Cryopyrin-Associated Periodic SyndromesPhase 1NCT06974877
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
A Phase 1 study testing 124I AT-01 in people with Cryopyrin-Associated Periodic Syndromes and Amyloidosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Anakinra is a drug used to treat people with certain diseases that affect their immune systems. Sometimes anakinra can cause proteins under the skin to clump together. These clumps are called amyloidosis; they can spread to other organs. Read more about this study
- Who is it for?
- Ages 18 to 120 years
- Conditions the study is for
- Cryopyrin-Associated Periodic Syndromes, Amyloidosis
- What is being tested?
- 124I AT-01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Sep 2025to 31 Jan 2032
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 120 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- An individual must meet all the following criteria to be eligible for this study:
- Aged 18 years and older.
- Currently enrolled on NIH protocol 17-I-0016 with a documented diagnosis of MWS or NOMID.
- Agree to allow data collected in this study to be shared with and stored on NIH protocol 17-I-0016 for that study s research analyses.
- Developed skin thickening at the site of anakinra injection.
- Participants who can become pregnant or who can impregnate their partner must agree to use 2 highly effective methods of contraception, at least 1 of which must be a barrier method, when engaging in sexual activities that can result in pregnancy, beginning 28 days prior to baseline until 90 days after the last PET/CT scan. Acceptable methods of contraception include the following:
Show all 15Show fewer
- Barrier methods:
- External or internal condom with spermicide.
- Diaphragm or cervical cap with a spermicide.
- Non-barrier methods:
- Hormonal contraception.
- Intrauterine device.
- Hysterectomy, oophorectomy, or tubal ligation in women
- Vasectomy in men
- Other.
Who cannot join
- Known hypersensitivity to 124I-AT-01, AT-01, or any of their excipients.
- Known hypersensitivity to KI.
- Pregnant or breastfeeding.
- Currently receiving dialysis.
- Currently taking heparin or heparin derivatives (eg, low molecular weight heparins) or other blood thinners for anticoagulation.
- Any condition that, in the opinion of the study team, contraindicates participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
NIH Clinical Center Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
Sara Alehashemi, M.D., (301) 761-7570, sara.alehashemi@nih.gov
About this study
Official title: A Phase 1 Study of Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using Amyloid-Reactive Peptide 124I-AT-01 (124I-p5+14, Iodine-124I-Evuzamitide) to Measure Changes in Organ-Specific Amyloid Load
Background:
Anakinra is a drug used to treat people with certain diseases that affect their immune systems. Sometimes anakinra can cause proteins under the skin to clump together. These clumps are called amyloidosis; they can spread to other organs. The only way to diagnose amyloidosis is to remove a piece of tissue (biopsy). Researchers want to find a way to locate amyloidosis in internal organs using positron emission tomography (PET)/computed tomography (CT).
Read more
Objective:
To test a new tracer used during PET/CT scans in people with amyloidosis. A tracer is a radioactive dye injected into the body.
Eligibility:
Adults aged 18 years or older with amyloidosis from anakinra injections. They must be enrolled in NIH protocol 17-I-0016.
Design:
Participants will come to the clinic once every 6 months for 2 years. Each visit will be 1 day.
They will have a PET/CT scan with the new tracer at each visit: The tracer will be given through a tube attached to a needle inserted into a vein.
The PET/CT scanner is a machine shaped like a doughnut. Participants will lie still on a padded table. The table will move in and out of the machine. The scan takes about 1 hour.
Radiation from the tracer will remain in the body for 24 hours after each scan. Participants will need to follow rules to avoid exposing pets and other people.
Participants will collect a 24-hour urine sample before each visit. They will also have blood tests and a physical exam at each visit.
Participants will receive a follow-up phone call about 1 week after each visit.
Read the detailed description
Study Description:
This is a phase 1 study to evaluate the feasibility of an investigational positron emission tomography (PET)/computed tomography (CT) radiotracer, 124I-AT-01, to screen for anakinra-induced amyloidosis in people with the cryopyrin-associated autoinflammatory syndromes (CAPS) (Muckle-Wells syndrome [MWS] or neonatal onset multisystem inflammatory disease [NOMID]), and to follow resolution of amyloidosis. This radiotracer binds to amyloid, so a PET/CT scan can show the presence and relative magnitude of amyloid.
Participants aged 18 years and older who developed local skin or systemic amyloidosis as a result of anakinra treatment will be recruited. They will have a PET/CT scan with radiotracer 124I-AT-01 about once every 6 months for 2 years. Blood and urine will be collected for clinical safety analyses, but no specimen will be collected for research or storage purposes.
