When a clinical trial ends, participants typically stop taking the study drug until it receives approval from their country’s regulatory agency. If participants are benefiting from the drug and would have no other treatment options without it, they may be allowed to continue taking it as part of an open-label extension (OLE). In an OLE, all participants receive the study drug and know they are receiving it. Only people who have completed the initial portion of the study can take part in an OLE.
OLEs often follow phase 3 trials, the last phase before regulatory approval. They also commonly follow phase 1 or phase 2 trials of ALS/MND treatments. Many organizations strongly advocate for including some form of OLE in all industry-supported clinical trials.
OLEs are an act of compassion for people living with serious illnesses such as ALS/MND who take part in clinical trials. ALS/MND is a severe disease, and people who volunteer for clinical trials make a sacrifice, knowing they may receive a placebo. For this reason, everyone who participates should have access to an OLE that could maximize the potential benefit of the treatment, should it ultimately prove effective.
Scientific gain
An OLE benefits trial participants directly. A well-designed OLE can also provide useful data about the therapy that could benefit more people. OLEs often extend a trial by several months, allowing researchers to collect more information on the long-term efficacy, tolerability, and safety of the drug.
However, the validity of this data is limited because participants know they are receiving the treatment. This may create a placebo effect, which makes the drug’s benefit appear greater only because participants believe it should work.
The Alliance position
The Alliance believes that open-label extension studies must be incorporated into all clinical trial designs and protocols. This ensures that people living with ALS/MND can continue to access experimental therapies once their participation in a clinical trial has ended.