Both Therachon and Ascendis Pharma have posted new information about their studies in the ClinicalTrials.gov website.
Ascendis
Pharma has already started their natural history study in US, Canada,
UK, Germany and Australia. Check it here. For now there is only one site recruiting, but things change quickly and these reports are not updated in real time.
Unfortunately, their registry does not disclose site contact information. Although it may not be hard to guess who are the investigators and their site locations, interested people could contact Ascendis directly for more information.
What is a natural history study?
The natural history trial is an observational study to track the growth patterns of participants. The objective is to compare these patterns with the growth observed after starting a therapy. As there is still scarce information about growth patterns in achondroplasia in children, these natural history studies would be probably an attempt to fill the information gap and to try to spot changes in growth patterns after drug intervention.
Unfortunately, their registry does not disclose site contact information. Although it may not be hard to guess who are the investigators and their site locations, interested people could contact Ascendis directly for more information.
Therachon
is recruiting participants for their natural history study as
well. Their registry is more complete, disclosing the hospitals where
the sites are located. You can check the list here.
What is a natural history study?
The natural history trial is an observational study to track the growth patterns of participants. The objective is to compare these patterns with the growth observed after starting a therapy. As there is still scarce information about growth patterns in achondroplasia in children, these natural history studies would be probably an attempt to fill the information gap and to try to spot changes in growth patterns after drug intervention.
The developers of vosoritide (Biomarin) also conducted a natural history study before the start of the phase 2 study. However, it is interesting to note that, regardless of the differences seen in growth patterns in their phase 2 trial (see page 1301, abstract #2347W), they were still required to use a standard double-blind, placebo-controlled study in their ongoing phase 3 study. In other words, differences in growth seen after drug intervention when compared with before therapy would be accepted as proof-of-concept but not as a valid efficacy endpoint. In this regard, only a placebo-controlled study would confirm that the drug was working.
As I've already expressed in the past, I think this approach is less than ideal in conditions such as achondroplasia, where interventions can only be successful for a limited time.
After all, growth has an expiry date.
As I've already expressed in the past, I think this approach is less than ideal in conditions such as achondroplasia, where interventions can only be successful for a limited time.
After all, growth has an expiry date.
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