The 124I-AT-01 PET/CT imaging is expected to be able to detect anakinra-induced amyloid in the skin and other organs (such as kidney, liver, and spleen). The percent change in quantitative uptake of 124I-AT-01 from baseline to each subsequent scan is expected to correlate with changes in clinical features related to load of amyloid in the body.
Primary Objectives:
- To screen participants with fixed skin thickening at the site of anakinra injection and/or biopsy-proven anakinra-induced amyloidosis of the skin for the presence of systemic amyloidosis, using 124I-AT-01 PET/CT imaging.
- To measure changes in organ-specific uptake of 124I-AT-01 from baseline in participants with anakinra-induced amyloidosis (local or systemic) using PET/CT imaging.
- To assess the safety profile of 124I-AT-01 in participants with CAPS.
Primary Endpoints:
Change in organ-specific 124I-AT-01 uptake from baseline PET/CT imaging to the follow-up PET/CT imaging every 6 months for 2 years. Frequency of CAPS disease flares after exposure to 124I-AT-01.
- Being tested
- 124I AT-01
- Keywords
- Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease, Interleukin 1 Receptor Antagonist Protein, Anakinra, AMYLOIDOSIS, AIL1RAP
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
124I AT-01Drug
124I-AT-01 is an amyloid-reactive synthetic 45-L amino acid polypeptide radiolabeled with iodine-124, with a theoretical molecular weight of 4763.6 Da (based on amino acid sequence). The polypeptide, AT-01, is not pharmacologically active. 124I-AT-01 binds many forms of human and murine amyloid and is intended to be a PET imaging agent for the detection of amyloid deposits.
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Experimental | Participants aged 18 years and older with anakinra-type amyloidosis (n=10) will be recruited from NIH protocol 17-I-0016. Upon confirmation of eligibility, they will undergo a PET/CT scan with the investigational radiotracer 124I-AT-01, which selectively binds to amyloid fibers. Blood and urine will also be collected for clinical and research analyses, including measurement of IL-1RA and other biomarkers. These scans and sample collections will be repeated about once every 6 months for 2 years. Receives: Drug: 124I AT-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in organ-specific 124I AT-01 uptake from baseline PET/CT imaging to the follow up PET/CT imaging every 6 months for 2 years.
The gold standard for detecting amyloid deposits is tissue biopsy. With this compound, we expect to detect evidence of tissue deposits in the tissue at a much earlier stage and in a non invasive manner. By lowering the exposure to anakinra in participants with anakinra-associated systemic amyloidosis, the burden of amyloidosis is expected to reduce over time.
Measured: 2 years
Frequency of CAPS disease flares after exposure to 124I AT 01.
This compound has not been used in patients with CAPS. We will monitor the participants to detect possible inflammatory disease flare following the exposure.
Measured: Through end of study.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2025
- Study start10 Sep 2025
- Primary completion1 Jul 2031 (estimated)
- Study completion31 Jan 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cryopyrin-Associated Periodic SyndromesStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- 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
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Sara Alehashemi, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06974877
- Sponsor's protocol ID
- 10001867
- Other IDs
- 001867-I
- Status last verified
- 14 May 2026
- First submitted
- 15 May 2025
- Last updated
- 19 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared in accordance with the NIH Data Management and Sharing Policy.
Publications
Alehashemi S, Dasari S, Metpally A, Uss K, Castelo-Soccio LA, Heller T, Kellman P, Chen MY, Ahlman M, Kim J, Wargo S, Kuhns DB, Fink D, de Jesus A, Martin PS, Chang R, Bolanos J, Lee CR, Nasr SH, Goldbach-Mansky R, McPhail E. Anakinra-Associated Systemic Amyloidosis. Arthritis Rheumatol. 2024 Jan;76(1):100-106. doi: 10.1002/art.42664. Epub 2023 Nov 29.
PubMed 37488949Nasr SH, Alehashemi S, Dasari S, Waldman M, Afzali B, Chiu A, Bolanos J, Goldbach-Mansky R, McPhail ED. Anakinra-associated renal amyloidosis. Kidney Int. 2024 Feb;105(2):395-396. doi: 10.1016/j.kint.2023.08.020. No abstract available.
PubMed 38245224Alehashemi S, Dasari S, de Jesus AA, Cowen EW, Lee CR, Goldbach-Mansky R, McPhail ED. Anakinra-Associated Amyloidosis. JAMA Dermatol. 2022 Dec 1;158(12):1454-1457. doi: 10.1001/jamadermatol.2022.2124.
