Phase 3
Completed N=225
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
Source: ClinicalTrials.gov NCT01960348 ↗
Enrolled (actual)
225
Serious AEs
37.8%
Results posted
Sep 2018
Primary outcomePrimary: Modified Neuropathy Impairment Score +7 (mNIS+7) — -6.03; 27.96 score on a scale — p=<0.0000001
◆ Published Evidence
Highly cited
461citations · ~66 / year
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
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.
Linked Publications (5)
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Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
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Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis.
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Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study.
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Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial.
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Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study.
Outcome Measures
| Outcome | Result | p-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 |
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
Data sourced from ClinicalTrials.gov (NCT01960348) and the linked publication. 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. Informational only — not medical advice.