NCT05256810

Brief 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 as add-on therapy in adult patients with gout

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
44

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Feb 2022

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 16, 2022

Completed
9 days until next milestone

First Posted

Study publicly available on registry

February 25, 2022

Completed
Same day until next milestone

Study Start

First participant enrolled

February 25, 2022

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 25, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 25, 2023

Completed
Last Updated

March 29, 2024

Status Verified

March 1, 2024

Enrollment Period

11 months

First QC Date

February 16, 2022

Last Update Submit

March 28, 2024

Conditions

Gout

Keywords

Serum uric acidsiRNA

Outcome Measures

Primary Outcomes (3)

  • Part A: Frequency of Adverse Events

    Up to 10 months

  • Parts B and C: Frequency of Adverse Events

    Up to 14 months

  • Parts B and C: Percentage of Participants with Serum Uric Acid (sUA) <6 mg/dL

    Up to 12.25 months

Secondary Outcomes (14)

  • Part A: Area Under the Plasma Concentration-time Curve (AUC) for ALN-XDH and Potential Metabolites

    Day 1 predose and up to 48 hours postdose

  • Part A: Maximum Plasma Concentration (Cmax) for ALN-XDH and Potential Metabolites

    Day 1 predose and up to 48 hours postdose

  • Part A: Time to Maximum Plasma Concentration (tmax) for ALN-XDH and Potential Metabolites

    Day 1 predose and up to 48 hours postdose

  • Part A: Fractional Excreted in Urine (fe) for ALN-XDH and Potential Metabolites

    Day 1 predose and up to 24 hours postdose

  • Part A: Percent Change from Baseline in sUA

    Baseline through Month 9

  • +9 more secondary outcomes

Study Arms (7)

Part A: ALN-XDH

EXPERIMENTAL

A single dose of ALN-XDH administered by subcutaneous (SC) injection.

Drug: ALN-XDH

Part A: Placebo

PLACEBO COMPARATOR

A single dose of placebo administered by SC injection.

Drug: Placebo

Part B: ALN-XDH Single Dose

EXPERIMENTAL

A single dose of ALN-XDH administered by SC injection.

Drug: ALN-XDH

Part B: ALN-XDH Multiple Dose

EXPERIMENTAL

Multiple doses of ALN-XDH administered by SC injection.

Drug: ALN-XDH

Part B: Placebo

PLACEBO COMPARATOR

Multiple doses of placebo administered by SC injection.

Drug: Placebo

Part C: ALN-XDH

EXPERIMENTAL

A single dose of ALN-XDH administered by SC injection.

Drug: ALN-XDH

Part C: Placebo

PLACEBO COMPARATOR

A single dose of placebo administered by SC injection.

Drug: Placebo

Interventions

ALN-XDHDRUG

ALN-XDH administered by SC injection

Part A: ALN-XDHPart B: ALN-XDH Multiple DosePart B: ALN-XDH Single DosePart C: ALN-XDH
PlaceboDRUG

Placebo administered by SC injection

Part A: PlaceboPart B: PlaceboPart C: Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Trial Site

London, United Kingdom

Location

Related Publications (1)

  • 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. 2025 Dec 12. doi: 10.1007/s40262-025-01606-0. Online ahead of print.

    PMID: 41388232DERIVED

MeSH Terms

Conditions

Gout

Condition Hierarchy (Ancestors)

ArthritisJoint DiseasesMusculoskeletal DiseasesCrystal ArthropathiesRheumatic DiseasesPurine-Pyrimidine Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 16, 2022

First Posted

February 25, 2022

Study Start

February 25, 2022

Primary Completion

January 25, 2023

Study Completion

January 25, 2023

Last Updated

March 29, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)