A Study of JNT-517 in Participants With Phenylketonuria (PKU)
ConditionPhenylketonuria
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.
About this trial
The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days.
Participants will:
Take 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Males and females ≥18 years of age on Day 1
Clinical diagnosis of PKU
Average of at least 3 plasma Phe levels (after >4-hour fast) during Screening period of ≥360 μmol/L
Not on pegvaliase within 4 weeks prior to Screening
Disqualifiers
Exclusion Criteria
Any acute or uncontrolled chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study
Positive for hepatitis B or C or human immunodeficiency virus
Any history of malignancy of any organ system (other than non-melanoma skin cancer or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases
Trial design
Design model
Parallel
Treatments tested in this trial
JNT-517 Tablet
DrugJNT-517: 75 mg BID
JNT-517 Tablet
DrugJNT-517: 150 mg BID
Placebo Tablet: BID
DrugPlacebo Tablet: BID
Treatment groups
120 Participants
are divided into 3 treatment groupsGroup A: Drug: JNT-517 - 150 mg BID (Tablet)Experimental treatment 1 intervention
Group B: Drug: JNT-517 - 75 mg BID (Tablet)Experimental treatment 1 intervention
Group C: Placebo - BIDPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Absolute Change in Plasma Phenylalanine (Phe) From Baseline to the Mean of Weeks 2, 4, and 6 in the JNT-517 150 mg BID Dose Group at End of Period 1
Time frame
Baseline to End of Period 1 (Week 6)Secondary outcomes
1
Percent Change in Plasma Phe From Baseline to the Mean of Weeks 2, 4, and 6 in the JNT-517 150 mg BID Dose Group at End of Period 1
Time frame
Baseline to End of Period 1 (Week 6)2
Percentage of Participants Achieving Plasma Phe <600 Micromoles per Liter (µmol/L) at End of Period 1 Among Participants With Baseline ≥600 µmol/L in the 150 mg BID and 75 mg BID Groups
Time frame
Baseline to End of Period 1 (Week 6)3
Percentage of Participants Achieving Plasma Phe <360 µmol/L at End of Period 1 in the 150 mg BID and 75 mg BID Groups
Time frame
Baseline to End of Period 1 (Week 6)4
Absolute Change in Plasma Phe From Baseline to the Mean of Weeks 2, 4, and 6 in the JNT-517 75 mg BID Group at End of Period 1
Time frame
Baseline to End of Period 1 (Week 6)Sponsors and contacts
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Source ClinicalTrials.gov