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Active clinical trials for "Phenylketonurias"

Results 31-40 of 147

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

Phenylketonuria

The purpose of this study is to determine whether tetrahydrobiopterin (BH4)is effective in treating patients with PKU.

Terminated8 enrollment criteria

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

Phenylketonurias

This study will evaluate the safety and tolerability of RTX-134 in adult patients with PKU.

Terminated16 enrollment criteria

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

PhenylketonuriasPAH Deficiency1 more

This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

Terminated13 enrollment criteria

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance...

Phenylalanine Hydroxylase Deficiencies

The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive to BH4. The primary objective is to assess the effect of BH4 on phenylalanine tolerance compared to placebo under optimal blood phenylalanine control and to demonstrate safety in 12 months long-term treatment. Additionally population PK will be assessed.

Terminated13 enrollment criteria

Gene Therapy Clinical Study in Adult PKU

PhenylketonuriasPAH Deficiency

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

Terminated18 enrollment criteria

Liver Cell Transplant for Phenylketonuria

Phenylketonuria

Human phenylketonuria (PKU) results from phenylalanine hydroxylase (PAH) deficiency, and represents one of the most common and extensively studied single-gene Mendelian disorders in humans. Unfortunately, optimum clinical outcome demands lifelong dietary restriction through adherence to an unpalatable and expensive artificial diet. Challenges in maintaining traditional therapy lead to increasing phenylalanine (Phe) levels in patients as they approach adulthood with an incumbent severe burden of psychosocial and intellectual difficulties. The recent introduction of the new medication Sapropterin for treatment of PKU has improved Phe control and dietary tolerance in some patients, but at enormous cost to patients and insurers for the FDA designated orphan product. Thus, there is an unmet need for novel therapies to correct PKU. PAH is almost exclusively expressed in the liver in humans. The main objective of the current proposal is to examine the safety and efficacy of hepatocyte transplantation in patients with PKU.

Terminated17 enrollment criteria

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Phenylketonuria

This is a Phase 2a multicenter, double-blind, placebo-controlled, randomized, 2-arm pilot trial designed to assess the effect of sapropterin on cognitive abilities in young adults with Phenylketonuria (PKU) over a 26-week treatment period.

Terminated18 enrollment criteria

Effects of Kuvan on Melatonin Secretion

Phenylketonuria (PKU)

This study examines the effect of tetrahydrobiopterin (Kuvan) and Large Neutral Amino Acid (LNAA) therapy on melatonin and dopamine levels in individuals with Phenylketonuria (PKU). The investigators hypothesize that Kuvan therapy will improve melatonin secretion and urine dopamine levels to some extent. However significantly greater responses in melatonin and dopamine secretions may be observed with combined treatment with Kuvan and supplementation of LNAA.

Completed5 enrollment criteria

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted...

Phenylketonurias

The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.

Completed14 enrollment criteria

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With...

Phenylketonuria (PKU)

This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570). The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses > 40 mg/day to 60 mg/day.

Completed15 enrollment criteria
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