Clinical trials

The main objective is to determine if in-home portable air cleaners provide persistent reductions in PM2.5 exposures and improvements in systolic blood pressure and biochemical parameters over 4-weeks in patients with metabolic syndrome residing in Qatar.

This randomised controlled trial aims to compare treatment outcomes between pulpotomy and pulpectomy when used to treat vital primary molars diagnosed with symptomatic irreversible pulpitis. Compared to the standard pulpectomy treatment, pulpotomy is a technically simpler procedure, less time consuming, easier for young patients to tolerate, while retaining the proprioceptive sensation of the tooth - all important advantages when treating young children.

Benign prostatic hyperplasia (BPH) is one of the most common performed surgical procedures in urology. Over the past few decades there have been an increasing development of newer surgical treatment options. Additionally, the outcome parameters for BPH treatments have been standardized. While data are available for the initial pivotal studies, post-market release data are lacking. Under the umbrella of uCARE, we have started a prospective, ongoing international registry for recording demographics and outcomes for patients undergoing surgical treatments for BPH.

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

BEYOND study is designed to generate the first real-world data from GCC countries on the patient characteristics, treatment patterns, survival outcomes, and safety of T-DXd in patients with HRpositive,HER2-low or HER2-ultralow mBC previously treated with ET. The evidence generated will help to optimise treatment strategies, inform clinical guidelines, and ultimately improve outcomes for patients with mBC across the region.

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care. Study details include: * Total Study duration: up to 38 weeks * Screening: * Initial Screening duration: up to 4 weeks * Pre-treatment Diet Standardization Period duration: up to 6 weeks * Blinded Treatment Duration: 24 weeks * 2-week blinded dose titration period * 22-week blinded assessment period * Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)

Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.

This study aims to assess the impact of dupilumab on the mental health and quality of life of moderate-to-severe Atopic Dermatitis (AD) patients. The study will recruit participants from AD patients who are already receiving dupilumab treatment. The study enrollment period will be about 9 months with each of the participants undergoing a 6-month observational study period.

A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 12 years of age with hyperkalaemia.

INTERSTELLAR study will generate critical prospective real-world evidence on the benefits of adding Anifrolumab to standard of care treatment for SLE in routine clinical practice, to inform physicians, payers and patients. The study will use clinical assessments that are relevant for SLE-treating physicians in routine clinical practice, as well as introduce a specific measure for skin manifestations to affirm the potency of anifrolumab in treating SLE-related skin manifestations. The study will use standardized objectives, inclusion/exclusion criteria and outcome measures across all countries participating in this study including GCC (Qatar, KSA), Mexico, CAMCAR (Costa Rica, Panama, Dominican Republic), Colombia, Argentina, Taiwan, and Egypt, and any other countries that may be included in the study, in order to facilitate a comparison and analysis across all countries included in this study.

Our objective is to create a cardiometabolic cohort that could be representative of the local population, consisting of Qataris and long-term residents, in order to identify the prevalence, risk factors, and clinical characteristics of cardiometabolic disorders, as well as the incidence of atherosclerotic cardiovascular disease events.

Non-Hodgkin lymphoma (NHL) is the most common hematologic malignancy, with over 80,500 estimated new cases diagnosed in the United States in 20231. Diffuse large B-cell lymphoma (DLBCL) is the most frequent subtype of NHL, accounting for 30%-40% of cases2. DLBCL is an aggressive malignancy with heterogeneous biology and behavior. Disease risk stratification and treatment planning involve various patient and clinical characteristics (e.g., age, stage, and tumor bulk), prognostic indices (e.g., International Prognostic Index (IPI) score), and gene expression profiling. Patients typically present with nodal or extranodal disease, usually exhibiting rapid tumor growth and symptoms that are highly dependent upon the tumor localization. The diagnosis and subtyping of DLBCL have significantly advanced, from morphological assessment of tissue slide to numerous ancillary tests, including immunophenotyping performed by immunohistochemistry (IHC), cytogenetics, and detailed molecular testing to classify the disease based on cell of origin (COO). With the advent of novel therapeutic options, molecular subtyping of DLBCL at diagnosis is expected to allow prognostic stratification of patients into distinct subgroups. This stratification could provide a preclinical rationale for therapeutic targeting the involved pathways and paving the application of personalized treatment. DLBCL is a potentially curable disease with an overall 60-70% chance of achieving durable complete remission (CR) with the currently used standard first-line immunochemotherapy. However, 30-40% of patients are either refractory to first-line treatment or experience relapse and eventually will die of disease progression7. Although high-dose chemotherapy followed by autologous stem cell transplant (ASCT) is the recommended SOC for eligible patients in the second-line setting based on results from the pivotal PARMA study, real-world SOC in this setting remains less clearly defined. Patients not cured with ASCT or ineligible to ASCT or refractory to salvage chemotherapy may be considered for Chimeric Antigen Receptor (CAR) T cell therapy targeting CD1910. Although ASCT and CAR-T cell therapy offer patients an opportunity for durable remission, many patients may not be eligible for ASCT or CAR-T cell therapy or relapse after these treatments. In the last decade, the investigation of novel antigens, which can be targeted by immunotherapy and identified to eliminate malignant cells regardless of their molecular pathogenesis, has been constantly pursued. This study aims to address this need by examining the demographic, clinical characteristics, and treatment patterns and exploring access to novel therapies for diffuse large B-cell lymphoma (DLBCL) patients, both treatment naïve and relapsed/refractory patients, in the Middle East and Africa (MEA) region.

