Metabolic Camp for Pregnant Women with Phenylketonuria
This trial will study whether teaching teens and young women with Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD) about their disease and nutrition in a camp environment improves pregnancy outcomes.
Tyrosine Kinase Inhibitor
Ponatinib for Chronic Myeloid Leukemia
This trial tests ponatinib hydrochloride as second line therapy for CML patients who didn't respond to initial treatment with imatinib mesylate, dasatinib, or nilotinib, or can't tolerate those treatments. Ponatinib hydrochloride may work by blocking a protein needed for cell growth.
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Trials for Acute Lymphoblastic Leukemia Patients
PARP Inhibitor
Veliparib + Topotecan ± Carboplatin for Leukemia
This trial is studying the side effects and best dose of veliparib when given with topotecan hydrochloride and carboplatin in treating patients with relapsed or refractory acute leukemia, high-risk myelodysplasia, or aggressive myeloproliferative disorders.
Trials for T-Cell Lymphoblastic Leukemia Patients
PARP Inhibitor
Veliparib + Topotecan ± Carboplatin for Leukemia
This trial is studying the side effects and best dose of veliparib when given with topotecan hydrochloride and carboplatin in treating patients with relapsed or refractory acute leukemia, high-risk myelodysplasia, or aggressive myeloproliferative disorders.
Trials for Philadelphia Chromosome Positive Patients
Monoclonal Antibodies
Blinatumomab + Chemotherapy/Dasatinib for Acute Lymphoblastic Leukemia
This trial is studying the side effects and efficacy of blinatumomab and combination chemotherapy or dasatinib and prednisone in treating older patients with acute lymphoblastic leukemia.
Monoclonal Antibodies
Immunotherapy + Chemotherapy for Acute Lymphoblastic Leukemia
This trial tests a new treatment combining inotuzumab ozogamicin and chemotherapy for elderly patients with acute lymphoblastic leukemia. The drug targets and kills cancer cells, aiming to improve outcomes for those who can't undergo intensive therapy or have had a recurrence. The study will determine the appropriate dose and evaluate the treatment's effectiveness and side effects.
Trials for BCR-ABL1 Positive Patients
Tyrosine Kinase Inhibitor
ABL001 Combo Therapy for Leukemia
This trial is testing ABL001 in combination with dasatinib and prednisone as a possible treatment for B-cell Acute Lymphoblastic Leukemia (BCR-ABL+ B-ALL) or Chronic Myeloid Leukemia (CML) in lymphoid blast crisis.
Tyrosine Kinase Inhibitor
Asciminib vs Other TKIs for Chronic Myeloid Leukemia
This trial is testing a new drug called asciminib against other existing treatments for adults newly diagnosed with a specific type of leukemia. The goal is to see if asciminib is more effective at blocking a protein that helps cancer cells grow. Asciminib has shown superior efficacy in patients with chronic myeloid leukemia (CML) previously treated with multiple other treatments.
Phase 3 Trials
Enzyme substitution therapy
Pegvaliase for Phenylketonuria
This 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.
PTC923 for Phenylketonuria
This 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.
Trials With No Placebo
Behavioral Intervention
Coping Skills Intervention for Phenylketonuria
"This trial aims to study the effectiveness of a short-term psychosocial therapy called Show Me FIRST for treating anxiety and depression in adolescents with PKU. The researchers also want to validate new assessment measures that
Enzyme substitution therapy
Pegvaliase for Phenylketonuria
This 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.
PTC923 for Phenylketonuria
This 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.
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Frequently Asked Questions
Introduction to Phenylketonuria
What are the top hospitals conducting Phenylketonuria research?
When it comes to advancing the understanding and treatment of Phenylketonuria (PKU), several hospitals are making significant strides. UPMC Children's Hospital in Pittsburgh, known for its commitment to pediatric care, is actively conducting three clinical trials focused on PKU. They have a total of nine past trials dedicated to this rare genetic disorder that affects the body's ability to process an amino acid called phenylalanine. The hospital embarked on its first PKU trial in 2011, aiming to improve the lives of those living with this condition.
