Efficacy and Safety of SYNB1618 in Adult Patients With Phenylketonuria (SynPheny-1)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04534842|
Recruitment Status : Recruiting
First Posted : September 1, 2020
Last Update Posted : November 12, 2020
|Condition or disease||Intervention/treatment||Phase|
|Phenylketonuria||Drug: SYNB1618||Phase 2|
This is an open-label, single-arm Phase 2 study; all participating PKU patients will receive active study drug, SYNB1618. This study is evaluating a dose-ramp regimen consisting of 4 dose levels of SYNB1618 over 15 days of treatment.
This study has been designed with the flexibility of being able to be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g., patient's home, hotel).
Patients will be screened for eligibility and complete a customized diet run-in period prior to starting the 15-day dose ramp ('treatment period'). During the treatment period patients will be required to adhere to the same customized diet menus as in the diet run-in period.
The efficacy of SYNB1618 will be assessed in this study by measuring the reduction of the area under the curve (AUC) for plasma D5-phenylalanine (D5-Phe) as well as the reduction of plasma Phe levels.
Safety will be monitored by documentation of adverse events (AEs), clinical laboratory measurements, vital signs, and physical examinations.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label Study of the Efficacy and Safety of SYNB1618 in Patients With Phenylketonuria (SynPheny-1)|
|Actual Study Start Date :||August 25, 2020|
|Estimated Primary Completion Date :||August 2021|
|Estimated Study Completion Date :||August 2021|
Dose ramp of SYNB1618
15-day dose-ramp regimen (4 dose levels) of orally administered SYNB1618
- Changes from baseline in labeled Phe (D5-Phe) in plasma [ Time Frame: Day 14 ]The primary efficacy outcome will be assessed by measuring change from baseline in plasma D5-Phe AUC over 24 hours after D5-Phe administration
- Changes from baseline in fasting levels of plasma Phe [ Time Frame: Day 14 ]The secondary efficacy outcome will be assessed by measuring fasting levels of plasma Phe as compared to baseline
- Incidence of Treatment-Emergent Adverse Events [ Time Frame: Day 43 ]Will be measured by assessing nature and frequency of AEs
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04534842
|Contact: Kendall Davis||(919) 788-6519||JamesDavisKendall@prahs.com|
|Contact: Andrew Marsh||(617) 401-9975 ext email@example.com|
|United States, Missouri|
|Washington University in St Louis||Recruiting|
|Saint Louis, Missouri, United States, 63110|
|Contact: Jennifer Bruns 314-747-5366 firstname.lastname@example.org|
|Principal Investigator: Dorothy Grange, MD|
|United States, Utah|
|PRA Health Sciences||Recruiting|
|Salt Lake City, Utah, United States, 84124|
|Contact: Shawn Searle 801-904-4545 email@example.com|
|Principal Investigator: Shawn Searle, MD|