Clinical Trial: Study of ALN-GO1 in Healthy Adult Subjects and Patients With Primary Hyperoxaluria Type 1

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adu

Brief Summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ALN-GO1 in healthy adult volunteers and subjects with PH1.

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome: The safety of ALN-GO1 evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Part A (SAD phase): Up to 181 days; Part B (MAD phase): Up to 321 days ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 141 days ]
    Cmax
  • Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 141 days ]
    tmax
  • Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 141 days ]
    AUC
  • Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 141 days ]
    t½
  • The effect of ALN-GO1 on plasma glycolate concentration [ Time Frame: Part A (SAD phase): Up to 181 days; Part B (MAD phase): Up to 321 days ]
  • The effect of ALN-GO1 on urinary glycolate excretion [ Time Frame: Part B (MAD phase): Up to 321 days ]
  • The effect of ALN-GO1 on urinary oxalate excretion [ Time Frame: Part B (MAD phase): Up to 321 days ]


Original Secondary Outcome: Same as current

Information By: Alnylam Pharmaceuticals

Dates:
Date Received: March 3, 2016
Date Started: March 2016
Date Completion: February 2018
Last Updated: February 13, 2017
Last Verified: February 2017