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Trial record 1 of 1 for:
ALN-GO1
Study of ALN-GO1 in Healthy Adult Subjects and Patients With Primary Hyperoxaluria Type 1
This study is currently recruiting participants.
Verified July 2017 by Alnylam Pharmaceuticals
Sponsor:
Alnylam Pharmaceuticals
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02706886
First received: March 3, 2016
Last updated: July 20, 2017
Last verified: July 2017
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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 405 days; Part B (MAD phase): Up to 546 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 169 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 169 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 169 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 169 days ]t½
- 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 ]
- The effect of ALN-GO1 on urinary glycolate excretion [ Time Frame: Part B (MAD phase): Up to 546 days ]
- The effect of ALN-GO1 on urinary oxalate excretion [ Time Frame: Part B (MAD phase): Up to 546 days ]
| Estimated Enrollment: | 64 |
| Study Start Date: | March 2016 |
| Estimated Study Completion Date: | January 2020 |
| Estimated Primary Completion Date: | February 2019 (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:
- 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
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: | Tracy McGregor, MD, MSCI | 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: | July 20, 2017 |
| Individual Participant Data (IPD) Sharing Statement: | |
| 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 July 27, 2017