Latest studies
- Recruiting
Exploring the hidden burden of living with Glanzmann thrombasthenia
This study wants to hear from people living with Glanzmann thrombasthenia (GT) and their caregivers. We want to understand the daily challenges and feelings of those affected. Your stories can help improve support and understanding for everyone with GT.
Glanzmann thrombastheniaQatar - RecruitingPHASE4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This study looks at a medicine called risdiplam for young children (under 2) with a muscle condition called spinal muscular atrophy (SMA). It's for those who had gene therapy but aren't getting better or are getting worse. The study checks if risdiplam is safe and helps them.
Muscular Atrophy, SpinalQatar - Active not recruitingPHASE3
Study of Durvalumab With Tremelimumab Versus SoC as 1st Line Therapy in Metastatic Non Small-Cell Lung Cancer (NSCLC) (NEPTUNE)
This study is looking at two new drugs, durvalumab and tremelimumab, for advanced lung cancer. It compares them to standard chemotherapy. Researchers want to see if the new combination helps people live longer and is safe as a first treatment for this type of cancer.
Non Small Cell Lung Carcinoma NSCLCQatar - Recruiting
BPH Global Registry
This study is gathering information on men who are treated for an enlarged prostate (BPH) and related bladder symptoms. We want to understand what treatments are being used globally and how successful they are, to help improve care in the future.
Benign Prostatic HyperplasiaLower Urinary Tract SymptomsQatar - Enrolling by invitationPHASE3
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
This study looks at a new treatment called pegtibatinase for Homocystinuria (HCU) in people aged 5 to 65. It's a long-term study to see how safe and effective the treatment is over time, especially for those already in other related studies.
HomocystinuriaQatar - Active not recruiting
An Observational Study to Assess Change in Disease Activity and Adverse Events of Upadacitinib in Adult Participants With Moderate to Severe Ulcerative Colitis (UC) in Real-World Practice
This study looks at how a medicine called upadacitinib works for adults with moderate to severe Ulcerative Colitis in everyday life. Researchers will check its safety and how well it helps manage the condition, following about 1000 people over three years.
Ulcerative ColitisQatar - Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
This study looks at how Homocystinuria, a rare genetic condition, affects children and adults. Researchers want to understand its natural progression in people aged 1 to 65 receiving standard care, to help improve treatments in the future.
Homocystinuria Due to CBS DeficiencyQatar - Active not recruitingPHASE3
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)
This study is for children and young people under 25 with a type of brain tumour called low-grade glioma. It compares a new medicine, tovorafenib, with standard chemotherapy to see which works best and is safest for those needing first-time treatment.
Low-grade GliomaRapidly Accelerated Fibrosarcoma (RAF) Altered GliomaPediatric Low-grade GliomaQatar - Active not recruitingPHASE3
RZ358 Treatment for Congenital Hyperinsulinism
This study is testing a new medicine, RZ358, for children and adults with congenital hyperinsulinism (CHI). This condition causes very low blood sugar. The study will see how well RZ358 works to control blood sugar and if it's safe, compared to current treatments alone.
Congenital HyperinsulinismQatar - RecruitingPHASE3
A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
This study is looking at a new medicine called pegtibatinase for people aged 12-65 with a rare condition called Homocystinuria (HCU). It aims to see if the drug is effective and safe compared to a dummy treatment.
HomocystinuriaQatar