The goal of this clinical trial is to learn if metformin can improve asthma control in overweight and obese children with mild, moderate, or severe asthma. It will also evaluate the safety and metabolic effects of metformin in this population. The main questions the study aims to answer are: 1\) Does metformin improve asthma control, as measured by the Asthma Control Test (ACT) score? 2) Does metformin improve lung function, reduce asthma exacerbations, and improve metabolic and inflammatory markers in overweight/obese children with asthma? Researchers will compare metformin with a placebo (a look-alike substance that contains no active drug) to see if metformin improves asthma outcomes in overweight/obese children with asthma. Participants will take either metformin or a placebo in addition to their standard asthma treatment during the study period. They will attend scheduled clinic visits for asthma assessments, lung function testing, and safety monitoring. Blood, stool, and saliva samples, along with clinical information, will be collected from participants to assess asthma control, and markers related to metabolism and inflammation. Participants will also complete asthma control questionnaires and report medication use and asthma symptoms throughout the study period.

CAROTID-STABILISE is a phase III, multicentre, randomised, double-blind, placebo-controlled trial evaluating whether alirocumab 150 mg subcutaneously every 2 weeks, added to high-intensity statin therapy, produces greater reduction in intraplaque haemorrhage (IPH) volume at 26 weeks compared with placebo in patients with recently symptomatic carotid stenosis of 50-69% harbouring IPH or lipid-rich necrotic core (LRNC) on high-resolution vessel-wall MRI. The study will enroll 280 participants across multiple centres with a 52-week extension for durability and clinical endpoints assessment.

This study aims to isolate endothelial progenitor cells (EPCs) from participants with type 2 diabetes (T2D) and cardiovascular complications and to comprehensively characterize EPC dysfunction. Specifically, the study will evaluate maladaptive angiocrine signaling, calcium signaling pathways, and the role of inflammation in EPC function and the progression of atherosclerosis during T2D development. A sub-study will assess EPC functionality by examining endothelial nitric oxide synthase (eNOS) expression and activity, as well as the effectiveness of in vitro eNOS gene enhancement.

REVIVE-CVST is a multicenter, prospective, randomized, open-label, blinded-endpoint (PROBE) trial evaluating whether early endovascular thrombectomy (EVT) combined with standard anticoagulation improves outcomes compared to anticoagulation alone in patients with severe cerebral venous sinus thrombosis (CVST). The study targets adult patients (aged 18 years or older) presenting within 14 days of symptom onset with imaging-confirmed CVST and at least one severity marker, such as a Glasgow Coma Scale score of 14 or below, intracerebral hemorrhage, venous infarction, or deep venous system involvement. Participants will be randomly assigned in a 1:1 ratio to either the intervention arm (EVT plus anticoagulation) or the control arm (anticoagulation alone). The primary endpoint is functional outcome at 180 days as measured by the modified Rankin Scale (mRS), using a shift analysis across all mRS categories. The trial aims to enroll 440 participants across approximately 15 centers in the Middle East, North Africa, South Asia, and Turkey (MENA-SINO network). The study duration is approximately 42 months, including 18 months of enrollment and 12 months of follow-up for the last enrolled patient.

