A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout

March 28, 2024 updated by: Alnylam Pharmaceuticals

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
44

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Part A: ALN-XDH
A single dose of ALN-XDH administered by subcutaneous (SC) injection.
Drug: ALN-XDH
ALN-XDH administered by SC injection
Placebo Comparator: Part A: Placebo
A single dose of placebo administered by SC injection.
Drug: Placebo
Placebo administered by SC injection
Experimental: Part B: ALN-XDH Single Dose
A single dose of ALN-XDH administered by SC injection.
Drug: ALN-XDH
ALN-XDH administered by SC injection
Experimental: Part B: ALN-XDH Multiple Dose
Multiple doses of ALN-XDH administered by SC injection.
Drug: ALN-XDH
ALN-XDH administered by SC injection
Placebo Comparator: Part B: Placebo
Multiple doses of placebo administered by SC injection.
Drug: Placebo
Placebo administered by SC injection
Experimental: Part C: ALN-XDH
A single dose of ALN-XDH administered by SC injection.
Drug: ALN-XDH
ALN-XDH administered by SC injection
Placebo Comparator: Part C: Placebo
A single dose of placebo administered by SC injection.
Drug: Placebo
Placebo administered by SC injection

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Part A: Frequency of Adverse Events
Time Frame: Up to 10 months
Up to 10 months
Parts B and C: Frequency of Adverse Events
Time Frame: Up to 14 months
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
Up to 12.25 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Part A: Maximum Plasma Concentration (Cmax) for ALN-XDH and Potential Metabolites
Time Frame: Day 1 predose and up to 48 hours postdose
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
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
Day 1 predose and up to 24 hours postdose
Part A: Percent Change from Baseline in sUA
Time Frame: Baseline through Month 9
Baseline through Month 9
Parts B and C: Percentage of Participants with sUA <5 mg/dL
Time Frame: Up to 12.25 months
Up to 12.25 months
Parts B and C: Percentage Change from Baseline in sUA Level
Time Frame: Up to 12.25 months
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
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
Treatment-associated gout flares are defined as gout flares that occur within 6 months of study drug initiation.
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
Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Xanthine
Time Frame: Baseline through Month 6
Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Xanthine
Time Frame: Baseline through Month 6
Baseline through Month 6
Parts B and C: Percent Change from Baseline in Serum Hypoxanthine
Time Frame: Baseline through Month 6
Baseline through Month 6
Parts B and C: Percent Change from Baseline in 24-hour Urine Hypoxanthine
Time Frame: Baseline through Month 6
Baseline through Month 6
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
Day 1 predose and up to 48 hours postdose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alnylam Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 25, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 25, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 25, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 16, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 16, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 25, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 29, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 28, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALN-XDH-001
  • 2021-005773-68 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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