The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease

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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-004407-23-GB

Enrollment

Registered

2015-12-21

Start date

2016-02-10

Completion date

Unknown

Last updated

2020-07-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders

Interventions

Product Name: ALN-GO1 Product Code: ALN-GO1 Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-65585 Current Sponsor code: ALN-65585 Concentration unit: mg/ml milligram(s)/millilitre

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion Criteria for Parts A and B: 1. Male and female subjects aged 18-64 years (or age of legal consent, whichever is older), inclusive (Part A) and 6-64 years, inclusive (Part B). 2. Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. 3. Willing to provide written informed consent and to comply with study requirements. Additional Inclusion Criteria for Part B: 4. confirmation of PH1 disease 5. 24-hour urinary oxalate excretion of >0.7 mmol/1.73m2/day. 6.Estimated GFR of >45 mL/min/1.73m2. 7. If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days Are the trial subjects under 18? yes Number of subjects for this age range: 16 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Exclusion Criteria for Parts A and B: 1. Any uncontrolled or serious disease, or any medical or surgical condition (with the exception of PH1 for patients in Part B) that may either interfere with participation in the clinical study, and/or put the subject at significant risk (according to the Investigator’s judgment) if he/she participates in the clinical study. 2. Mental illness, alcoholism, drug abuse, or heavy smokers and users of nicotine 3. History of multiple drug allergies or intolerance to subcutaneous injection 4. Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study 5. Known history of allergic reaction to an oligonucleotide or GalNAc 6. History of intolerance to SC injection or relevant abdominal scarring 7. Women who are pregnant or breast feeding Part B only 8. Echocardiography (ECHO) assessment of normal left ventricular systolic function, defined as left ventricular ejection fraction the upper limit of normal (ULN) at screening

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety and tolerability of single- and multiple-ascending doses of ALN-GO1, respectively, in healthy adult subjects and in patients with PH1;Secondary Objective: -To characterize the PK of ALN-GO1 in healthy adult subjects and PH1 patients -To assess the PD effect of ALN-GO1 on plasma glycolate;Primary end point(s): The safety of ALN-GO1 evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation;Timepoint(s) of evaluation of this end point: Part A (SAD phase): 1) assessed throughout to Day 57 and 2) until plasma glycolate decreases to a level that is no more than 20% above of baseline or until plasma glycolate is below the upper limit of normal Part B (MAD phase): assessed throughout to 12 weeks (84 days) after the last dose of study drug and until either: - enrolment into the open label extension study - or until recovery of 24-hour urinary oxalate to >80% of baseline and recovery of plasma glycolate to <20% above baseline or = the upper limit of normal. - or until the investigator determines that the patient can discontinue follow-up, where this is endorsed by the Safety Review Committee based upon ALN-GO1 safety data and the individual patient’s safety and pharmacodynamic data	—

Measure

Time frame

Main Objective: To evaluate the safety and tolerability of single- and multiple-ascending doses of ALN-GO1, respectively, in healthy adult subjects and in patients with PH1;Secondary Objective: -To characterize the PK of ALN-GO1 in healthy adult subjects and PH1 patients -To assess the PD effect of ALN-GO1 on plasma glycolate;Primary end point(s): The safety of ALN-GO1 evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation;Timepoint(s) of evaluation of this end point: Part A (SAD phase): 1) assessed throughout to Day 57 and 2) until plasma glycolate decreases to a level that is no more than 20% above of baseline or until plasma glycolate is below the upper limit of normal Part B (MAD phase): assessed throughout to 12 weeks (84 days) after the last dose of study drug and until either: - enrolment into the open label extension study - or until recovery of 24-hour urinary oxalate to >80% of baseline and recovery of plasma glycolate to <20% above baseline or = the upper limit of normal. - or until the investigator determines that the patient can discontinue follow-up, where this is endorsed by the Safety Review Committee based upon ALN-GO1 safety data and the individual patient’s safety and pharmacodynamic data

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Secondary

Measure	Time frame
Secondary end point(s): The Pharmacokinetics (PK) of ALN-GO1 (Cmax, tmax, AUC, t1/2) The Pharmacodynamics (PD) of ALN-GO1;Timepoint(s) of evaluation of this end point: Part A (SAD phase): 1) assessed throughout to Day 57 and 2) until plasma glycolate decreases to a level that is no more than 20% above of baseline or until plasma glycolate is below the upper limit of normal Part B (MAD phase): assessed throughout to 12 weeks (84 days) after the last dose of study drug and until either: - enrolment into the open label extension study - or until recovery of 24-hour urinary oxalate to >80% of baseline and recovery of plasma glycolate to <20% above baseline or = the upper limit of normal. - or until the investigator determines that the patient can discontinue follow-up, where this is endorsed by the Safety Review Committee based upon ALN-GO1 safety data and the individual patient’s safety and pharmacodynamic data	—

Measure

Time frame

Secondary end point(s): The Pharmacokinetics (PK) of ALN-GO1 (Cmax, tmax, AUC, t1/2) The Pharmacodynamics (PD) of ALN-GO1;Timepoint(s) of evaluation of this end point: Part A (SAD phase): 1) assessed throughout to Day 57 and 2) until plasma glycolate decreases to a level that is no more than 20% above of baseline or until plasma glycolate is below the upper limit of normal Part B (MAD phase): assessed throughout to 12 weeks (84 days) after the last dose of study drug and until either: - enrolment into the open label extension study - or until recovery of 24-hour urinary oxalate to >80% of baseline and recovery of plasma glycolate to <20% above baseline or = the upper limit of normal. - or until the investigator determines that the patient can discontinue follow-up, where this is endorsed by the Safety Review Committee based upon ALN-GO1 safety data and the individual patient’s safety and pharmacodynamic data

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Countries

France, Germany, Israel, Jordan, Netherlands, United Kingdom, United States

Contacts

Public ContactClinical Trial Hotline

Alnylam Pharmaceuticals Inc

clinicaltrials@alnylam.com001866330 0326

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026