GoutPhase 1/2NCT05256810
A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout
A Phase 1/2 study testing ALN-XDH in people with Gout, run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
Trial summary
The purpose of this study is to: Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in adult patients with gout Part C: Evaluate the safety, tolerability and efficacy of ALN-XDH… Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Gout
- What is being tested?
- ALN-XDH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 25 Feb 2022to 25 Jan 2023
- Phase
- Phase 1/2
- 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Part A: has serum uric acid (sUA) level ≥4 mg/dL and ≤7 mg/dL
- Parts B and C: meets the American College of Rheumatology criteria for acute arthritis of primary gout
- Part B: has an sUA level ≥8 mg/dL while not receiving urate-lowering therapy (ULT) or following a 1-week washout period of prior ULT
- Part C: has been on a stable dose of allopurinol for gout for ≥6 weeks prior to screening
- Part C: has an sUA level ≥6 mg/dL
Who cannot join
- Parts A, B and C: Has received an investigational agent within the last 30 days
- Parts A, B and C: Has experienced or is being treated for acute gout flare(s) within 14 days prior to randomization
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single Ascending and Multiple Dose Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALN-XDH in Healthy Adult Subjects and Adult Patients With Gout
The purpose of this study is to:
- Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants
- Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in adult patients with gout
- Part C: Evaluate the safety, tolerability and efficacy of ALN-XDH as add-on therapy in adult patients with gout
- Conditions
- Gout
- Being tested
- ALN-XDH, Placebo
- Keywords
- Serum uric acid, siRNA
- Medical subject headings
- Gout
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-XDHDrug
ALN-XDH administered by SC injection
PlaceboDrug
Placebo administered by SC injection
| Study group | Role | What they receive |
|---|---|---|
| Part A: ALN-XDH | Experimental | A single dose of ALN-XDH administered by subcutaneous (SC) injection. Receives: Drug: ALN-XDH |
| Part A: Placebo | Placebo comparator | A single dose of placebo administered by SC injection. Receives: Drug: Placebo |
| Part B: ALN-XDH Single Dose | Experimental | A single dose of ALN-XDH administered by SC injection. Receives: Drug: ALN-XDH |
| Part B: ALN-XDH Multiple Dose | Experimental | Multiple doses of ALN-XDH administered by SC injection. Receives: Drug: ALN-XDH |
| Part B: Placebo | Placebo comparator | Multiple doses of placebo administered by SC injection. Receives: Drug: Placebo |
| Part C: ALN-XDH | Experimental | A single dose of ALN-XDH administered by SC injection. Receives: Drug: ALN-XDH |
| Part C: Placebo | Placebo comparator | A single dose of placebo administered by SC injection. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Frequency of Adverse Events
Measured: Up to 10 months
Parts B and C: Frequency of Adverse Events
Measured: Up to 14 months
Parts B and C: Percentage of Participants with Serum Uric Acid (sUA) <6 mg/dL
Measured: Up to 12.25 months
Secondary outcomes (14)
Part A: Area Under the Plasma Concentration-time Curve (AUC) for ALN-XDH and Potential Metabolites
Measured: Day 1 predose and up to 48 hours postdose
Part A: Maximum Plasma Concentration (Cmax) for ALN-XDH and Potential Metabolites
Measured: Day 1 predose and up to 48 hours postdose
Part A: Time to Maximum Plasma Concentration (tmax) for ALN-XDH and Potential Metabolites
Measured: Day 1 predose and up to 48 hours postdose
Part A: Fractional Excreted in Urine (fe) for ALN-XDH and Potential Metabolites
Measured: Day 1 predose and up to 24 hours postdose
Part A: Percent Change from Baseline in sUA
Measured: Baseline through Month 9
Parts B and C: Percentage of Participants with sUA <5 mg/dL
Measured: Up to 12.25 months
Parts B and C: Percentage Change from Baseline in sUA Level
Measured: Up to 12.25 months
Parts B and C: Plasma Concentrations of ALN-XDH and Potential Major Metabolite(s)
Measured: Day 1 and Day 85 predose and up to 4 hours postdose
Parts B and C: Frequency of Treatment-associated Gout Flares
Treatment-associated gout flares are defined as gout flares that occur within 6 months of study drug initiation.
Measured: Up to 6 months
Parts B and C: Percent Change from Baseline in 24-hour Urine Uric Acid (uUA)
Measured: Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Xanthine
Measured: Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Xanthine
Measured: Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Hypoxanthine
Measured: Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Hypoxanthine
Measured: Baseline through Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2022
- Study start25 Feb 2022
- Primary completion25 Jan 2023
- Study completion25 Jan 2023
Why it stopped: The Sponsor elected not to continue with the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GoutStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05256810
- Sponsor's protocol ID
- ALN-XDH-001
- Other IDs
- 2021-005773-68
- Status last verified
- Mar 2024
- First submitted
- 16 Feb 2022
- Last updated
- 29 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
PubMed 41388232
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single Ascending and Multiple Dose Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALN-XDH in Healthy Adult Subjects and Adult Patients With Gout
- ClinicalTrials.gov ID
- NCT05256810
- Acronym / sponsor ID
- ALN-XDH-001
- Conditions
- Gout
- Keywords
- Serum uric acid, siRNA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The Sponsor elected not to continue with the study
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 25 Feb 2022 (actual)
- Primary completion
- 25 Jan 2023 (actual)
- Study completion
- 25 Jan 2023 (actual)
- First posted
- 25 Feb 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.
