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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-22

Study Completion Date

2023-05-31

Brief Summary

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

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Detailed Description

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Conditions

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Rett Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Drug - trofinetide

Oral dose of trofinetide

Group Type EXPERIMENTAL

Trofinetide

Intervention Type DRUG

Trofinetide solution of 10-30 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Interventions

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Trofinetide

Trofinetide solution of 10-30 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Years

Maximum Eligible Age

5 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No