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Phase 3 N=225 Randomized Double-blind Treatment

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

TTR-mediated Amyloidosis · Amyloidosis, Hereditary · Amyloid Neuropathies, Familial · Familial Amyloid Polyneuropathies · Amyloid Neuropathies

Bottom Line

View on ClinicalTrials.gov: NCT01960348 ↗
Enrolled (actual)
225
Serious AEs
37.8%
Results posted
Sep 2018
Primary outcome: Primary: Modified Neuropathy Impairment Score +7 (mNIS+7) — -6.03; 27.96 score on a scale — p=<0.0000001

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
patisiran (ALN-TTR02) (Drug); Sterile Normal Saline (0.9% NaCl) (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Alnylam Pharmaceuticals
Primary completion
Aug 2017

Outcome Measures

OutcomeResultp-value
PRIMARY
Modified Neuropathy Impairment Score +7 (mNIS+7)		 -6.03; 27.96 <0.0000001 sig
SECONDARY
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire		 -6.7; 14.4 <0.0000001 sig
SECONDARY
Neurological Impairment Score-Weakness (NIS-W) Score		 0.05; 17.93 <0.0000001 sig
SECONDARY
Rasch-built Overall Disability Scale (R-ODS) Score		 0.0; -8.9 <0.0000001 sig
SECONDARY
Timed 10-meter Walk Test (10-MWT, Gait Speed)		 0.077; -0.235 <0.0000001 sig
SECONDARY
Modified Body Mass Index (mBMI)		 -3.7; -119.4 <0.0000001 sig
SECONDARY
Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31])		 -5.29; 2.24 0.0008 sig

Summary

The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.

Eligibility Criteria

Inclusion Criteria

  • Male or female of 18 to 85 years of age (inclusive);
  • Have a diagnosis of FAP
  • Neuropathy Impairment Score requirement of 5-130
  • Meet Karnofsky performance status requirements
  • Have adequate complete blood counts and liver function tests
  • Have adequate cardiac function
  • Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)

Exclusion Criteria

  • Had a prior liver transplant or is planned to undergo liver transplant during the study period;
  • Has untreated hypo- or hyperthyroidism;
  • Has known human immunodeficiency virus (HIV) infection;
  • Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
  • Recently received an investigational agent or device
  • Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01960348). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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