Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria (PKU)
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|ClinicalTrials.gov Identifier: NCT03516487|
Recruitment Status : Recruiting
First Posted : May 4, 2018
Last Update Posted : November 16, 2018
|Condition or disease||Intervention/treatment||Phase|
|Phenylketonuria Healthy||Drug: SYNB1618 Drug: Placebo||Phase 1 Phase 2|
This Phase 1/2a, first-in-human, oral single and multiple dose-escalation, randomized, double-blinded, placebo-controlled study of SYNB1618 in healthy adult volunteers and adult subjects with phenylketonuria will evaluate safety, tolerability, kinetics, and pharmacodynamics within the following 2 study parts:
Part 1 comprises a single-ascending dose (SAD) study conducted (3 treated:1 placebo) over 4 days in HV male and female subjects evaluated in up to 6 dose cohorts to identify maximum tolerated dose (MTD) within the single dose-dose range studied. Up to 24 healthy subjects may be enrolled in this part of the study. Following attainment of the MTD in HV, a SAD cohort of up to 4 subjects (male and female, >18 years old) previously diagnosed with PKU will be enrolled (3 treated:1 placebo).
Part 2 comprises a multiple-ascending dose (MAD) study conducted in an inpatient setting (6 treated:2 placebo) over 10 days in HV male and female subjects evaluated in up to 4 cohorts at doses that will not exceed the MTD from the SAD part of the study to identify the MTD of SYNB1618 within the multiple-dose range studied. Up to 32 healthy subjects may be enrolled in this part of the study. Once the highest MAD cohort and the SAD PKU cohort have been completed, a multiple-dose cohort of male and female subjects (>18 years old) previously diagnosed with PKU will be evaluated. Up to 20 subjects with PKU may be enrolled in the MAD PKU cohort (12 treated: 8 placebo).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Dose-escalating, Randomized, Double-blinded, Placebo-controlled|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 1/2a, First-in-human, Oral Single and Multiple Dose-Escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria to Evaluate Safety, Tolerability, Kinetics, and Pharmacodynamics|
|Actual Study Start Date :||April 2, 2018|
|Estimated Primary Completion Date :||August 2019|
|Estimated Study Completion Date :||September 2019|
A single or multiple oral dose of SYNB1618 in a chilled buffered solution
A single (Part 1) or multiple (Part 2) ascending oral dose of SYNB1618 in a buffered solution. In Part 1, subjects receive one dose of SYNB1618 on Day 1. In Part 2, subjects receive SYNB1618 TID for 7 days.
Placebo Comparator: Placebo
A single or multiple oral dose of placebo (100mL of a chilled buffer solution)
A single (Part 1) or multiple (Part 2) oral dose of a buffered 100mL solution. In Part 1, subjects receive one dose of buffered solution on Day 1. In Part 2, subjects receive a buffered solution TID for 7 days.
- Incidence of treatment-emergent Adverse Events [ Time Frame: Up to 90 days following enrollment. ]Will be measured by assessing nature and frequency of Adverse Events.
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): NCT03516487
|Contact: Kendall Davis||919-788-6519||JamesDavisKendall@prahs.com|
|United States, Massachusetts|
|Boston Children's Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Vera Anastasoaie 617-355-7346 Vera.firstname.lastname@example.org|
|Principal Investigator: Stephanie Sacharow, MD|
|United States, Utah|
|PRA Health Sciences||Recruiting|
|Salt Lake City, Utah, United States, 84124|
|Contact: Christian Ventrello 801-904-4662 VentrelloChristian@prahs.com|
|Principal Investigator: Shawn Searle, MD|