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

APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-002987-17-SE

Enrollment

200

Registered

2013-09-13

Start date

2013-11-25

Completion date

Unknown

Last updated

2017-10-30

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

Conditions

Transthyretin mediated amyloidosis (ATTR) MedDRA version: 19.0 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders MedDRA version: 19.0 Level: PT Classification code 10019889 Term: Hereditary neuropathic amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: ALN-TTR02 Product Code: ALN-TTR02 Pharmaceutical Form: Solution for injection INN or Proposed INN: Patisiran Current Sponsor code: Patisiran Other descriptive name: ALN-TTR02 Concentra

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Male or female of 18 to 85 years of age (inclusive); • Have a diagnosis of FAP • Meet Neuropathy Impairment Score requirements and Karnofsky performance status requirements • Have adequate complete blood counts and liver function tests • Have adequate cardiac function • No active infection with hepatitis B or hepatitis C Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 100 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 100

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the efficacy of ALN-TTR02;Secondary Objective: To determine the effect of ALN-TTR02 on various clinical parameters;Primary end point(s): The difference between the ALN TTR02 and placebo groups in the change from baseline of modified Neuropathy Impairment Score+7 (mNIS+7);Timepoint(s) of evaluation of this end point: The difference between the ALN TTR02 and placebo groups in the change from baseline of modified NIS+7 (mNIS+7) at 18 months	—

Measure

Time frame

Main Objective: To determine the efficacy of ALN-TTR02;Secondary Objective: To determine the effect of ALN-TTR02 on various clinical parameters;Primary end point(s): The difference between the ALN TTR02 and placebo groups in the change from baseline of modified Neuropathy Impairment Score+7 (mNIS+7);Timepoint(s) of evaluation of this end point: The difference between the ALN TTR02 and placebo groups in the change from baseline of modified NIS+7 (mNIS+7) at 18 months

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Secondary

Measure	Time frame
Secondary end point(s): The difference between ALN-TTR02 and placebo in the change from baseline in the following measurements: • Quality of Life • Modified Body Mass Index • Autonomic function • Muscle weakness as assessed by walking test ;Timepoint(s) of evaluation of this end point: The difference between ALN-TTR02 and placebo in the change from baseline in the various clinical parameters at 18 months	—

Measure

Time frame

Secondary end point(s): The difference between ALN-TTR02 and placebo in the change from baseline in the following measurements: • Quality of Life • Modified Body Mass Index • Autonomic function • Muscle weakness as assessed by walking test ;Timepoint(s) of evaluation of this end point: The difference between ALN-TTR02 and placebo in the change from baseline in the various clinical parameters at 18 months

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Countries

Argentina, Brazil, Bulgaria, France, Germany, Italy, Korea, Republic of, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactClinical Trials Information

Medpace Finland OY

regsubmissions@medpace.com+46 702524055

Outcome results

None listed

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