1500 Participants Needed

Observational Study for Transthyretin Amyloidosis

(ConTTRibute Trial)

Recruiting at 39 trial locations
AC
Overseen ByAlnylam Clinical Trial Information Line
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Alnylam Pharmaceuticals
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to study ATTR amyloidosis, a condition where abnormal proteins accumulate in the body and cause damage. Researchers seek to understand the progression of this condition and evaluate the effectiveness of two treatments, patisiran and vutrisiran (both RNA interference therapies), in real-world settings. They also aim to track individuals with the genetic marker for the condition who do not yet show symptoms. Suitable candidates for this trial include those diagnosed with ATTR amyloidosis or those carrying the gene known to cause it, but who cannot participate in another clinical trial simultaneously. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important early findings.

Do I have to stop taking my current medications for this trial?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are in Germany, you must be treated according to the approved treatment guidelines for ATTR amyloidosis.

Why are researchers excited about this trial?

Researchers are excited about the ConTTRibute observational study because it offers a comprehensive look at patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) worldwide, which can provide valuable insights into the progression and management of this condition. Unlike treatments that focus solely on managing symptoms, this study aims to gather real-world data that could lead to better understanding of disease patterns and patient outcomes. By collecting and analyzing this data, researchers hope to improve future treatment strategies and potentially uncover new therapeutic targets, ultimately enhancing patient care.

Who Is on the Research Team?

MD

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Are You a Good Fit for This Trial?

Inclusion Criteria

Diagnosis of ATTR amyloidosis or documented known disease-causing TTR mutation for the cohort of pre-symptomatic carriers
Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Study

Participants are observed to describe epidemiological and clinical characteristics, natural history, and real-world clinical management of ATTR amyloidosis patients

Up to 11 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Observational Study

Find a Clinic Near You

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials
81
Recruited
16,100+

Dr. Yvonne Greenstreet

Alnylam Pharmaceuticals

Chief Executive Officer since 2021

MD from the University of Leeds, MBA from INSEAD

Dr. Pushkal Garg

Alnylam Pharmaceuticals

Chief Medical Officer since 2016

MD from Columbia University