An interventional study of DBS Phe monitoring once weekly and DBS Phe monitoring once monthly in Phenylketonuria (PKU), sponsored by Michał Kania. Recruiting at 1 site in Poland. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-17.
Sponsored by Michał Kania · Not applicable, Interventional, and Treatment
Recommendations regarding the frequency of phenylalanine level monitoring lack solid support from evidence derived from prospective randomized trials, including in the adult population with classic PKU. Current recommendations indicate that in adults (excluding the period of pregnancy planning and pregnancy itself), Phe levels should be assessed at least once a month or more frequently if additional indications exist. As part of this study, we plan to obtain, for the first time, high-quality data on the impact of Phe monitoring frequency on Phe control in adults. The primary objective of the study will be to assess the effect of the frequency (once a week versus once a month) of Phe level measurements on the metabolic control of phenylketonuria, as expressed by Phe concentration in DBS, in adult patients with the classic form of phenylketonuria.
191 studies on the registry are indexed under Phenylketonurias; 40 are open to participants now.
This study's planned enrollment of 36 is above the median of 27 across 122 interventional studies indexed under Phenylketonurias.
Browse Phenylketonurias studies →This is the only study on the registry with Michał Kania as lead sponsor.
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Clinical diagnosis of classic phenylketonuria (PKU) documented in the medical history by at least 2 measurements of blood phenylalanine concentration
≥600 μmol/l and a predicted PAH enzyme activity \<1% (GPV), requiring treatment with a low-protein diet supplemented with special low-phenylalanine amino acid mixtures
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.
Sequence 1 starts with intervention period 1 with DBS Phe measurements once a week for 24 weeks, followed by a 2-week wash-out period and intervention period 2 with DBS Phe measurements once a month for 24 weeks.
Other: DBS Phe monitoring once weekly · Other: DBS Phe monitoring once monthly
Sequence 2 starts with intervention period 1 with DBS Phe measurements once a month for 24 weeks, followed by a 2-week wash-out period and intervention period 2 with DBS Phe measurements once a week for 24 weeks.
Other: DBS Phe monitoring once weekly · Other: DBS Phe monitoring once monthly
In the intervention period participants will measure their Phe levels via DBS once a week as compared to once weekly.
In the control period participants will measure their Phe levels via DBS once monthly.
Change in Phe concentration in the DBS measurement
Change in Phe concentration in the DBS measurement from the baseline (O1) to the 5th and 6th months in each intervention period (average of measurements O140 and O168 \[measurements once a month\] OR O119 O168 \[measurements taken once a week\]).
Time frame: from the baseline (O1) to the 5th and 6th months in each intervention period (average of measurements O140 and O168 [measurements once a month] OR O119 O168 [measurements taken once a week]).
Plan to share: No — Sponsor requirement.
No publications or documents are linked to this record.
Started Sep 2026 (actual). Primary completion is estimated for Mar 2028, 18 months after the start.
Of 33 interventional studies with no phase under Phenylketonurias, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 9 and three quarters within 28. This study's planned 18 months is below that.
How these figures are worked out, and which studies are left out of them.
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