What the trial was testing
The trial enrolled 22 patients with amyloidosis. The study was sponsored by Boston Medical Center and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
86% of patients had their disease markers drop significantly within weeks of starting treatment.
Blood · 2020 · NCT02841033
These findings — that had their disease markers drop significantly — were published in the Blood and represent the headline result of the study.
Researchers tracked outcomes across 22 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This was an initial testing (Phase 2) study and daratumumab is not yet FDA-approved specifically for AL amyloidosis, though it is approved for multiple myeloma. The results are promising for people whose disease has returned after other treatments. Ask your doctor whether daratumumab might be an option for you through a clinical trial or off-label use.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open amyloidosis trials
Quantitative-imaging in Cardiac Transthyretin Amyloidosis
Transthyretin amyloid cardiomyopathy (ATTR-CM), is a heart muscle disease that's stops the heart muscle working properly. With an ageing population, it is increasingly common but untreated, it has a poor prognosis. Several novel expensive treatments have become available, although we do not understand exactly how they work and why some patients respond, and others do not. The challenge is to develop better methods for monitoring the effects of these treatments, maximizing their benefits and cost-effectiveness. In I-CARE we aim to bring a new imaging technique, named 18F-fluoride PET, to the clinic and thereby improve the care of patients with ATTR-CM. Hypotheses: 1. A delayed imaging protocol and state-of-the-art PET motion correction will optimise 18F-fluoride imaging in ATTR-CM and provide a clear threshold in myocardial TBR values for the diagnosis of ATTR-CM. 2. Optimised 18F-fluoride PET will provide a quantitative marker of the ATTR-CM burden that will allow disease progression and treatment response to be tracked. 3. Myocardial 18F-fluoride TBR values will reduce in patients responding to tafamidis treatment and increase in non-responders and patients not receiving therapy
Clinical Evaluation of a Toothpaste for Dental Plaque Reduction and Prevention of Gum Problems
Qualified subjects will be enrolled and randomized to either one of the three study groups described above based on their initial modified gingivitis and plaque scores. Subjects will be instructed to use the products according to the instructions provided. Subjects will return to the dental office for evaluation after three months and six months of product use. All subjects will be followed for adverse events throughout the study.