Cardiovascular disease is the leading cause of death worldwide, and individuals with diabetes or other cardiometabolic conditions are at increased risk of adverse cardiovascular outcomes. Although advances in prevention and treatment have reduced cardiovascular events globally, cardiometabolic disease continues to represent a significant health burden, particularly in regions with high diabetes prevalence. In Qatar and other Gulf Cooperation Council countries, the prevalence of diabetes and obesity is increasing, contributing to a high proportion of participants presenting with acute coronary syndrome who have type 2 diabetes or prediabetes. This observational study will use electronic medical record data from patients hospitalized at the Heart Hospital with acute coronary syndrome and a concomitant diagnosis of diabetes or prediabetes. The study will assess trends in cardiovascular risk factors and cardiovascular events, including readmission and mortality. An artificial intelligence component will be used to develop and validate machine learning based risk prediction models to forecast adverse cardiovascular outcomes in participants with cardiometabolic disease. These models will integrate clinical, biochemical, imaging, and other non-invasive data routinely collected during participants care to identify predictors of cardiovascular events.

This prospective, non-randomized, unblinded pilot study evaluates and compares the intrapulmonary distribution of exogenous surfactant in preterm neonates when administered via Less Invasive Surfactant Administration (LISA) versus conventional endotracheal intubation (ETT). Lung ultrasound (LUS) will be utilized to assess the pioneer Surfactant Distribution Homogeneity Index (SDHI) to quantify the evenness and extent of surfactant-induced lung aeration. Secondary objectives include evaluating changes in LUS scores, short-term clinical respiratory outcomes, and feasibility parameters for guiding future larger-scale trials.

When patients leave the emergency department, mistakes with their medications are common and can lead to complications or hospital readmissions. Pharmacists are trained to help prevent these problems, but pharmacist-led transition of care services are not routinely provided in emergency departments. This study is a small pilot randomized controlled trial designed to see whether a pharmacist-led transition of care program can be carried out successfully in the emergency department at Al-Wakra Hospital. The study will help determine if a larger trial is feasible in the future. Patients who are being discharged home from the emergency department and meet the study criteria will be invited to participate. Those who agree will be randomly assigned to one of two groups: Usual care, or Usual care plus the pharmacist-led transition of care program The pharmacist-led program includes reviewing the discharge prescription, checking and updating the medication list, providing medication education, arranging follow-up with a pharmacist-run clinic, communicating with outpatient pharmacists, and following up with the patient after discharge. The pilot trial will help determine how many patients are eligible, how many agree to participate, how well the intervention can be delivered in the emergency department, and whether patients and staff find it acceptable. The results will be used to plan a larger study that will test whether this program can reduce healthcare use after discharge.

