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NCT Number: NCT01960348

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

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.

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Key information

Conditions

TTR-mediated Amyloidosis Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Amyloidosis, Hereditary Amyloidosis, Hereditary, Transthyretin-Related Congenital, Hereditary, and Neonatal Diseases and Abnormalities Familial Amyloid Polyneuropathies Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Proteostasis Deficiencies

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

patisiran (ALN-TTR02)

Drug

administered by intravenous (IV) infusion

Sterile Normal Saline (0.9% NaCl)

Drug

administered by intravenous (IV) infusion

Primary outcomes

  1. Modified Neuropathy Impairment Score +7 (mNIS+7)

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.

Secondary outcomes

  1. Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in Norfolk QoL-DN at 18 months. The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.

  2. Neurological Impairment Score-Weakness (NIS-W) Score

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in NIS-W at 18 months. NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The minimum and maximum values are 0 and 192, respectively. A higher score indicates a worse outcome.

  3. Rasch-built Overall Disability Scale (R-ODS) Score

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in R-ODS score at 18 months. The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations in patients. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.

  4. Timed 10-meter Walk Test (10-MWT, Gait Speed)

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in 10-MWT at 18 months. Ability to ambulate (gait speed) was assessed through the 10-meter walk test (10-MWT). The walk had to be completed without assistance from another person; ambulatory aids such as canes and walkers were permitted.

  5. Modified Body Mass Index (mBMI)

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mBMI at 18 months. The nutritional status of patients was evaluated using the mBMI; calculated as the product of BMI (weight in kilograms divided by the square of height in meters) and serum albumin (g/L).

  6. Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31])

    Time frame: 18mo

    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in COMPASS 31 at 18 months. The COMPASS 31 is a measure of autonomic neuropathy symptoms. The questions evaluated 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor). The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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)

Important dates

Study start
2013
Primary completion
2017
Study completion
2017
First posted
Oct 10, 2013
Registry last updated
Apr 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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