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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Alnylam Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02706886
First received: March 3, 2016
Last updated: February 13, 2017
Last verified: February 2017
  Purpose
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.

Condition Intervention Phase
Primary Hyperoxaluria Type 1 (PH1)
Drug: ALN-GO1
Drug: Sterile Normal Saline (0.9% NaCl)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant
Primary Purpose: Treatment
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 Adult Subjects, and Patients With Primary Hyperoxaluria Type 1

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

  • 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 ]

Estimated Enrollment: 60
Study Start Date: March 2016
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-GO1 Drug: ALN-GO1
Single or multiple doses of ALN-GO1 by subcutaneous (sc) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:   6 Years to 64 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria for Parts A and B:

  • Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.
  • Willing to provide written informed consent and to comply with study requirements.

Additional Inclusion Criteria for Part B:

  • Confirmation of PH1 disease
  • Meet 24 hour urine oxalate excretion requirements
  • Estimated GFR of >45 mL/min/1.73m2.
  • If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion Criteria for Parts A and B:

  • No clinically significant health concerns (with the exception of PH1 for patients in Part B)
  • Clinically significant ECG abnormalities
  • Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant
  • Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study
  • Known history of allergic reaction to an oligonucleotide or GalNAc
  • History of intolerance to subcutaneous injection
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02706886

Contacts
Contact: Alnylam Clinical Trials Hotline Call for Complete Site List 617-575-7400
Contact: Alnylam Clinical Trials Hotline Call for Complete Site List 1-866-330-0326

Locations
France
Clinical Trial Site Recruiting
Bordeaux, France
Clinical Trial Site Recruiting
Lyon, France
Clinical Trial Site Recruiting
Paris, France
United Kingdom
Clinical Trial Site Recruiting
Birmingham, United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Patrick Haslett, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02706886     History of Changes
Other Study ID Numbers: ALN-GO1-001
Study First Received: March 3, 2016
Last Updated: February 13, 2017
Individual Participant Data  
Plan to Share IPD: Undecided

Keywords provided by Alnylam Pharmaceuticals:
PH1
Primary Hyperoxaluria
RNAi therapeutic
siRNA
AGT

Additional relevant MeSH terms:
Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on March 29, 2017