PubMed 36223107
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using Amyloid-Reactive Peptide 124I-AT-01 (124I-p5+14, Iodine-124I-Evuzamitide) to Measure Changes in Organ-Specific Amyloid Load
- ClinicalTrials.gov ID
- NCT06974877
- Acronym / sponsor ID
- 10001867
- Keywords
- Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease, Interleukin 1 Receptor Antagonist Protein, Anakinra, AMYLOIDOSIS, AIL1RAP
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 10 Sep 2025 (actual)
- Primary completion
- 1 Jul 2031 (estimated)
- Study completion
- 31 Jan 2032 (estimated)
- First posted
- 16 May 2025 (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.
Background:
Anakinra is a drug used to treat people with certain diseases that affect their immune systems. Sometimes anakinra can cause proteins under the skin to clump together. These clumps are called amyloidosis; they can spread to other organs. The only way to diagnose amyloidosis is to remove a piece of tissue (biopsy). Researchers want to find a way to locate amyloidosis in internal organs using positron emission tomography (PET)/computed tomography (CT).
Objective:
To test a new tracer used during PET/CT scans in people with amyloidosis. A tracer is a radioactive dye injected into the body.
Eligibility:
Adults aged 18 years or older with amyloidosis from anakinra injections. They must be enrolled in NIH protocol 17-I-0016.
Design:
Participants will come to the clinic once every 6 months for 2 years. Each visit will be 1 day.
They will have a PET/CT scan with the new tracer at each visit: The tracer will be given through a tube attached to a needle inserted into a vein.
The PET/CT scanner is a machine shaped like a doughnut. Participants will lie still on a padded table. The table will move in and out of the machine. The scan takes about 1 hour.
Radiation from the tracer will remain in the body for 24 hours after each scan. Participants will need to follow rules to avoid exposing pets and other people.
Participants will collect a 24-hour urine sample before each visit. They will also have blood tests and a physical exam at each visit.
Participants will receive a follow-up phone call about 1 week after each visit.
Detailed description
Study Description:
This is a phase 1 study to evaluate the feasibility of an investigational positron emission tomography (PET)/computed tomography (CT) radiotracer, 124I-AT-01, to screen for anakinra-induced amyloidosis in people with the cryopyrin-associated autoinflammatory syndromes (CAPS) (Muckle-Wells syndrome [MWS] or neonatal onset multisystem inflammatory disease [NOMID]), and to follow resolution of amyloidosis. This radiotracer binds to amyloid, so a PET/CT scan can show the presence and relative magnitude of amyloid.
Participants aged 18 years and older who developed local skin or systemic amyloidosis as a result of anakinra treatment will be recruited. They will have a PET/CT scan with radiotracer 124I-AT-01 about once every 6 months for 2 years. Blood and urine will be collected for clinical safety analyses, but no specimen will be collected for research or storage purposes.
The 124I-AT-01 PET/CT imaging is expected to be able to detect anakinra-induced amyloid in the skin and other organs (such as kidney, liver, and spleen). The percent change in quantitative uptake of 124I-AT-01 from baseline to each subsequent scan is expected to correlate with changes in clinical features related to load of amyloid in the body.
Primary Objectives:
- To screen participants with fixed skin thickening at the site of anakinra injection and/or biopsy-proven anakinra-induced amyloidosis of the skin for the presence of systemic amyloidosis, using 124I-AT-01 PET/CT imaging.
- To measure changes in organ-specific uptake of 124I-AT-01 from baseline in participants with anakinra-induced amyloidosis (local or systemic) using PET/CT imaging.
- To assess the safety profile of 124I-AT-01 in participants with CAPS.
Primary Endpoints:
Change in organ-specific 124I-AT-01 uptake from baseline PET/CT imaging to the follow-up PET/CT imaging every 6 months for 2 years. Frequency of CAPS disease flares after exposure to 124I-AT-01.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventional | Experimental | Drug: 124I AT-01Participants aged 18 years and older with anakinra-type amyloidosis (n=10) will be recruited from NIH protocol 17-I-0016. Upon confirmation of eligibility, they will undergo a PET/CT scan with the investigational radiotracer 124I-AT-01, which selectively binds to amyloid fibers. Blood and urine will also be collected for clinical and research analyses, including measurement of IL-1RA and other biomarkers. These scans and sample collections will be repeated about once every 6 months for 2 years. |
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Aged 18 years and older.
