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

Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

Status and phase

Completed
Phase 2

Conditions

RNAi Therapeutic
PH1
siRNA
Primary Hyperoxaluria
AGT

Treatments

Drug: Lumasiran

Study type

Interventional

Funder types

Industry

Identifiers

NCT03350451
2016-003134-24 (EudraCT Number)
ALN-GO1-002

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.

Enrollment

20 patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Enrollment within 12 months of completion of Study ALN-GO1-001
  • In the opinion of the investigator tolerated the study drug
  • If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration
  • Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
  • Willing to provide written informed consent and to comply with study requirements

Exclusion criteria

  • Clinically significant health concerns (with the exception of PH1)
  • Clinically significant cardiovascular abnormality
  • Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant
  • Requirement for chronic dialysis

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

20 participants in 2 patient groups

Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M
Experimental group
Description:
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).
Treatment:
Drug: Lumasiran
Lumasiran (ALN-GO1): 3.0 mg/kg QM
Experimental group
Description:
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.
Treatment:
Drug: Lumasiran

Trial documents
2

Trial contacts and locations

9

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Data sourced from clinicaltrials.gov

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