Urinary incontinence (UI), Chronic low back pain (CLBP) in women are prevalent and often coexist. Multiple conditions when coexist, it leads to reduced Quality of life in turn causing physical and mental impairment. Musculoskeletal structures and fascial restrictions is one of the attribute to the coexistence of urinary incontinence and chronic back pain in women. External myofascial mobilization (EMM) with core exercises have been proven beneficial for conditions in males that is caused by musculoskeletal and fascial structures. However there are limited research which proves the effect of EMM in conditions involving musculoskeletal and fascial structures . Therefore, the aim of the study is to find the effect of MFTs in reducing the disability in women with CLBP and UI PRIMARY OBJECTIVE To evaluate the effectiveness of PEMM treatment in reducing disability, pain and improving mental health in women with CLBP and UI Study design : Simple randomization Sampling method: Simple Random sampling Random numbers will be generated in Excel, then random numbers will be presented in a concealed envelope for the participants to choose Sample Size : 130 (Experimental 65, Control 65) Sample size was determined using expected effect size i.e., mean change in the primary outcome variable disability index reported in the recent research study . With effect size (change in the disability index score) 3.5 and standard deviation of the change in the outcome 6.45, statistical power 80% and level of significance 5%, the required sample size would be n=55 participants in each group. However, to accounts for multiple secondary outcome measures, possible dropouts and non-response it would be good to increase an additional 20% in calculated sample size i.e., a total of 130 participants (65 participants in each group) will be included in this study. The following sample size equation was used to determine adequate sample size:n = \[2 (Z?/2 + Z?)2 \* ?2\] / (m1-m2)2 Where Z? and Z? are the values standard normal variate e.g., at 5% level of significance Z?/2 = 1.96, and with 80% power the value of Z?= 0.84; ? is the polled Standard deviation and m1 and m2 are the mean outcome values s in group 1 and group 2 respectively. Blinding: Single blinding: patient will be blinded to the group allocation. The assessor is blinded to the treatment Material and tools : Theragun - Theragun Elite, 20 V, Myofascial tools - foam roll, foam ball, Questionnaires - SF 3642,43,44, Oswestry Low back pain questionnaire (ODI)45 and The International Consultation on Incontinence Questionnaire Urinary Incontinence Short Form (ICIQ-UI-SF)46 PROCEDURE In the course of the study the total number of patients referred to physiotherapy department with LBP will be interviewed to identify patients with or without UI having LBP. The data obtained from this will be recoded as prevalence in results. The documents of the patients having LBP with UI will be reviewed to check who fulfills the inclusion criteria. All who meets the inclusion criteria will be approached face to face with the aim of the study. Informed consent form will be given to patients who are volunteering to participate in the study. The data of the participants who do not wish to participate in the study will also be recorded. The participants who have consented to participate in the study will be then assessed. Since the participants has UI she will be directed to a Urologist for an assessment. After urologist assessment, patients will be randomly allocated to experimental and control groups. Random allocation will be administered by the random numbers generated and will be presented in a concealed envelope for the participants to choose. 1st week, the demographic data and Outcome measure of pain, SF36, ODI, ICIQ-UI-SF will be recorded (1st time) on the day of assessment. The outcome measures will be printed on paper and will be given to the patients to fill up with a pen, all questionnaires are self reported outcome measures. Treatment will commence a week after, where experimental group will undergo PEMM therapy with strengthening for 6 weeks duration and the control group will have conventional symptomatic pain treatment with strengthening (2nd to 7th week). Treatment will be given for 6 weeks (1 sessions in a week). The treatment day may vary any day between 4th and 7th day post one session. After 6 sessions of treatment, patient will be advised HEP (Home exercise program) to continue at home. Reassessment will be done on the 8th week where the outcome measures of pain, SF36, ODI, ICIQ-UI-SF will be recorded. Participants will be educated and advised regarding HEP. Exercises will be taught to the patient by explanation, demonstration, teach-back method, and printed sheet of exercise. Compliance to HEP will be recorded before final Outcome measure

Brief Summary The goal of this clinical trial is to evaluate whether scheduled maternal position changes using a peanut birthing ball improve labor outcomes in low-risk, primiparous women receiving epidural analgesia during labor. The main questions it aims to answer are: Does scheduled maternal positioning with a peanut ball reduce the duration of the first and second stages of labor? Does scheduled maternal positioning with a peanut ball influence mode of delivery and maternal and neonatal outcomes? This non-invasive randomized controlled trial will be conducted at the Women's Wellness and Research Center in Qatar. Participants will be randomly assigned to either an intervention group receiving scheduled maternal position changes using a peanut birthing ball or a control group receiving standard intrapartum care without peanut ball use. Researchers will compare outcomes between the two groups to determine whether structured positioning with a peanut ball improves labor progression and delivery outcomes in women receiving epidural analgesia. Participants in the intervention group will undergo scheduled position changes throughout labor using a peanut birthing ball, including left lateral, right lateral, semi-sitting, and Taylor positions, under the supervision of trained nursing and midwifery staff. Participants in the control group will receive routine intrapartum care following epidural analgesia without the use of a peanut ball or a structured positioning schedule. Primary maternal outcomes include duration of the first and second stages of labor, mode of delivery, estimated blood loss, postpartum hemorrhage, use of oxytocin augmentation, degree of perineal trauma, and hospitalization cost. Neonatal outcomes include Apgar scores at 5 and 10 minutes, NICU admission, birth-related injuries, and umbilical cord blood pH. Findings from this study are expected to support evidence-based intrapartum care practices and inform clinical protocols for women receiving epidural analgesia.

