Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

Summary

This clinical trial study aims to Evaluate whether iron supplementation for 3 months may help reduce Phe blood concentration in PKU patients by enhancing the hydroxylation of Phe to tyrosine (Tyr).

84 patients affected by PKU with low Phe-controlled diet therapy (aged 3-25 years, both genders, 35 females and 49 males), were randomly enrolled in this 3 years study using Randomized Block Design. Patients were divided in two different groups: group (A) was treated with low Phe diet therapy and iron supplementation while group (B) was treated only with low Phe diet therapy. The duration of iron supplementation was 3 consecutive months for each patient in group (A). During this study, 3 hematological and clinical controls were performed at T0 (the enrolment day), T3 (3 months from T0), and T6 (6 months from T0) another TG was done at T1, T2, T4, and T5. During the clinical control blood samples for phenylalanine, and tyrosine were performed on all patients.

A computer-generated block sequence balanced randomly assigned subjects to two different groups of 42 patients. The investigator who generated the randomization sequence was independent of the research staff. Energy calculation and diet preparation are according to the "Reference intake levels of nutrients and energy for the Italian population (LARN)" and RDA (USA- Recommended Daily Allowance). The patients were informed through the consent form about all study details. The researcher clarified that the iron supplementation was provided to patients for the purpose of studying "Phe" levels, oxidative stress, and inflammatory reactions, and not for treating Anemia before enrolling in this study.

The amount of supplementary iron was decided for patients according to the daily requirement (8- 18 mg / day), according to LARN 2012 and (RDAs) for Iron.

The number of patients in group A (42 patients) that consumed iron of 1 sac (14 mg/day) was 26 patients (61.9 %), of them 18 males and 8 females, in comparison with patients that consumption 1 sac an alternative day (14mg/every 2 days) = (7 mg/day- ½ sac/ day) was 16 patients (38.1%) of them 9 males and 7 females.

Conclusions and Relevance:

* Our data suggests that regular iron supplementation could be needed to obtain better metabolic control in PKU patients, as it seems to be directly linked to lowering blood Phe levels. Thus, support that iron supplementation to PKU patients plays a relevant role for accurate function of PAH enzyme, probably represents one activator and it may can help to decrease the b-Phe value near to the normal range with - 46.7% of Phe, - 74.52% of Phe/Tyr ratio and increase 91.07% Tyr all with (P\<00.1) from the basal values
* It is also confirmed that oral iron supplementation with FERALGINE (FB + SA) to these patients could represent "as part of a comprehensive PKU management strategy" considering the high effectiveness and tolerability profile.
* additional study is required before the information should be used support our results The investigators declare that no conflict of interest.

Conditions

• Phenylketonuria (PKU)

Interventions

DIETARY_SUPPLEMENT

iron supplementation

The amount of supplementary iron was decided for patients according to the daily requirement (8- 18 mg / day), according to LARN 2012 and (RDAs) for Iron. (25) . This study took in consideration also, the recommendation of the National Institutes of Health iron dietary supplement fact of sheet as reference to calculate iron dose for our candidates because this dose isn't against LARN and more practical to applicate. The minimum RDA values is applied, which coincides within the LARN daily requirement limits. (7 mg-18 mg of iron per day). Applying the minimum recommended RDA dose, that can satisfy the daily iron requirement by age, approximating it to be adequate for the clinical dose administered orally once a day (e.g. 1.98 g ==\> 2 g, 1.53 g ==\> 1.5 g

DIETARY_SUPPLEMENT

control group

Non intervension: Only restricted low and controlled Phe diet therapy

Sponsors & Collaborators

• Università Luigi Vanvitelli della Campania

  collaborator UNKNOWN

• ASST Santi Paolo e Carlo

  lead OTHER

Principal Investigators

• Raed Selmi, MD-PhD · ASST Santi Paolo e Carlo

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

3 Years

Max Age

25 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-01-07

Primary Completion

2023-01-09

Completion

2023-04-03

Countries

• Italy

Study Locations