Moving southward to Tampa, Florida, we find the University of South Florida taking part in two ongoing PKU trials and having completed ten previous studies related to this metabolic disorder. Since their initial venture into PKU research back in 2008, they have been working diligently towards finding innovative approaches for managing and treating the symptoms associated with this challenging condition.
In Boston, another leading medical institution championing research and treatments is Boston Children's Hospital. With two active clinical trials focusing on PKU and a remarkable seventeen past studies dedicated to improving outcomes for those affected by this inherited disorder, they continue pushing boundaries since embarking on their initial investigation into PKU management strategies in 2013.
Even smaller healthcare providers recognize the importance of contributing to advancements in understanding and treating rare diseases like Phenylketonuria. The Community Health Clinic located in Topeka has joined efforts by conducting two current PKU trials following their recent entry into researching these novel therapies starting from 2022 itself.
Lastly yet importantly,Rare Disease Research situated within Atlanta proves how crucial every contribution can be as they conduct one active trial specifically addressing Phenylketonuria; despite being new entrants themselves only recording their first-ever experiment concerning such conditions just last year-2022.They relentlessly strive towards better solutions which hold potential life-altering impacts for individuals grappling with uncommon disorders worldwide
Which are the best cities for Phenylketonuria clinical trials?
Cities that offer notable opportunities for Phenylketonuria clinical trials include Pittsburgh, Pennsylvania; Indianapolis, Indiana; Gainesville, Florida; Aurora, Colorado; and Houston, Texas. These cities collectively host a range of active trials focused on studying medications such as JNT-517 Suspension Then Tablet Fasted Then Tablet Fed (Part C), Pegvaliase, HMI-103, PTC923, and others. With four active trials in both Pittsburgh and Indianapolis and three each in Gainesville, Aurora, and Houston respectively - individuals with Phenylketonuria can find potential treatment options through participation in these studies conducted across these locations.
Which are the top treatments for Phenylketonuria being explored in clinical trials?
Phenylketonuria, a rare genetic disorder, is the subject of ongoing clinical trials exploring potential treatments. Pegvaliase stands out as one of the top contenders with its current active trial and three all-time Phenylketonuria trials since its listing in 2018. Another promising treatment on the horizon is HMI-103, currently being tested in an active trial after its recent introduction to the scene in 2022. Additionally, PTC923 has shown promise with an active trial underway and two all-time Phenylketonuria trials since its first listing in 2021. These clinical trials offer hope for patients afflicted by this challenging condition and may pave the way for advancements in Phenylketonuria treatment options.
What are the most recent clinical trials for Phenylketonuria?
Recent clinical trials have brought about promising developments in the field of Phenylketonuria (PKU) research, offering potential advancements in treatment options. One such trial focuses on JNT-517 Suspension and its efficacy when administered orally to patients with PKU under different fasting conditions. Another study explores the use of Pegvaliase, a novel drug that aims to reduce phenylalanine levels in individuals with PKU. Additionally, HMI-103 is being investigated for its potential as a therapeutic intervention for this metabolic disorder. Lastly, PTC923 is undergoing Phase 3 trials to evaluate its effectiveness in managing PKU symptoms. With these significant strides forward, there is hope for improved care and outcomes for individuals living with PKU.
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What Phenylketonuria clinical trials were recently completed?
Recent advancements in the field of Phenylketonuria (PKU) treatment have been marked by the completion of two notable clinical trials. In September 2021, PTC Therapeutics successfully concluded their trial exploring the potential of PTC923 as a therapeutic option for PKU patients. Additionally, Synlogic wrapped up their SYNB1618 trial in August 2020. These significant milestones highlight ongoing efforts to advance and improve treatments for individuals living with PKU, offering hope for better management and outcomes for these patients.