Loading clinical trials...
Loading clinical trials...
Showing 1-20 of 62 trials
NCT06628128
The goal of this Phase 3, open-label extension study is to evaluate the long-term safety and efficacy of JNT-517 in participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 or JNT517-201. In this long-term extension (LTE) study, all adults (aged ≥18 years) who complete Study JNT517-101 will be randomized 1:1 to receive JNT-517 at 75 mg twice daily (BID) or 150 mg BID, regardless of their previous dose. Adolescent participants who complete Study JNT517-201 will receive the same JNT-517 dose of the cohort they were initially assigned to, either 75 mg BID or 150 mg BID.
NCT05579548
This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.
NCT07241234
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
NCT05051657
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.
NCT04595656
Brief Summary: Mortality data are important in low and middle-income countries to assess the population health status and trends. Mortality statistics in the populations of Bangladesh, Myanmar and Lao PDR are considered as generally limited and a recent assessment of vital registration systems of those countries reported a poor performance of the death registration system. Majority of deaths occur at home in rural area of those countries without cause of death assigned. To address this problem, the investigators will use verbal autopsy (VA) method developed by WHO to ascertain the cause of a death based on an interview with a family member or caregiver of deceased person to obtain information about causes of death in study rural communities. Death related to a specific cause will be defined by ICD-10 codes and described as primary, secondary or underlying COD. The study is planned to conduct in approximately 390 rural village communities covered by Southeast Asia clinical trial network (SEACTN) network in proposed 3 countries. The study findings will help establish a better understanding in signs, symptoms, medical history and circumstances preceding death; prioritize future interventions for early and effective diagnostics and treatment for diseases and how to keep mortality surveillance ongoing in study rural settings. This study is funded by Welcome Trust. Welcome Trust grant reference number is \[215604/Z/19/Z\].
NCT07090213
This prospective, multicentre observational study translates the 13-item Pelvi-Perineal Surgery Sexuality Questionnaire (PPSSQ) from French to Turkish, culturally adapts it, and evaluates its reliability, validity and responsiveness in Turkish-speaking women covering from pelvic floor surgery.
NCT07318168
This randomized pilot trial tests a brief psycho-educational group program designed to enhance maternal reflective functioning (RF)-the capacity to understand one's own and the baby's behavior in terms of thoughts and feelings-among first-time mothers in the early postpartum. Primiparous women aged ≥18 years, 1-5 months postpartum, were randomized 1:1 to the intervention or a wait-list control. The intervention consists of four weekly 90-minute Zoom sessions with short home practices. The primary outcome is interview-based RF assessed with the FMSS-RF (blind-coded) at post-intervention. Exploratory outcomes include self-reported RF (PRFQ-CMS), depressive symptoms (EPDS), emotion-regulation (ERQ-Suppression), and feasibility/acceptability indices (recruitment, retention, attendance, satisfaction). This single-site study is conducted at the University of Haifa (Faculty of Education). If effective, the low-intensity, telehealth format may offer a scalable postpartum support option for first-time mothers.
NCT06560736
While 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.
NCT06332807
This 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.
NCT05270837
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
NCT06682416
This study investigates how the temperature of local anesthetics affects maternal fever related to epidural analgesia during childbirth. The research is a prospective, randomized controlled trial involving 424 participants from two hospitals. The primary objective is to investigate the impact of local anesthetic solution temperature on intrapartum fever in parturients. Secondary goals include assessing the impact on the efficacy of epidural analgesia and various maternal and neonatal outcomes. Participants will receive either 37°C or 23°C anesthetic solutions, and data will be collected on fever rates, pain scores, and other health indicators. The study runs from January 2024 to December 2026.
NCT05678699
The goal of this study is to learn about the effects of structural inventions, such as equity-focused Medicaid polices, on severe maternal morbidity (SMM) and mortality and maternal health. The main questions it aims to answer are: 1. What is the effect of Medicaid healthcare quality interventions on SMM? 2. What is the effect of Medicaid healthcare quality interventions + doula care? 3. What are Medicaid beneficiaries' experiences in receiving services and the potential impact of integration of doula services and equity practices? Participants will be asked to describe experiences as a result of structural interventions and focused Medicaid policies.
NCT06965647
This randomized controlled study aims to evaluate the effect of fetal heart sound (FHS) measurement performed by the partner on prenatal maternal and paternal expectations and attachment levels. A total of 120 couples (60 intervention, 60 control) participated in the study.
