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

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ClinicalTrials.gov Identifier: NCT02706886
Recruitment Status : Recruiting
First Posted : March 11, 2016
Last Update Posted : February 2, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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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.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (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
Study Start Date : March 2016
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020

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U.S. FDA Resources

Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. The safety of ALN-GO1 evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Part A (SAD phase): Up to 405 days; Part B (MAD phase): Up to 546 days ]

Secondary Outcome Measures :
  1. Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 169 days ]
    Cmax

  2. Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 169 days ]
    tmax

  3. Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 169 days ]
    AUC

  4. Profile of pharmacokinetics (PK) of ALN-GO1 [ Time Frame: Part A (SAD phase): Up to 2 days; Part B (MAD phase): Up to 169 days ]
    t½

  5. The effect of ALN-GO1 on plasma glycolate concentration [ Time Frame: Part A (SAD phase): Up to 405 days; Part B (MAD phase): Up to 546 days ]
  6. The effect of ALN-GO1 on urinary glycolate excretion [ Time Frame: Part B (MAD phase): Up to 546 days ]
  7. The effect of ALN-GO1 on urinary oxalate excretion [ Time Frame: Part B (MAD phase): Up to 546 days ]

Eligibility Criteria
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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:

  • 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
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
United States, Minnesota
Clinical Trial Site Recruiting
Rochester, Minnesota, United States
France
Clinical Trial Site Recruiting
Bordeaux, France
Clinical Trial Site Recruiting
Lyon, France
Clinical Trial Site Recruiting
Paris, France
Germany
Clinical Trial Site Recruiting
Bonn, Germany
Israel
Clinical Trial Site Recruiting
Haifa, Israel
Clinical Trial Site Recruiting
Jerusalem, Israel
Jordan
Clinical Trial Site Recruiting
Amman, Jordan
Netherlands
Clinical Trial Site Recruiting
Amsterdam, Netherlands
United Kingdom
Clinical Trial Site Recruiting
Birmingham, United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Tracy McGregor, MD, MSCI Alnylam Pharmaceuticals
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:
For information about the drug ALN-GO1 and Primary Hyperoxaluria Type 1 (PH1), clink this link  This link exits the ClinicalTrials.gov site

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02706886     History of Changes
Other Study ID Numbers: ALN-GO1-001
First Posted: March 11, 2016    Key Record Dates
Last Update Posted: February 2, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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