What is a natural history study?
A natural history study is a specific type of clinical trial that tracks the progression of a disease over time in patients who are receiving their currently available treatments only. The information collected in a natural history study can be used to understand how a disease typically progresses and how an investigational treatment might affect the course of the disease.
Why is Clementia conducting the Natural History Study in patients with FOP?
The Natural History Study will document the patient experience with FOP in a scientifically rigorous manner. The information gathered will be very useful for designing palovarotene’s clinical plan and understanding its place in the treatment of FOP. The Natural History Study will be conducted in parallel with the Phase II clinical trial.
What does Clementia’s Natural History Study entail?
Annual visits will be performed over 3 years, during which the patient will complete questionnaires about their FOP and how it impacts their life, undergo an assessment of range of motion across the major body regions and joints, and have blood tests to assess potential biomarkers of the disease. In addition, the amount of HO as measured by an imaging study will also be performed.
Should a patient experience a flare-up during the 3-year observation period, more careful assessments at the time of the flare-up will also be performed, including x-rays and low-dose CT scan at the flare-up site.
Specific details about the Natural History Study are provided on www.clinicaltrials.gov.
Who can participate in the Natural History Study?
Patients with a diagnosis of FOP due to the R206H mutation between the ages of 18 and 65 years (Part A) and 0 and 65 years (Part B) are eligible to participate. The gene mutation will be determined at study entry, and patients with mutations other than R206H will not be able to participate. Specific study details are provided on www.clinicaltrials.gov.
Will a patient who is enrolled in the Natural History Study be able to enroll in the Phase II clinical trial for palovarotene?
Yes, a patient who is enrolled in the Natural History Study and experiences a flare-up will be able to enroll in the Phase II clinical trial if that patient meets the Phase II clinical trial enrollment criteria and the Phase II clinical trial is still enrolling patients. The patient cannot be enrolled in both trials, so the patient would be discontinued from the Natural History Study once enrolled the Phase II trial.
How is this Natural History Study different from the flare-up survey and the registry the IFOPA is planning?
The flare-up survey was a one-time, cross-sectional patient report of disease and flare-up symptoms in 500 patients, conducted by the Center for Research in FOP & Related Disorders at The University of Pennsylvania.
The Natural History Study is a prospective longitudinal study that will collect specific standardized information over a 3-year period in up to 100 patients.
The IFOPA registry will collect information directly from patients, and eventually collect clinical assessments and test results from doctors. The registry will be available to all FOP patients and doctors worldwide and is expected to last for 10 or 20 years. The choice of information collected in the IFOPA registry will be determined by the flare-up survey, by Clementia’s natural history study, and by experts who will help advise the registry.
All three approaches – the flare-up survey, Clementia’s Natural History Study, and the IFOPA Registry will provide complementary information that will improve the understanding of FOP, and will help to inform the design of trials for investigational products for FOP.