NCT06901323
Effect of L-carnitine supplementation on phenylalanine and brain-derived neurotrophic factor levels in infants and children with phenylketonuria L-carnitine is mainly obtained from foods such as red meat and milk that are limited for PKU patients due to dietary protein restriction. So, L-carnitine deficiency has been described in phenylketonuric patients The aim of this study is to : 1. Compare level of Brain-derived neurotrophic factor in infants and children with phenylketonuria and healthy infants and children. 2. Compare level of phenylalanine and Brain-derived neurotrophic factor before and after L-carnitine supplementation in phenylketonuric patients. 3. Assess Intelligence quotient test before and after L-carnitine supplementation in phenylketonuric patients
NCT06792240
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.
NCT06780332
The 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
NCT04623008
Moms Are Worth It is a lifestyle behavior intervention study aims to prevent excessive gestational weight gain in overweight or obese pregnant women through promotion of the stress management, healthy eating, and physical activity. Eligible women will be randomly assigned to a usual care or an intervention plus usual care group. The intervention will last 20 weeks and will be delivered via weekly web and individual health coaching session (10 of them)
NCT04480567
This 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.
NCT06680765
Evidence on the effectiveness of interventions aimed at creating demand and access to pre-conceptual and antenatal care (ANC) among adolescent girls and young women (AGYW) from sub-Saharan Africa is scarce. A program called Adolescents 360 (A360) led by Population Services International in Nigeria, Ethiopia, Kenya and Tanzania supports adolescent girls to identify contraceptives as a relevant tool for enabling them to pursue their self-defined life aspirations. Avoiding or delaying childbirth is crucial for AGYW to pursue their life goals, but motherhood remains a vital aspiration for many. A360 respects these aspirations and supports AGYW to pursue motherhood when the time comes, safely and aligning with their unique preferences. In 2022, A360 set out to design a maternal, neonatal and child health (MNCH) component that would be layered to the existing sexual and reproductive health interventions in Ethiopia and Nigeria. A360 employed human-centered design (HCD) to design these components. The implementation of these components in real-world settings began in 2023. A360 has designed an evaluation to determine the effectiveness of the MNCH components in improving the AGYW's comprehensive knowledge of what is needed to pursue healthy pregnancies, increase their self-efficacy to access ANC and garner support from their key influencers in the process of pursuing healthy pregnancies. The evaluation's primary objective is to determine the effectiveness of the MNCH components at increasing comprehensive knowledge, self-efficacy and key influencer support to pursue healthy pregnancies among married AGYW in Nigeria and Ethiopia. The secondary objectives are: (a) to evaluate the component's effectiveness at increasing comprehensive knowledge of what care is required to attain healthy pregnancies, (b) to evaluate the component's effectiveness at increasing self-efficacy to attend ANC, (c) to determine the component's efficacy at eliciting support from AGYW's key influencers when they are pursuing healthy pregnancies, and (d) to surface the system-level barriers, facilitators, and opportunities for program improvement during the implementation of the MNCH components. The MNCH components are implemented in Kaduna and Jigawa states in Nigeria and in Oromia, Sidama, Amhara, South and Central regions in Ethiopia. The evaluation is based in the same geographies where the components are implemented. The evaluation uses an implementation-effectiveness hybrid design, blending an outcome evaluation and an implementation science component. The outcome evaluation constitutes of a quantitative survey using a longitudinal approach with two study arms, an intervention, and a comparison arm. It has three assessments: a recruitment phase, and two follow-ups (3 months after recruitment and 8 weeks post-partum (only for pregnant AGYW). Participant recruitment is conducted using recruitment scripts after obtaining oral consent and the administration of a set of screening questions to assess eligibility. Full consenting procedures are executed and written consent obtained prior to involvement in any of the evaluation components. A structured questionnaire is used to gather quantitative data from the survey using a Computer Assisted Personal Interview (CAPI) approach. Furthermore, FGD, IDI and KII facilitation guides are used to gather qualitative data. During this process, all sessions are audio-recorded. Audio records are transcribed verbatim and translated. Data is collected by trained enumerators and qualitative researchers who are certified to conduct human subjects' research. Analysis of quantitative data will follow a repeated measures approach employing generalized estimating equations. The repeated measurements of each subject will be made at two times. Between group t-tests and chi-square tests of comparison will be conducted to identify secondary outcomes which show statistically significant differences between participants in the intervention and comparison arms. The qualitative data transcripts will be rigorously analyzed using NVivo or Dedoose. Open-coding and closed-coding will be utilized to identify themes in the qualitative data. Claim statements attributing change to key intervention drivers will be drawn from narrative stories of program beneficiaries guided by the intervention's theory of change. The evaluation received ethical approvals from Ethiopian Midwives Association (EMwA) Institutional Review Commitee (IRC) and the PSI Research Ethics Board (REB) prior to the enumerators training and field data collection. Subsequently, the ethical approval letters were submitted to the sub-national health departments for their reference and support in the management of the field work.
NCT03419819
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.