Subarachnoid hemorrhage (SAH) is a devastating neurological disorder associated with significant mortality and morbidity rates, arising not just from the hemorrhage itself but also because of the catastrophic multisystem sequelae that can accompany the condition. Rupture of an intracranial aneurysm accounts for up to 85% of instances of SAH, occurring in approximately 3 to 25 people per 100,000 annually in most populations. Treatment of aneurysmal SAH (aSAH) includes prevention of re-bleeding, evacuation of space-occupying hematomas, management of hydrocephalus, and prevention of secondary cerebral insult. Severe headache is the predominant characteristic symptom of aSAH, developing almost instantaneously at ictus in 50% of cases and continuing into the first days. Its severity has a variety of physiological and psychological effects on the patient. Scalp blocks have been suggested to alleviate this headache in case series. However, there is no strong evidence supporting this intervention. In this study, we aim to assess the impact of scalp blocks on headache reduction in patients undergoing endovascular treatment of an aneurysm (coiling or flow diversion) with aneurysmal subarachnoid bleeding.

Chronic Kidney Disease (CKD) is recognized as a leading health problem globally. It is associated with multiple consequences such as cardiovascular diseases, infections, reduced cognitive function, and higher mortality rates. In Qatar, it is estimated that 13% of the population suffers from CKD. Management of CKD is associated with polypharmacy (the use of multiple medications), which burdens the patients and leads to adverse health and economic outcomes. As documented by previous studies, CKD setting is associated with a high medication burden, which leads to non-adherence, reduced quality of life, and other negative sequelae. These consequences can be minimized or averted by implementing a deprescribing program. Deprescribing is defined as the supervised process of intentionally stopping a medication, altering the dose or introducing a safer alternative to improve a person's clinical and quality of life outcomes. Previous deprescribing initiatives in inpatient and outpatient hospital settings were successfully implemented. In general, there are limited deprescribing initiatives in CKD settings. There is a need to provide evidence of the impact of deprescribing programs on improving clinical and economic outcomes in this setting. In Qatar, there is no evidence of the effectiveness of implementing deprescribing programs in clinical settings. Therefore, we have built a team of researchers, clinicians, and stakeholders, and initiated a collaboration with deprescribing experts to fit into the Qatar healthcare system. This project aims to initiate a deprescribing multidisciplinary team and to evaluate the impact of providing such services on the clinical and economic outcomes among CKD patients in Qatar using a randomized controlled trial approach. The findings could have a potential positive impact on the professional practice and patient safety represented by health and economic outcomes.

Regular exercise can improve function and quality of life as well as have other positive behavioral and health-related benefits in people Parkinson's disease (PD). Despite the benefits, insufficient exercise is common among PD individuals, often due to common barriers, emphasizing the importance of empowering individuals with adequate knowledge and self-management skills. This project aims to develop "Engage-Qatar PD," an online physical activity self-management program for people with PD in Qatar. Engage-Qatar PD will be grounded in a previously developed coaching intervention used in people with Huntington's disease (Engage HD) and people with PD. The main focus of this project will be the development work to adapt this intervention for online delivery for people with PD and within the Qatari context. This development stage will entail participatory design, in which users will be actively involved as co-designers to ensure fully the required cultural adaptations. Following this development phase, a pilot randomized controlled trial will be conducted to assess the feasibility, acceptability, and potential benefits of the developed intervention. The innovative approach proposed for this project would have wide-reaching impact, advancing the development of new therapeutic options. It will deliver a realistic, culturally adapted therapeutic option for people with PD in Qatar that has the potential to be implemented in a variety of healthcare settings and other countries in the region. Importantly, future work could replicate this program in a larger cohort of individuals with other neurodegenerative diseases and extend this work to other countries in the region.

People with stroke often experience limitations in motor functions and activity participation. Occupation-based bilateral upper limb training (OBBT) using occupations as meaningful daily activities, and as part of the therapy. The activities in OBBT are tailored to the patient's life with the goal of boosting motivation and engagement to enhance activity performance and participation. This study is a randomized control trial that aims to explore the effect of OBBT along with the conventional therapy in comparison with the conventional therapy alone on motor functions and activity participation among stroke survivors in inpatient rehabilitation setting. This study will be conducted in Qatar Rehabilitation Institute, Hamad Medical Corporation, Doha, Qatar. Participants will be allocated randomly to either the OBBT group or the conventional therapy group. Participants in the OBBT group will receive 30 minutes OBBT program in addition to conventional therapy (90 minutes). Intervention sessions will be conducted 5 times per week over 6 weeks. The participants in the conventional therapy group will receive only the conventional therapy (90 minute). All participants will be assessed using the outcome measure immediately the day after completing the last intervention session at 4th week and 6th week.