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15 Phenylketonuria Trials
Power is an online platform that helps thousands of Phenylketonuria patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Gene Therapy for Phenylketonuria
Cleveland, OhioThis is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria (PKU) on a chronic, stable diet. The purpose of the study is to evaluate the safety and efficacy of SAR444836 in reducing phenylalanine (Phe) levels and in the elimination of a Phe restricted diet.
Participants will receive a one-time intravenous (IV) administration of SAR444836.
The study is constituted of 2 separate parts: a dose escalation part, and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part. In both study parts, clinical and laboratory assessments will be collected to: a) assess the incidence of adverse events, and b) evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet.
The study duration will be approximately 102 weeks (approximately 2 years) for each participant and includes a 6-week screening phase and 96-week follow-up period after SAR444836 administration.
There will be a total of 41 study visits. Many study visits may occur as remote visits and be performed by a qualified in-home service provider.
Actual study duration for an individual participant may be longer than 102 weeks due to the administration of SAR444836 to participants in Stage 1A in a serial fashion, or other factors such as delays in scheduling study visits.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Liver Disease, HIV, Hepatitis B, Others
32 Participants Needed
PTC923 for Phenylketonuria
Pittsburgh, PennsylvaniaThis trial is testing the safety of a medication called PTC923 in people with phenylketonuria (PKU). PKU patients need to manage their blood phenylalanine levels carefully. PTC923 aims to help control these levels, making it easier for patients to manage their condition. PTC923 is a newer treatment for PKU, following earlier treatments like sapropterin and pegvaliase.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Renal Impairment, Neuropsychiatric Illness, Others
Must Not Be Taking:Folate Inhibitors, BH4 Supplements
200 Participants Needed
JNT-517 for Phenylketonuria
Pittsburgh, PennsylvaniaThis trial tests a new oral medication, JNT-517, for safety and tolerance. It involves healthy participants and people with phenylketonuria (PKU). Researchers aim to see if JNT-517 can safely reduce certain amino acids in people with PKU.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65
Sex:All
112 Participants Needed
Pegvaliase for Phenylketonuria
Lexington, KentuckyThis trial is testing pegvaliase injections in adolescents with PKU. The goal is to see if these injections are safe and effective. Pegvaliase helps the body break down a harmful substance that people with PKU cannot process on their own. Pegvaliase is a newer treatment for PKU, following earlier treatments like dietary restriction and sapropterin.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12 - 17
Sex:All
55 Participants Needed
Sepiapterin for Phenylketonuria
Indianapolis, IndianaThe main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:< 12
Sex:All
Key Eligibility Criteria
Disqualifiers:Neuropsychiatric Illness, Renal Disease, Others
Must Not Be Taking:BH4 Supplementation
56 Participants Needed
L-Phenylalanine for Phenylketonuria Carriers
Guelph, OntarioThis is a clinical intervention study of PKU carriers (cases) and non-carriers (controls). Upon completing the informed consent process, participants will complete baseline measures of chronic mental health prior to the intervention (PHQ-9, GAD-7, BIS-11). Participants will attend the Human Nutraceutical Research Unit (HNRU) at the University of Guelph, fasted, and first undergo baseline measures of cognition and acute mental health (mood) and provide samples or saliva, urine and dried blood spots to evaluate phenylalanine (Phe), tyrosine (Tyr) and their metabolites (PAH pathway functioning) as well as for genetic testing of the PAH gene. Participants will also complete a brief questionnaire which will include age, sex, ethnicity, income, weight and height (measured using a stadiometer and calibrated weigh scale), and confirmation that participants arrived to the lab fasted (i.e. have only had water to drink and no other foods/ beverages prior to analyses). Blood pressure and heart rate will also be measured at baseline.
Following baseline tests, participants will consume a pure L-Phe supplement dosed at 100 mg/kg mixed with 125 mL of water and 125mL of orange juice. Blood pressure and heart rate will be repeated at 1-hour post-L-Phe consumption. Two-hours postprandial, participants will repeat the cognitive tests and acute mental health (mood) assessment, blood pressure and heart rate measurement and provide follow-up saliva, urine and dried blood spot samples. Participants will also be asked to report any side effects they experienced with the L-Phe consumption.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:PKU, Neurodegenerative Conditions, Hypertension, Others
Must Not Be Taking:Monoamine Oxidase Inhibitors
50 Participants Needed
Rapid Drug Desensitization for Allergic Reactions
Chevy Chase, MarylandThe purpose of this study is to determine if rapid drug desensitization (RDD) to Palynziq will improve drug tolerability and treatment persistence in adult patients on commercial Palynziq experiencing hypersensitivity reactions (HSRs) leading to treatment interruption or reduction of dose or dosing frequency. See Section 10.8 for full list of HSR preferred terms. Study details include:
* Study duration: Up to 30 weeks (up to 6 weeks for Screening, then RDD, and 24 weeks of follow-up)
* RDD duration: 1 day
* Palynziq dosing/follow-up duration: 24 weeks
* Palynziq dosing frequency: Individualized
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Asthma, Cardiovascular Disease, Others
Must Be Taking:Palynziq
10 Participants Needed
SAB-176 for Flu
Bethesda, MarylandThis study will evaluate the safety and tolerability of an intramuscular injection of SAB-176 intended for use as a pre/post prophylactic for influenza.
