Familial Mediterranean FeverPhase 2NCT05448391
A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
A Phase 2 study testing RIST4721 in people with Familial Mediterranean Fever, run by Aristea Therapeutics, Inc..
Trial summary
A 12-week Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects with Familial Mediterranean Fever followed by an additional Open-label Extension Phase. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Familial Mediterranean Fever
- What is being tested?
- RIST4721
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 13 Sep 2022to 30 Dec 2024
- 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 18 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of type 1 FMF disease but no active flare at the time of screening according to Tel Hashomer criteria or EUROFEVER/Paediatric Rheumatology International Trials Organization (PRINTO) criteria for FMF
- Documented to be heterozygous or homozygous for at least one of the known clearly pathogenic MEFV gene exon 10 mutations
- Males and females must be willing to use birth control as indicated
Who cannot join
- Breastfeeding or pregnant
- Known immunodeficiency or subject is immunocompromised
- Active/latent infection with HBV, HCV, HIV, SARS-CoV-2 or TB
About this study
Official title: An Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects With Familial Mediterranean Fever
A 12-week Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects with Familial Mediterranean Fever followed by an additional Open-label Extension Phase.
- Conditions
- Familial Mediterranean Fever
- Being tested
- RIST4721
- Medical subject headings
- Familial Mediterranean Fever
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RIST4721Drug
RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets
| Study group | Role | What they receive |
|---|---|---|
| RIST4721 400 mg | Experimental | RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks Receives: Drug: RIST4721 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
Measured: Baseline to Week 12
Secondary outcomes (1)
Proportion of responders who achieve resolution of their index flare after initiating study treatment and do not experience a new flare from the time of resolution of the index flare until Week 12
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2022
- Study start13 Sep 2022
- Primary completion30 Nov 2023 (estimated)
- Study completion30 Dec 2024 (estimated)
Why it stopped: Due to safety findings in other ongoing Phase 2 trials.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Mediterranean FeverStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aristea Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05448391
- Sponsor's protocol ID
- RIST4721-212
- Status last verified
- Feb 2023
- First submitted
- 1 Jul 2022
- Last updated
- 13 Feb 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects With Familial Mediterranean Fever
- ClinicalTrials.gov ID
- NCT05448391
- Acronym / sponsor ID
- RIST4721-212
- Conditions
- Familial Mediterranean Fever
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Due to safety findings in other ongoing Phase 2 trials.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Aristea Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Sep 2022 (actual)
- Primary completion
- 30 Nov 2023 (estimated)
- Study completion
- 30 Dec 2024 (estimated)
- First posted
- 7 Jul 2022 (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.
A 12-week Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects with Familial Mediterranean Fever followed by an additional Open-label Extension Phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RIST4721 400 mg | Experimental | Drug: RIST4721RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of type 1 FMF disease but no active flare at the time of screening according to Tel Hashomer criteria or EUROFEVER/Paediatric Rheumatology International Trials Organization (PRINTO) criteria for FMF
- Documented to be heterozygous or homozygous for at least one of the known clearly pathogenic MEFV gene exon 10 mutations
- Males and females must be willing to use birth control as indicated
Key exclusion criteria (3 listed)
- Breastfeeding or pregnant
- Known immunodeficiency or subject is immunocompromised
- Active/latent infection with HBV, HCV, HIV, SARS-CoV-2 or TB
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 |
|---|---|---|
| RIST4721RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets | Drug | RIST4721 400 mg |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
Time frame: Baseline to Week 12
1 secondary outcome
Proportion of responders who achieve resolution of their index flare after initiating study treatment and do not experience a new flare from the time of resolution of the index flare until Week 12
Time frame: Baseline to Week 12
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 13 Feb 2023.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RIST4721 in people with Familial Mediterranean Fever, run by Aristea Therapeutics, Inc..
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 3 inclusion and 3 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 13 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
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 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 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?
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs (time frame: Baseline to Week 12). 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 13 Feb 2023.