An observational study in Phenylketonuria (PKU) and Hyperphenylalaninemia, sponsored by Aptatek BioSciences, Inc. Recruiting at 2 sites in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2026-08-25.
Sponsored by Aptatek BioSciences, Inc · Observational
Among 93 studies in Phenylketonurias with a site in United States — 16 open to participants now, 21 under way, 4 of them phase 3. Phenylketonurias · United States.
Among the 93 studies in Phenylketonurias with a site in United States, this one is the 9th largest by planned enrollment of the 29 observational ones that give a count, is one of 5 that started in 2026, is one of 29 observational ones, reaches primary completion later than 6 of the 20 other live ones, and was the 91st registered.
191 studies on the registry are indexed under Phenylketonurias; 40 are open to participants now.
Browse Phenylketonurias studies →16 of the 93 studies in Phenylketonurias with a site in United States are open now. Recruiting first, the newest first:
Otsuka Pharmaceutical Development & Commercialization, Inc. · Sites in United States
Ages 18–65 · Healthy volunteers
Maze Therapeutics · Sites in United States, France, United Kingdom
Ages 18–75
BioMarin Pharmaceutical · Sites in United States
Ages 18–65
Agios Pharmaceuticals, Inc. · Sites in United States, Poland
Ages 18–69
University of Missouri-Columbia · Sites in United States
Ages 18–60 · Healthy volunteers
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 31 Phenylketonurias studies recruiting now, 14 (45%) set no upper age limit and 17 (55%) stop at 75 or younger; 15 take children under 18; 8 take healthy volunteers.
This study: 1 Year and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
93 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 16 · Phase 2 21 · Phase 3 16 · Phase 4 4
First in 1984; this study’s year is marked. 5 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 60 is above the median of 37 across 61 observational studies indexed under Phenylketonurias.
Median enrollment 33 across the 63 interventional studies, 28 across the 29 observational. Of the 70 that completed or were terminated, 28 (40%) have results posted.
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54 of 93 have an industry lead sponsor. The 10 most frequent:
This is the only study on the registry with Aptatek BioSciences, Inc as lead sponsor.
Aptatek BioSciences, Inc has 1 study filed under Phenylketonurias — fewer than ten, so there is no comparison to draw.
6 of the studies in Phenylketonurias with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06628128Phase 3Otsuka Pharmaceutical Development & Commercialization, Inc.A Long-Term Study of JNT-517 in Participants With Phenylketonuria | 2027-11-01 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT07713758Otsuka Pharmaceutical Development & Commercialization, Inc.A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function | 2027-02-09 |
| NCT07220265University of Missouri-ColumbiaImpact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers | 2027-03-01 |
| NCT07551921Aptatek BioSciences, IncPheCheck™ Validation Study | 2027-03-20 |
Add all 6 to your calendar (.ics) → With an account.
Among the 93 studies in Phenylketonurias with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The goal of this observational study is to evaluate the performance of the PheCheck™ test for the rapid detection of phenylalanine (Phe) from a fingerstick sample, in patients 1 year or older with phenylketonuria (PKU) or hyperphenylalaninemia. The main objective is comparing the concentration of phenylalanine (Phe) in capillary blood with the reference method (DBS).
The primary objective of this study is to evaluate the performance of the PheCheck™ test for the rapid, quantitative detection of phenylalanine (Phe) direct from a capillary (fingerstick) sample. Results will be compared to reference testing from Dried Blood Spot Cards (DBS).
The PheCheck™ test performs a quantitative determination of phenylalanine in a self-collected or parent/guardian-collected capillary blood sample.
The analysis will evaluate the performance of the analyte concentration of phenylalanine in prospectively collected samples self-collected and tested by patients with PKU, measured by the PheCheck™ test as compared to the standard of care DBS testing performed by a reference lab
Patients 1 year or older with phenylketonuria (PKU) or hyperphenylalaninemia
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Device: PheCheck Test Description: Quantitative, single-use, rapid test Intended for home-based self-testing and monitoring of PKU. 1 fingerstick specimen collected at each study visit Comparator: Dried Blood Spot Cards. 1 fingerstick specimen collected at each study visit
Device: PheCheck · Diagnostic Test: Dried Blood Spot Cards.
Quantitative, single-use, rapid test Intended for home-based self-testing and monitoring of PKU. 1 fingerstick specimen collected at each study visit Sample read using an electronic reader. Results available within 1 hour
Laboratory-based reference method 1 fingerstick specimen at each study visit to collect 5 samples to fill 5 spots on the card. Card sent to a laboratory, results available within 72 hours
Performance of the PheCheck™ test for the detection of phenylalanine (Phe) concentration from capillary blood as compared to the reference method (DBS)
The primary objective of this study is to evaluate the performance of the PheCheck™ (Aptatek Biosciences) test for the rapid, quantitative detection of phenylalanine (Phe) direct from a capillary (fingerstick) sample. PheCheck™ is a self-test to be performed by PKU patients. Results will be compared to reference testing from Dried Blood Spot Cards (DBS) tested in a designated reference laboratory. Phe results will be blinded to the study participant
Time frame: Day 1
Started Aug 2026 (actual). Primary completion is estimated for Mar 2027, 7 months after the start.
Of 22 observational studies under Phenylketonurias, half reached primary completion within 45 months of starting (the median), counting those still running; a quarter within 17 and three quarters within 95. This study's planned 7 months is below that.
How these figures are worked out, and which studies are left out of them.
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