A new paper has been published in Clinical Therapeutics
Assessing Experiences With Trofinetide for Rett Syndrome: Interviews With Caregivers of Participants in Clinical Trials
The paper is based on interviews which were conducted with caregivers of participants in the LILAC/LILAC-2 open-label extension trials and in the DAFFODIL open-label trial in participants aged 2 to 4 years. The caregivers were asked about the effect of Rett syndrome on their child, their experiences with trofinetide, the meaningfulness of treatment effects, and their level of satisfaction.
The following is the conclusion from the interviews but I urge you to read the full paper, especially for the direct quotations from the carers.
Conclusions
Caregivers of participants in the DAFFODIL and LILAC/LILAC-2 clinical trials reported sustained and meaningful improvements with trofinetide treatment, giving insight into the effect of trofinetide in individuals experiencing different stages of the course of RTT. The improvements had significant impact on the social and physical function of participants, including better ability to communicate and interact with others and be included in family activities.
The results of these qualitative interviews provide a detailed description of the wide-ranging and profound effects of RTT as well as insight into the range and meaningfulness of improvements that patients receiving trofinetide experienced as observed by caregivers. The results and direct quotations from caregivers provide depth and detail regarding the treatment effects of trofinetide that were initially reported in the phase 3 LAVENDER trial and that continued to improve over the long-term in the LILAC/LILAC-2 extension studies as well as in the DAFFODIL trial of younger girls with RTT. These patient-experience data, although proxy-reported by caregivers, address the consistent requests of the FDA for data supporting the relevance and meaningfulness of treatment benefit and provide valuable insight for regulators, patient communities, health care providers, and payers. To our knowledge, this is the first caregiver-focused report of treatment effects for RTT.
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