The 111-902 Study is an observational clinical study. In an observational clinical study, participants agree to have information collected – in this case growth information. In the 111-902 Study, study doctors will analyze the data to learn more about hypochondroplasia, which may eventually help others in the future.
A subgroup of eligible children from the 111-902 study will be asked to participate in a clinical trial of a new treatment for hypochondroplasia. To participate in the clinical trial, all children must first be enrolled in the 111-902 study for at least 6 months to assess their current growth.
Clinical studies follow specific rules to protect the rights, safety, well-being, and confidentiality of study participants.
Participation in the 111-902 Study is completely voluntary. If you decide to allow your child to participate in the study, you can end their participation at any time and for any reason without affecting their usual medical care.
A clinical trial is a research study done to evaluate new potential treatments in people. During a clinical trial, information is collected to determine whether a product candidate is safe and effective, as well as to evaluate the risks and benefits of the medicine. For more information about clinical trials, please visit ClinicalTrials.gov or The Center for Information and Study on Clinical Research Participation
Research and development occurs in several stages before clinical trials commence in people. Preclinical testing is conducted for each investigational product to evaluate the safety, efficacy, best practices for administration, and many other properties before entering human clinical trials. Once preclinical testing is complete, an Investigational New Drug (IND) application is submitted to a regulatory authority, such as the U.S. Food and Drug Administration, so the regulator can evaluate the safety of the product candidate and ensure that clinical trial participants will not be subjected to an unreasonable risk.
Clinical trials are conducted in four phases:
A study drug is evaluated in people, often in volunteers without the condition, for the first time to evaluate its safety and best practices for administering the product candidate.
The product candidate is evaluated to determine a tolerable dose or range of doses, to further evaluate its safety, and to begin testing in participants with the condition of interest to determine if it has the intended or predicted effects.
The product candidate is evaluated, often in larger trials of longer duration, to confirm its effectiveness and to further evaluate safety. Phase 3 trials often compare the study drug to commonly used treatments (if any) or to placebo treatment, if it is scientifically and ethically appropriate to do so.
Post-marketing studies are performed after regulatory agency approval occurs. These studies are designed to collect additional information including the medicine’s risks, benefits and optimal use in a broader patient population, often over extended periods of time. For example, a Phase 4 study could be a registry of patients with a certain condition to collect medical information and better understand outcomes, both in patients receiving the medicine and those who are not.
When phase 1–3 clinical trials are complete, a regulatory application is submitted to regulatory agencies. The application contains all data gathered about the safety and effectiveness of the study drug from the preclinical studies and clinical trials that have been performed. The application also contains information about the chemistry, toxicology, pharmacology and manufacturing processes of the product. The regulatory agency reviews the data and, if approved, the new medicine can be marketed and distributed to the public through prescription by a qualified physician.
When testing potential new therapies in clinical trials, it is critical to include the broadest possible range of individuals, to ensure these medicines have the best chance of improving outcomes for the diversity of patients who will ultimately receive them. At BioMarin, we create pioneering medicines for people with genetic conditions around the world, and we are deeply committed to enrolling representative populations in our clinical trials.