Alport SyndromePhase 1NCT03373786
A Study of RG-012 in Subjects With Alport Syndrome
A Phase 1 study testing RG012 in people with Alport Syndrome, run by Genzyme, a Sanofi Company at 7 sites in United States.
Trial summary
This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Alport Syndrome
- What is being tested?
- RG012
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 7 sitesUnited States
- Study dates
- 22 Dec 2017to 20 May 2019
- Phase
- Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females, ages 18 to 65 years
- Confirmed diagnosis of Alport syndrome
- eGFR between 40 and 90 mL/min/1.73m2
- Proteinuria of at least 300 mg protein/g creatinine
- For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
- Willing to comply with contraception requirements
Who cannot join
- Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
- End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
- Any other condition that may pose a risk to the subject's safety and well-being
- Female subjects who are pregnant or lactating
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Houston Nephrology Research
Cypress, Texas, 77429
eStudySite
La Mesa, California, 91942
Academic Medical Research Institute
Los Angeles, California, 90022
Apex Research of Riverside
Riverside, California, 92505
Utah Kidney Research Institute
Salt Lake City, Utah, 84107
Eminence Medical & Clinical Research
Tampa, Florida, 33604
Allegiance Research Specialists, LLC
Wauwatosa, Wisconsin, 53226
About this study
Official title: A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome
This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.
Read the detailed description
This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.
- Conditions
- Alport Syndrome
- Being tested
- RG012
- Keywords
- Kidney disease, Nephritis, Hereditary kidney disease, Hereditary nephritis
- Medical subject headings
- Nephritis, Hereditary, Kidney Diseases, Nephritis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RG012Drug
RG012 in 0.3% sodium chloride
| Study group | Role | What they receive |
|---|---|---|
| RG-012 Single Dose | Experimental | 1.5 mg/kg RG012 subcutaneous injection Receives: Drug: RG012 |
| RG012 Every Other Week | Experimental | 1.5 mg/kg RG012 subcutaneous injections every other week Receives: Drug: RG012 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety - Adverse Events
Incidence and severity of adverse events
Measured: 8 weeks
Effect of RG-012 on renal microRNA-21 (miR-21)
Change in miR-21 expression in renal tissue
Measured: 8 weeks
Secondary outcomes (3)
Pharmacokinetic (PK) parameter - Cmax
Maximum observed plasma concentration
Measured: 8 weeks
Pharmacokinetic (PK) parameter - Tmax
Time to maximum observed plasma concentration
Measured: 8 weeks
Pharmacokinetic (PK) parameter - AUC
Area under the plasma concentration vs. time curve
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2017
- Study start22 Dec 2017
- Primary completion20 May 2019
- Study completion20 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alport SyndromeStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, M.D., Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03373786
- Sponsor's protocol ID
- PDY16327
- Other IDs
- RG012-06
- Status last verified
- Apr 2022
- First submitted
- 22 Oct 2017
- Last updated
- 25 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome
- ClinicalTrials.gov ID
- NCT03373786
- Acronym / sponsor ID
- PDY16327
- Conditions
- Alport Syndrome
- Keywords
- Kidney disease, Nephritis, Hereditary kidney disease, Hereditary nephritis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 22 Dec 2017 (actual)
- Primary completion
- 20 May 2019 (actual)
- Study completion
- 20 May 2019 (actual)
- First posted
- 14 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.
This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.
Detailed description
This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RG-012 Single Dose | Experimental | Drug: RG0121.5 mg/kg RG012 subcutaneous injection |
| RG012 Every Other Week | Experimental | Drug: RG0121.5 mg/kg RG012 subcutaneous injections every other week |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males or females, ages 18 to 65 years
- Confirmed diagnosis of Alport syndrome
- eGFR between 40 and 90 mL/min/1.73m2
- Proteinuria of at least 300 mg protein/g creatinine
- For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
Key exclusion criteria (4 listed)
- Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
- End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
- Any other condition that may pose a risk to the subject's safety and well-being
- Female subjects who are pregnant or lactating
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 |
|---|---|---|
| RG012RG012 in 0.3% sodium chloride | Drug | RG-012 Single Dose, RG012 Every Other Week |
Primary outcome
Safety - Adverse Events
Time frame: 8 weeks
Incidence and severity of adverse events
Effect of RG-012 on renal microRNA-21 (miR-21)
Time frame: 8 weeks
Change in miR-21 expression in renal tissue
3 secondary outcomes
Pharmacokinetic (PK) parameter - Cmax
Time frame: 8 weeks
Pharmacokinetic (PK) parameter - Tmax
Time frame: 8 weeks
Pharmacokinetic (PK) parameter - AUC
Time frame: 8 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 25 Apr 2022.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences & Operations, M.D. study director
Sanofi
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RG012 in people with Alport Syndrome, run by Genzyme, a Sanofi Company at 7 sites in United States.
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 4 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 25 Apr 2022. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety - Adverse Events (time frame: 8 weeks). The study lists 2 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 25 Apr 2022.