Oakland

Researchers are evaluating the efficacy and safety of trontinemab in people with early symptomatic Alzheimers disease, ranging from mild cognitive impairment to mild dementia due to Alzheimers. This Phase III trial aims to understand how trontinemab affects cognitive decline and disease progression in this population. Participants are randomly assigned to receive either intravenous trontinemab or a placebo in a parallel-group design. Treatment is administered by IV infusion, and the effects are compared over a period of 72 weeks. The study includes comprehensive safety and efficacy assessments throughout this period. During the 72 weeks of the study, participants will undergo various evaluations including cognitive tests such as the Clinical Dementia Rating-Sum of Boxes CDR-SB, Alzheimers Disease Assessment Scales, brain imaging with PET and MRI scans, and biomarker measurements in cerebrospinal fluid and blood. Safety monitoring includes tracking adverse events, infusion reactions, and antibody development. The study requires participants to have a study partner and to complete all study procedures over this time.

Researchers are evaluating a culturally-tailored home-based physical activity program designed for Hispanic or LatinoLatina adolescent and young adult childhood cancer survivors. These survivors may face long-term effects like weight gain, fatigue, and reduced physical fitness after cancer treatment. The study aims to see if this culturally-relevant program can help increase physical activity and improve overall health compared to using a Fitbit tracker alone. The study has two stages. In Stage 1, 20 Latinx survivors participate in developing the intervention using Fitbit trackers, text messages, social media support, wearable activity devices, and interviews over 9 months. In Stage 2, 170 survivors who do not meet physical activity guidelines are randomized to either the intervention group, which includes Fitbit use, weekly goal-setting, peer support via social media and Zoom meetings, and optional activity partners, or a control group that only uses Fitbit trackers for 12 weeks. The intervention includes an intensive phase with weekly sessions followed by a 4-week maintenance phase. Participants will wear Fitbit trackers daily and engage in goal-setting, peer discussions, and physical activity reminders. Researchers will measure moderate to vigorous physical activity, sedentary time, and health-related quality of life over 12 weeks. Additional evaluations include physiological markers of heart and metabolic health and qualitative interviews to improve the program. The study lasts through the intervention phases with ongoing monitoring and support for participants.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

This research aims to observe and track growth patterns and the clinical progression of Hypochondroplasia HCH in children. It focuses on collecting various growth measurements and other relevant data over time to better understand this condition. The study is sponsored by BioMarin Pharmaceutical and involves children diagnosed with Hypochondroplasia, up to 15 years old. Participants include children confirmed to have Hypochondroplasia through genetic testing. The study is observational, meaning there are no experimental treatments involved. Children will be monitored regularly to record changes in growth and body proportions, with data collection occurring every six months over the course of the study, which may last up to 15 years. Throughout the study, children will undergo assessments every six months to measure growth velocity, height, body mass index BMI, and body segment ratios. Quality of life and severity impressions from both patients and caregivers will be evaluated annually. Researchers will also track medical events, use of growth hormone treatments, and any limb lengthening surgeries. This long-term follow-up allows for comprehensive monitoring of growth and health outcomes in children with Hypochondroplasia.

This research aims to evaluate the effects of different doses of vosoritide and compare the therapeutic dose of vosoritide to human growth hormone hGH in children diagnosed with idiopathic short stature ISS. The study is a Phase 2, randomized, controlled trial that seeks to understand how these treatments influence growth in affected children. Participants will first undergo a minimum 6-month observation period to assess their baseline growth. Then, those assigned to the vosoritide and placebo groups will receive randomized treatment for at least 6 months, with placebo limited to a maximum of 6 months. After this, open-label vosoritide treatment will continue until participants reach near-final adult height or at least 16 years for females and 18 years for males, whichever is later. Participants randomized to the hGH group will receive open-label hGH treatment for a minimum of 4 years. Study treatments involve daily injections. Throughout the study, participants will attend regular visits for clinical and imaging assessments, including evaluations of hips and lower extremities. Researchers will monitor safety concerns such as hypotension, fractures, and slipped capital femoral epiphysis, with oversight from an independent Data Monitoring Committee. The main outcomes measured include changes in annualized growth velocity at 6 months and height changes over 4 years. Follow-up assessments will continue as needed, including safety monitoring, until study completion, which could last up to 15 years.

