: 26 weeks
Ophthalmology assessments
Change from Baseline in visual acuity
Measured: 26 weeks
Quality of life questionnaires
Change from Baseline in EQ-5D-5L questionnaires. 5 dimensions scored on a 5-point scale will be assessed: mobility, self-care, usual activities, pain/discomfort, anxiety/depression
Measured: 26 weeks
Quality of life questionnaires
Change from Baseline in Zarit caregiver burden questionnaires. Scale in 22 items scored on a 5-point scale with 0 = never and 5 = nearly always
Measured: 26 weeks
Quality of life questionnaires
Change from Baseline in Fatigue Severity Scale questionnaires. 9 questions scored on a 7-point scale with 1 = strongly disagree and 7= strongly agree
Measured: 26 weeks
Pharmacokinetics: odiparcil concentration in plasma
Odiparcil concentration in plasma at visit V2 (up to 12 hours post dose).
Measured: 12 hours
¨Pharmacodynamics: GAG concentrations
GAG concentration in leukocytes isolated from peripheral
Measured: 26 weeks
¨Pharmacodynamics: GAG concentrations
GAG concentrations in urine
Measured: 26 weeks
Pharmacodynamics: GAG concentrations
GAG concentrations in skin
Measured: 26 weeks
¨Pharmacodynamics: anti-thrombin activity IIa
Change from Baseline in anti-thrombin activity IIa in plasma
Measured: 26 weeks
¨Pharmacodynamics: Thrombin Generation Assay (TGA)
Change from Baseline in TGA in plasma
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2017
- Study start30 Dec 2017
- Primary completion24 Sep 2019
- Study completion22 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis VIStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Preliminary safety assessment (N=2): an open-label, escalating dose (2 doses) study. Core study: conducted on 2 populations in parallel: * A first cohort (N=18): MPS VI patients receiving ERT assigned in 3 arms: * Placebo (N=6) * Odiparcil 500 mg per day (250 mg BID) (N=6) * Odiparcil 1000 mg per day (500 mg BID) (N=6). * A second cohort (N=6): MPS VI patients not receiving ERT (odiparcil 1000 mg per day (500 mg BID)).
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Inventiva Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Derralynn HUGHES, MD, Principal investigator, Royal Free Hospital, London UKJulia HENNERMANN, MD, Principal investigator, Villa Metabolica, Mainz GERMANYNathalie GUFFON-FOUILHOUX, MD, Principal investigator, Hôpital Femme-Mère-EnfantElisa LEAO-TELES, MD, Principal investigator, Centro Hospitalar S. João, Porto, Portugal
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03370653
- Sponsor's protocol ID
- IVA_01_ODI_HMPS_17_002
- Acronym
- iMProveS
- Status last verified
- Oct 2019
- First submitted
- 23 Nov 2017
- Last updated
- 1 Nov 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI
- ClinicalTrials.gov ID
- NCT03370653
- Acronym / sponsor ID
- iMProveS / IVA_01_ODI_HMPS_17_002
- Conditions
- Mucopolysaccharidosis VI
- Keywords
- MPS VI, Lysosomal disease, Glycoaminoglycans (GAG), Odiparcil
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Inventiva PharmaIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2017 (actual)
- Primary completion
- 24 Sep 2019 (actual)
- Study completion
- 22 Oct 2019 (actual)
- First posted
- 12 Dec 2017 (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.
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). Medical need for treatment of MPS is still very high due to the poor penetration of the recombinant enzymes into the blood brain barrier as well as the ocular barriers and into tissues that are poorly vascularized, such as cartilages and bones. Odiparcil is an orally active compound that allows the synthesis of soluble glycosaminoglycans (GAGs), mainly chondroitin sulfate (CS) and dermatane sulfate (DS). The neosynthesized solubles GAGs are then excreted in urine. By diverting endogenous GAG synthesis to the synthesis of soluble odiparcil linked GAGs, odiparcil should decrease the intracellular pool of GAGs and consequently decrease the lysosomal GAG accumulation.
The primary objective of the study is to assess the safety and efficacy of two doses of odiparcil in MPS VI patients and to provide evidence to enable the selection of the relevant dose of odiparcil for phase III study. The secondary objective of this study is to characterize the dose response, PK and PD of odiparcil.
Detailed description
Study design: This phase IIa study consists of 2 parts performed sequentially: a preliminary safety assessment followed by the core study with a double-blind, randomized, dose-ranged cohort of patients receiving Enzyme Replacement Therapy (ERT) and an open-label cohort of patients not receiving ERT.
Preliminary safety assessment (N=2): open-label, escalating dose (2 doses) study. If acceptable safety profile is achieved, patients will be then included in the open-label arm of the core study.
Core study
Core study will be conducted on 2 populations in parallel:
- A first cohort (N=18): MPS VI patients receiving ERT assigned in 3 arms:
- Placebo (N=6)
- Odiparcil 500 mg per day (250 mg BID) (N=6)
- Odiparcil 1000 mg per day (500 mg BID) (N=6).
- A second cohort (N=6): MPS VI patient not receiving ERT (odiparcil 1000 mg per day (500 mg BID)).
Study duration: The overall study duration will be 20 months, including the 10-month enrolment period.
For each patient, the study duration will be:
- Preliminary safety assessment: 6 weeks including a 4-week run-in period followed by 2-week treatment period. Then, patients will go on treatment period in core study.