The purpose of this study is to:
- Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants
- Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in adult patients with gout
- Part C: Evaluate the safety, tolerability and efficacy of ALN-XDH as add-on therapy in adult patients with gout
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Part A: ALN-XDH | Experimental | Drug: ALN-XDHA single dose of ALN-XDH administered by subcutaneous (SC) injection. |
| Part A: Placebo | Placebo comparator | Drug: PlaceboA single dose of placebo administered by SC injection. |
| Part B: ALN-XDH Single Dose | Experimental | Drug: ALN-XDHA single dose of ALN-XDH administered by SC injection. |
| Part B: ALN-XDH Multiple Dose | Experimental | Drug: ALN-XDHMultiple doses of ALN-XDH administered by SC injection. |
| Part B: Placebo | Placebo comparator | Drug: PlaceboMultiple doses of placebo administered by SC injection. |
| Part C: ALN-XDH | Experimental | Drug: ALN-XDHA single dose of ALN-XDH administered by SC injection. |
| Part C: Placebo | Placebo comparator | Drug: PlaceboA single dose of placebo administered by SC injection. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Part A: has serum uric acid (sUA) level ≥4 mg/dL and ≤7 mg/dL
- Parts B and C: meets the American College of Rheumatology criteria for acute arthritis of primary gout
- Part B: has an sUA level ≥8 mg/dL while not receiving urate-lowering therapy (ULT) or following a 1-week washout period of prior ULT
- Part C: has been on a stable dose of allopurinol for gout for ≥6 weeks prior to screening
- Part C: has an sUA level ≥6 mg/dL
Key exclusion criteria (2 listed)
- Parts A, B and C: Has received an investigational agent within the last 30 days
- Parts A, B and C: Has experienced or is being treated for acute gout flare(s) within 14 days prior to randomization
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 |
|---|---|---|
| ALN-XDHALN-XDH administered by SC injection | Drug | Part A: ALN-XDH, Part B: ALN-XDH Multiple Dose, Part B: ALN-XDH Single Dose, Part C: ALN-XDH |
| PlaceboPlacebo administered by SC injection | Drug | Part A: Placebo, Part B: Placebo, Part C: Placebo |
Primary outcome
Part A: Frequency of Adverse Events
Time frame: Up to 10 months
Parts B and C: Frequency of Adverse Events
Time frame: Up to 14 months
Parts B and C: Percentage of Participants with Serum Uric Acid (sUA) <6 mg/dL
Time frame: Up to 12.25 months
14 secondary outcomes
Part A: Area Under the Plasma Concentration-time Curve (AUC) for ALN-XDH and Potential Metabolites
Time frame: Day 1 predose and up to 48 hours postdose
Part A: Maximum Plasma Concentration (Cmax) for ALN-XDH and Potential Metabolites
Time frame: Day 1 predose and up to 48 hours postdose
Part A: Time to Maximum Plasma Concentration (tmax) for ALN-XDH and Potential Metabolites
Time frame: Day 1 predose and up to 48 hours postdose
Part A: Fractional Excreted in Urine (fe) for ALN-XDH and Potential Metabolites
Time frame: Day 1 predose and up to 24 hours postdose
Part A: Percent Change from Baseline in sUA
Time frame: Baseline through Month 9
Parts B and C: Percentage of Participants with sUA <5 mg/dL
Time frame: Up to 12.25 months
Parts B and C: Percentage Change from Baseline in sUA Level
Time frame: Up to 12.25 months
Parts B and C: Plasma Concentrations of ALN-XDH and Potential Major Metabolite(s)
Time frame: Day 1 and Day 85 predose and up to 4 hours postdose
Parts B and C: Frequency of Treatment-associated Gout Flares
Time frame: Up to 6 months
Parts B and C: Percent Change from Baseline in 24-hour Urine Uric Acid (uUA)
Time frame: Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Xanthine
Time frame: Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Xanthine
Time frame: Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Hypoxanthine
Time frame: Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Hypoxanthine
Time frame: Baseline through Month 6
Results and safety
The study was stopped early (reason given: The Sponsor elected not to continue with the study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2024.
- Derived from this study: PubMed 41388232Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
- 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.
Medical Director study director
Alnylam Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ALN-XDH in people with Gout, run by Alnylam Pharmaceuticals 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 5 inclusion and 2 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: stopped early, as of the sponsor's last update on 29 Mar 2024. 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. 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 combined 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.
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 stopped early (reason given: The Sponsor elected not to continue with the 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?
Part A: Frequency of Adverse Events (time frame: Up to 10 months). The study lists 3 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 NCT05256810, which also lists 1 linked publication.
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 29 Mar 2024.