Researchers are evaluating the safety and effectiveness of oral brepocitinib in adults with lichen planopilaris, a condition affecting the scalp. This Phase 23 trial aims to understand how well this medication works compared to a placebo in treating active and symptomatic lichen planopilaris. The study is sponsored by Priovant Therapeutics, Inc. and uses a randomized, double-blind design to ensure reliable results. Participants are randomly assigned to receive either oral brepocitinib or a placebo. The study is conducted in parallel groups, with neither the participants nor the researchers knowing who receives the active drug or placebo. The treatment period lasts 24 weeks, during which the participants take the assigned oral medication. The main goal is to measure improvement in the Investigator Global Assessment IGA score by Week 24. Throughout the study, participants will be regularly monitored for safety and symptom changes. Researchers will assess the proportion of participants who achieve significant improvement in their IGA scores at Week 24 and track changes in symptom severity using a numerical rating scale. The total study duration extends until July 2029, allowing for thorough evaluation of treatment effects and safety over time.

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

Researchers are comparing two chemotherapy treatment plans for patients with newly diagnosed intermediate-risk rhabdomyosarcoma, a type of soft tissue cancer. This phase III trial evaluates whether a higher dose chemotherapy over a shorter time Regimen A is better than a lower dose chemotherapy followed by maintenance treatment over a longer time Regimen B, both combined with standard surgery and radiation. The study also aims to assess survival rates, treatment side effects, and molecular features of the tumor. Participants are randomly assigned to one of two treatment groups. Regimen A involves multiple cycles of vincristine, dactinomycin, and cyclophosphamide given intravenously every 21 days, with possible surgery during week 12 and radiation treatments during specified cycles. Regimen B includes alternating cycles of vincristine, dactinomycin, cyclophosphamide, and irinotecan, followed by 24 weeks of maintenance chemotherapy with vinorelbine and oral cyclophosphamide. Both groups undergo imaging scans and biopsies as needed during treatment. Throughout the study, participants will have regular CT or MRI scans and blood tests, along with other procedures such as lymph node biopsies, bone marrow tests, and lumbar punctures to monitor disease status. After treatment, follow-up visits occur every 3 months for the first year, then less frequently up to 5 years to check for event-free survival and overall health. The trial also collects biospecimens for future research and evaluates treatment effects on fertility and quality of life.

Researchers are investigating the best way to combine chemotherapy and radiation therapy for patients aged 3 to 29 years with localized non-germinomatous germ cell tumors NGGCT in the brain. This phase II trial aims to optimize treatment based on how well the tumor responds to initial chemotherapy, with the goal of reducing spinal cord relapses and adjusting therapy for better disease control. The study also compares different radiation types and examines cognitive and physical effects in children and young adults with NGGCT. Participants first receive induction chemotherapy consisting of carboplatin, etoposide, and ifosfamide over six cycles every 21 days. Based on tumor response, patients are assigned to one of two plans Plan A involves whole ventricular plus spinal canal irradiation WVSCI, delivered daily for 6 weeks, while Plan B includes high-dose chemotherapy with stem cell transplant followed by radiation therapy to the whole brain and spine. Some patients may undergo second-look surgery depending on tumor response before continuing treatment. Throughout the study, participants undergo MRI scans, collection of cerebrospinal fluid and blood samples, and questionnaires assessing cognitive, social, and behavioral functioning. Researchers monitor tumor response, progression-free survival, overall survival, and patterns of disease recurrence for up to 10 years. Safety and side effects are also evaluated to better understand long-term outcomes of these treatment approaches.

Researchers are studying BBP-812, an investigational gene therapy using an AAV9-based vector, to evaluate its safety, tolerability, and pharmacodynamic effects in children with Canavan disease. Canavan disease is a very rare, severe, and fatal disorder with no approved treatments. BBP-812 is designed to deliver a gene called ASPA to restore its expression in brain and other cells. Participants will receive a single intravenous infusion of BBP-812. The study includes a dose-finding phase where participants receive either a low or high dose of BBP-812 on Day 0, followed by an expansion phase where participants receive the selected dose from the earlier phase. This gene therapy is given only once during the study. During the trial, participants will be closely monitored through clinical evaluations, urine and brain imaging tests measuring N-acetylaspartate levels, and assessments of motor, cognitive, communication, and adaptive functions up to one year after infusion. Safety is tracked by recording any adverse events. The total study duration includes baseline assessments and follow-ups up to 12 months post-treatment.