- Core study: 34 weeks including a 4-week run-in period followed by 26-week treatment period and 4-week of follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- Preliminary safety assessment (N=2): an open-label, escalating dose (2 doses) study. Core study: conducted on 2 populations in parallel: * A first cohort (N=18): MPS VI patients receiving ERT assigned in 3 arms: * Placebo (N=6) * Odiparcil 500 mg per day (250 mg BID) (N=6) * Odiparcil 1000 mg per day (500 mg BID) (N=6). * A second cohort (N=6): MPS VI patients not receiving ERT (odiparcil 1000 mg per day (500 mg BID)).
| Arm | Type | Receives |
|---|---|---|
| Double-blind - odiparcil 1000 mg per day | Experimental | Drug: Odiparcil2 tablets of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - odiparcil 500 mg per day | Experimental | Drug: Odiparcil, Other: Placebo1 tablet of placebo and 1 tablet of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - placebo | Placebo comparator | Other: Placebo2 tablets of placebo per os, twice daily (BID) |
| Open Label - odiparcil 1000 mg per day | Experimental | Drug: Odiparcil2 tablets of odiparcil 250 mg per os, twice daily (BID) |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 1. Patients with MPS Type VI receiving enzyme replacement therapy (Naglazyme) for at least 6 months on the licensed dosage or as per local guidelines.
- Patients with MPS Type VI not receiving enzyme replacement therapy for the following reasons:
- Patients previously treated with ERT but have discontinued for more than 3 months either due to medical decision or personal choice
- Patients allergic to ERT therapy
- Patients that have had a previous hematopoietic stem cell transplant (HSCT)
Key exclusion criteria (17 listed)
- Use of any investigational product or investigational medical device within 30 days prior to screening. This will include product bought over the counter specifically compounds like genistein and pentosane polysulphate which may not be considered as investigational products by patients and some health care professionals.
- Concurrent disease or condition that would interfere with study participation or pose a safety concern for example patient with: severe cardiac insufficiency as define NYHA class > II, and severe restrictive chronic respiratory insufficiency as reflected by serum [HCO3-] ≥28 mEq/L.
- Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
- Patient with spinal cord compression requiring surgical intervention.
- Subjects with the following liver test anomalies: any ALT, AST > 3xULN or bilirubin >1.5xULN (except if Gilbert syndrome) at screening visit.
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 |
|---|---|---|
| OdiparcilInvestigational product: odiparcil 250 mg tablets | Drug | Double-blind - odiparcil 1000 mg per day, Double-blind - odiparcil 500 mg per day, Open Label - odiparcil 1000 mg per day |
| PlaceboComparator: placebo tablets similar to odiparcil 250 mg tablets | Other | Double-blind - odiparcil 500 mg per day, Double-blind - placebo |
Primary outcome
Number of patients with modified clinical signs
Time frame: 26 weeks
Changes in physical examination and vital signs
Number of patients with modified biological values
Time frame: 26 weeks
Change from baseline in laboratory safety tests (coagulation, liver enzymes and crystalluria) 12-lead-ECG and bone biomarkers.
Incidence of AEs/SAEs
Time frame: 26 weeks
Incidence of AEs/SAEs, patient withdrawals from study due to AEs/SAEs,
12-lead ECG
Time frame: 26 weeks
Change from Baseline in ECG
25 secondary outcomes
Mobility: 6-minute walk test
Time frame: 26 weeks
Mobility: 9-hole PEG test
Time frame: 26 weeks
Mobility: range of motion of the shoulder
Time frame: 26 weeks
Pain assessment
Time frame: 26 weeks
Respiratory function
Time frame: 26 weeks
Respiratory function
Time frame: 26 weeks
Respiratory function
Time frame: 26 weeks
Cardiac and vascular function
Time frame: 26 weeks
Cardiac and vascular function
Time frame: 26 weeks
Audiology assessments
Time frame: 26 weeks
Audiology assessments
Time frame: 26 weeks
Ophthalmology assessments
Time frame: 26 weeks
Ophthalmology assessments
Time frame: 26 weeks
Ophthalmology assessments
Time frame: 26 weeks
Ophthalmology assessments
Time frame: 26 weeks
Ophthalmology assessments
Time frame: 26 weeks
Quality of life questionnaires
Time frame: 26 weeks
Quality of life questionnaires
Time frame: 26 weeks
Quality of life questionnaires
Time frame: 26 weeks
Pharmacokinetics: odiparcil concentration in plasma
Time frame: 12 hours
¨Pharmacodynamics: GAG concentrations
Time frame: 26 weeks
¨Pharmacodynamics: GAG concentrations
Time frame: 26 weeks
Pharmacodynamics: GAG concentrations
Time frame: 26 weeks
¨Pharmacodynamics: anti-thrombin activity IIa
Time frame: 26 weeks
¨Pharmacodynamics: Thrombin Generation Assay (TGA)
Time frame: 26 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2019.
- iMProveS Study web siteLink provided by the sponsor.
- 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. This study is not recruiting at the moment.
Derralynn HUGHES, MD principal investigator
Royal Free Hospital, London UK
Julia HENNERMANN, MD principal investigator
Villa Metabolica, Mainz GERMANY
Nathalie GUFFON-FOUILHOUX, MD principal investigator
Hôpital Femme-Mère-Enfant
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Odiparcil in people with Mucopolysaccharidosis VI, run by Inventiva Pharma at 4 sites in 4 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 6 inclusion and 17 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: finished, as of the sponsor's last update on 1 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France, Germany, Portugal, 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of patients with modified clinical signs (time frame: 26 weeks). The study lists 4 primary outcomes in all. 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 1 Nov 2019.