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The primary objective of Part A of this study is to assess the efficacy of voxelotor with long term, daily dosing compared to placebo in pediatric participants with SCD as measured by improvement in anemia.
Actively Enrolling: The study is currently recruiting participants.
Closed for Enrollment: The study is not currently recruiting participants.
Interventional: A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the study's protocol. Participants may receive diagnostic, therapeutic, or other types of interventions.
Observational: A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to a specific interventions/treatment. A patient registry is a type of observational study.
Early Phase 1 Trials (formerly listed as Phase 0): Exploratory trials conducted before traditional phase 1 trials to investigate how or whether a drug affects the body. They involve very limited human exposure to the drug and have no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
Phase 1 Trials: Trials that focus on the safety of a drug. They are usually conducted with healthy volunteers, and the goal is to determine the drug's most frequent and serious adverse events and, often, how the drug is broken down and excreted by the body. These trials usually involve a small number of participants.
Phase 2 Trials: Trials that gather preliminary data on whether a drug works in people who have a certain condition/disease (that is, the drug's effectiveness). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.
Phase 3 Trials: Trials that gather more information about a drug's safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. These studies typically involve more participants.
Phase 4 Trials: Trials occurring after FDA has approved a drug for marketing. They include postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. These trials gather additional information about a drug's safety, efficacy, or optimal use.
The online start date is the day that this trial first appeared on the UMMC website, which is not necessarily the day that the trial first began at UMMC.
Studies may have specific age requirements. Some may only accept participants over a specified age and/or under a specified age. Please read the study description and additional eligibility criteria for specific information.
Indicates the sex of people who may participate in a clinical study (all, female, male). Sex is a person's classification as female or male based on biological distinctions. Sex is distinct from gender-based eligibility.
Some studies may be targeted to cancer-related treatments or research. Please read the study description and additional eligibility criteria for specific information.
Some studies may be targeted to child-related treatments or research. Please read the study description and additional eligibility criteria for specific information.
Indicates whether people who do not have the condition/disease being studied can participate in the clinical study.
Healthy volunteers play an important role in research because they help define "normal" ranges. While they may receive no direct benefit, healthy volunteers choose to participate in clinical research because they want to help others and contribute to advancing science.