An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome (DAFFODIL™)

Acadia Pharmaceuticals

Status and phase

Terminated

Phase 3

Phase 2

Conditions

Rett Syndrome

Treatments

Drug: Trofinetide

Study type

Interventional

Funder types

Industry

Identifiers

NCT04988867

ACP-2566-009

Details and patient eligibility

About

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

Enrollment

15 patients

Sex

Female

Ages

2 to 5 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Female subject
1. 2 to 4 years of age and body weight ≥9 kg and <20 kg at Screening OR
2. 5 years of age and body weight ≥9 kg and <12 kg at Screening
Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome Diagnostic Criteria
Has a documented disease-causing mutation in the MECP2 gene
Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to Screening
Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 4 weeks prior to Screening

Exclusion criteria

Has been treated with insulin within 12 weeks of Baseline
Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
Has a history of, or current, cerebrovascular disease or brain trauma
Has significant, uncorrected visual or uncorrected hearing impairment
Has a history of, or current, malignancy
Has any of the following:
1. QTcF interval of >450 ms at Screening or Baseline
2. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
3. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
4. Other clinically significant finding on ECG at Screening or Baseline

Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all prespecified entry criteria).