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A Phase 1/Phase 2 open-label study to evaluate the safety, tolerability, and efficacy of a single intravenous administration of an investigational medication in adult participants with phenylketonuria (PKU)-Tx Tester
Recruiting
Transgender
Phase
2/3
1 Location
Aperçu de l'étude
This study is looking for individuals 18 to 65 years old with PKU. Sanofi believes that everyone
should have the opportunity to take part in clinical trials. It is important to include people who
have been historically under-represented in clinical trials. Sanofi is committed to inclusivity in
our studies.
If you are interested in learning more about this study, we encourage you to complete a brief
questionnaire to help determine if you may qualify to participate and be referred to a site
recruiting in your area for further evaluation.
PKU is an inherited disorder caused by abnormalities in the gene coding for the phenylalanine
hydroxylase (PAH) enzyme, which increases the levels of Phenylalanine (Phe), a building block
of proteins in the blood. If PKU is not adequately treated, Phe can increase to harmful levels in
the body, causing injury to the brain and the nerves in your body. The usual treatment for
participants with PKU is strict dietary protein restriction along with manufactured medical foods
that are low in protein and Phe. A daily medication injection is also available.
The purpose of the study is to evaluate the safety and efficacy of an investigational gene
therapy in reducing phenylalanine (Phe) levels and in the elimination of a Phe restricted diet.
Détails de l'étude
The investigational gene therapy will insert a copy of the gene into your liver to stimulate your
liver to produce an enzyme (a protein that breaks down Phe) to lower blood Phe levels. This
enzyme converts the Phe present in your blood into other essential proteins that keep the brain
and nerves in your body healthy.
• This is an open label study (everyone on the study will receive the investigational gene
therapy) which is injected once (into the arm).
• Approximately 32 individuals will participate in this study.
• The study will last approximately 102 weeks (about 2 years). This includes
approximately 6 weeks for the screening period, 1 day for the treatment period and
approximately 96 weeks for the follow-up period.
1. Screening period (6 weeks): Various tests will be done to determine if you are
eligible for the study.
2. Treatment: The injection will be administered, and you will stay overnight for
observation for a minimum duration of 24 hours for monitoring.
3. Follow-up (96 weeks): Approximately 41 study visits, up to two times per week.
Some visits are at the study site and some visits can be performed as remote visits,
where study assessments can be done by a qualified in-home service provider at
your home.
Critère d'éligibilité
You may be eligible for this study if you meet the following criteria:
- Conditions: PKU, Phenylketonuria, gene therapy
-
Gender: Transgender
Inclusion Criteria:
Exclusion Criteria:
Updated on
23 Dec 2024.
Study ID: NCT215545
Pre-Screener