This is a Phase 1, randomized, double-blind, placebo-controlled clinical trial in which a total of 28 subjects will receive an injection of either SAB-176 or placebo (normal saline). The investigational product will be administered intramuscularly (IM) on Day 1. Four dose escalation cohorts of 7 subjects (5 active and 2 placebo) each are planned. Subjects will be randomized to receive either SAB-176 or placebo in a double-blinded manner.
Progression to subsequent dose-escalating cohorts will be dependent on safety measured through Day 5 after dosing of the previous cohort.
Blood specimens will be collected at prescribed intervals to examine pharmacokinetics and immunogenicity. Safety will be actively monitored during investigational product administration and for 60 days following dosing. The decision to advance to the next cohort will be based solely on the safety assessment through Day 5. All safety data will be summarized and reviewed by the PI, the Sponsor's Clinical Monitor, and the Research Monitor prior to next cohort dose-escalation.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:18 - 60
Sex:All
28 Participants Needed
Gene Therapy for Phenylketonuria
Atlanta, GeorgiaThis is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 55
Sex:All
Key Eligibility Criteria
Disqualifiers:Non-PAH PKU, Liver Disease, Others
Must Not Be Taking:Kuvan, Palynziq
12 Participants Needed
Metabolic Camp for Pregnant Women with Phenylketonuria
Atlanta, GeorgiaThe purpose of this project is to study the effectiveness of teaching teens and young women with Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD) about their disease and nutrition related issues in a camp environment. It will also look at pregnancy outcome results in women with PKU who attended Metabolic Camp and compare their results to other women with PKU who have not attended the Metabolic Camp.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:11+
Sex:Female
Key Eligibility Criteria
Disqualifiers:Not Female, Under 11, Others
200 Participants Needed
Gene Therapy for Phenylketonuria
Morristown, New JerseyThis is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:15+
Sex:All
100 Participants Needed
L-Phenylalanine for Gut Health
New York, New YorkThe purpose of this study is to explore how the dietary supplement L-Phenylalanine affects the production of the metabolite phenylpropionic acid (PPA) and changes fungal populations of the gut microbiome.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18+
Sex:All
20 Participants Needed
Coping Skills Intervention for Phenylketonuria
Columbia, MissouriWhile previous PKU intervention research has largely focused on pharmacological treatment of elevated Phe levels, the adaptation of evidence-based psychosocial therapy holds promise for addressing cognitive and psychological symptoms and significantly improving quality of life. Study goals include: (1) to demonstrate the efficacy and feasibility of a short-term skills-based intervention (Show Me FIRST) for anxiety and depression in adolescents with PKU, and (2) to further establish the psychometric validity of novel assessment measures recently developed by the Investigators that utilize ecological momentary assessment (EMA) to capture "in the moment" neurocognitive and psychological function. The Investigators propose to conduct a randomized controlled trial wherein 30 adolescents with PKU will be randomized to either an immediate intervention group or wait-list/delayed intervention group.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:10 - 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Intellectual Disability, Suicidal Ideation, Others
30 Participants Needed
Theophylline for Pseudohypoparathyroidism
Boston, MassachusettsThis trial tests a medication in patients with a condition where the body doesn't respond well to hormones to help their bodies respond better.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:5 - 21
Sex:All
Key Eligibility Criteria
Disqualifiers:Kidney Disease, Heart Disease, Seizure, Others
Must Not Be Taking:Chronic Theophylline Interactors
100 Participants Needed
This is a Phase I, Single-Center, Open-Label study evaluating the safety and pharmacokinetics of single doses of ZID-FEP and metronidazole alone or in combination utilizing a 3-period, crossover study design. Thirty eligible male and female healthy adult subjects will participate in the study and receive single doses of (1) ZID-FEP 3g IV (ZID 1 g plus FEP 2 g) administered over 1 hour (h); (2) metronidazole 0.5 g IV alone administered over 1 h; and (3) metronidazole 0.5 g IV over 1 h, followed by ZID-FEP 3g IV over 1 h over 3 treatment periods separated by a 48 h washout period.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 45
Sex:All
Key Eligibility Criteria
Disqualifiers:Cardiovascular, CNS, Respiratory, Others
Must Not Be Taking:NSAIDs, Sucralfate, Others
30 Participants Needed
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Frequently Asked Questions
How much do Phenylketonuria clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Phenylketonuria clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Phenylketonuria trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Phenylketonuria is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Phenylketonuria medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Phenylketonuria clinical trials ?
Most recently, we added A Single-center, Open-label, Study Evaluating Safety and Pharmacokinetics of Single Doses of Zidebactam-Cefepime and Metronidazole Alone or in Combination., Gene Therapy for Phenylketonuria and Rapid Drug Desensitization for Allergic Reactions to the Power online platform.