An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

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• Hyperoxaluria, Primary 47
• Kidney Diseases 3,843
• Urologic Diseases 197
• Metabolism, Inborn Errors 110
• Genetic Diseases, Inborn 422
• Metabolic Diseases 1,002

Keywords

• Hyperoxaluria, Primary
• Hyperoxaluria
• Kidney Diseases
• Urologic Diseases
• Metabolism, Inborn Errors
• Genetic Diseases, Inborn
• Metabolic Diseases

Hyperoxaluria, Primary

47 studies on the registry are indexed under Hyperoxaluria, Primary; 13 are open to participants now.

This study's enrollment of 20 is below the median of 23 across 29 interventional studies indexed under Hyperoxaluria, Primary.

Lead sponsor

Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) have results posted.

Study arms

• Experimental
  Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M

  Participants enrolling from study 001B (NCT02706886), received lumasiran, subcutaneous (SC) injection, at a starting dose of 1.0 milligrams per kilograms (mg/kg) once monthly (QM) or 3.0 mg/kg once every 3 months \[Q3M\]) from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the Safety Review Committee (SRC).

  Drug: Lumasiran

• Experimental
  Lumasiran (ALN-GO1): 3.0 mg/kg QM

  Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.

  Drug: Lumasiran

Interventions

• DrugLumasiran

  Multiple doses of lumasiran by SC injection

  Also known as: ALN-GO1

Primary outcomes

1. Number of Participants With at Least One Adverse Event (AE)

   AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug.

   Time frame: Baseline (Day -1) up to 54 months

Secondary outcomes

1. Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months

   Oxalate produced by the liver is the key toxic metabolite that drives disease pathology in participants with primary hyperoxaluria type 1 (PH1). The risk of disease complications increase continuously as oxalate levels increase. 24-hour urinary oxalate (millimole \[mmol\]/ 24 hour \[h\]/1.73 meters squared \[m\^2\]) corrected for BSA at each visit per participant was calculated as follows: \[Urine oxalate concentration (micromole per liter \[umol/L\])/1000 (umol/mmol)\]\*\[24hour urine volume (mL)/1000 (mL/L)\]\* \[24 hours/actual collection hours\]\*1.73/(BSA). Baseline was the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicated a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Overall number of participants analyzed are the number of participants with data available for analysis.

   Time frame: Baseline (Day -1) up to 54 months

2. Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months

   Baseline is the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicates a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD.

   Time frame: Baseline (Day -1) up to 54 months

3. Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months

   Baseline was defined as the last measurement prior to the first dose of lumasiran in the ALN-GO1-001 study. eGFR was calculated based on the Modification of Diet in Renal Disease (MDRD) formula for participants \>=18 years of age at enrollment and the Schwartz Bedside formula for participants \<18 years of age at enrollment. eGFR based on MDRD formula was calculated as follows: eGFR (mL/min/1.73 m\^2) = 175 × (serum creatinine {SCr} \[μmol/deciliter(dL)\]/88.4)-1.154 × (age)-0.203 × (0.742, if female), or × (1.212, if African American) and based on Schwartz formula: eGFR (mL/min/1.73m2) = (36.2 × height \[cm\])/ SCr (μmol /dL). Safety analysis set included all participants who received any amount of study drug.

   Time frame: Baseline (Day -1) up to 54 months

Baseline characteristics

Study documents

• Study protocol · May 4, 2020
• Statistical analysis plan · May 23, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.