- Currently enrolled on NIH protocol 17-I-0016 with a documented diagnosis of MWS or NOMID.
- Agree to allow data collected in this study to be shared with and stored on NIH protocol 17-I-0016 for that study s research analyses.
- Developed skin thickening at the site of anakinra injection.
- Participants who can become pregnant or who can impregnate their partner must agree to use 2 highly effective methods of contraception, at least 1 of which must be a barrier method, when engaging in sexual activities that can result in pregnancy, beginning 28 days prior to baseline until 90 days after the last PET/CT scan. Acceptable methods of contraception include the following:
Key exclusion criteria (6 listed)
- Known hypersensitivity to 124I-AT-01, AT-01, or any of their excipients.
- Known hypersensitivity to KI.
- Pregnant or breastfeeding.
- Currently receiving dialysis.
- Currently taking heparin or heparin derivatives (eg, low molecular weight heparins) or other blood thinners for anticoagulation.
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 |
|---|---|---|
| 124I AT-01124I-AT-01 is an amyloid-reactive synthetic 45-L amino acid polypeptide radiolabeled with iodine-124, with a theoretical molecular weight of 4763.6 Da (based on amino acid sequence). The polypeptide, AT-01, is not pharmacologically active. 124I-AT-01 binds many forms of human and murine amyloid and is intended to be a PET imaging agent for the detection of amyloid deposits. | Drug | Interventional |
Primary outcome
Change in organ-specific 124I AT-01 uptake from baseline PET/CT imaging to the follow up PET/CT imaging every 6 months for 2 years.
Time frame: 2 years
The gold standard for detecting amyloid deposits is tissue biopsy. With this compound, we expect to detect evidence of tissue deposits in the tissue at a much earlier stage and in a non invasive manner. By lowering the exposure to anakinra in participants with anakinra-associated systemic amyloidosis, the burden of amyloidosis is expected to reduce over time.
Frequency of CAPS disease flares after exposure to 124I AT 01.
Time frame: Through end of study.
This compound has not been used in patients with CAPS. We will monitor the participants to detect possible inflammatory disease flare following the exposure.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2026.
- Background: PubMed 37488949Alehashemi S, Dasari S, Metpally A, Uss K, Castelo-Soccio LA, Heller T, Kellman P, Chen MY, Ahlman M, Kim J, Wargo S, Kuhns DB, Fink D, de Jesus A, Martin PS, Chang R, Bolanos J, Lee CR, Nasr SH, Goldbach-Mansky R, McPhail E. Anakinra-Associated Systemic Amyloidosis. Arthritis Rheumatol. 2024 Jan;76(1):100-106. doi: 10.1002/art.42664. Epub 2023 Nov 29.
- Background: PubMed 38245224Nasr SH, Alehashemi S, Dasari S, Waldman M, Afzali B, Chiu A, Bolanos J, Goldbach-Mansky R, McPhail ED. Anakinra-associated renal amyloidosis. Kidney Int. 2024 Feb;105(2):395-396. doi: 10.1016/j.kint.2023.08.020. No abstract available.
- Background: PubMed 36223107Alehashemi S, Dasari S, de Jesus AA, Cowen EW, Lee CR, Goldbach-Mansky R, McPhail ED. Anakinra-Associated Amyloidosis. JAMA Dermatol. 2022 Dec 1;158(12):1454-1457. doi: 10.1001/jamadermatol.2022.2124.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. IPD will be shared in accordance with the NIH Data Management and Sharing Policy.
Related trials for Cryopyrin-Associated Periodic Syndromes
RecruitingObservational study
Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases (NOMID/CAPS, DIRA, CANDLE, SAVI, NLRC4-MAS, Still'S-like Diseases, and Other Undifferentiated Autoinflammatory Diseases)
NOMID, DIRA
RecruitingObservational study
Ilaris NIS in Korea
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Recruiting
Circadian Rhythm Deregulation in Patients With CAPS
Cryopyrin Associated Periodic Syndrome, Familial Cold Urticaria
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.
Sara Alehashemi, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 124I AT-01 in people with Cryopyrin-Associated Periodic Syndromes and Amyloidosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site 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 15 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 19 May 2026. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sara Alehashemi, M.D. ((000) 000-0000, sara.alehashemi@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in organ-specific 124I AT-01 uptake from baseline PET/CT imaging to the follow up PET/CT imaging every 6 months for 2 years. (time frame: 2 years). 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?
On the ClinicalTrials.gov record for NCT06974877, which also lists 3 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 19 May 2026.