Clinical Trials
Our Clinical Trials Unit is dedicated to providing access to research therapies for rare metabolic and genetic diseases.
Want to stay informed about research in rare diseases? View our VIPeR website to learn how to stay up to date on research in rare diseases and to participate in clinical trials:
Our Clinical Trials
Below is a list of all our clinical trials. Want to learn more? Email us at research@magiccalgary.ca.
Active Recruiting:
Active-Not Recruiting:
Completed:
Active Recruiting:
PKU: JNT517-301
Short Summary:
This Phase 3 clinical trial is evaluating the safety and effectiveness of an investigational treatment called repinatrabit (JNT-517) for adults with PKU (Phenylketonuria). Approximately 120 participants will take part in the study, which includes a screening period of up to 35 days to confirm eligibility and dietary stability, followed by up to 14 months of study participation.
Who May Qualify:
• Adults 18 years of age or older
• Individuals diagnosed with Phenylketonuria (PKU)
• Body weight greater than 40 kg
• Not currently receiving pegvaliase treatment or have stopped pegvaliase at least 4 weeks prio
Contacts:
Additional Information:
Please see the clinical trials page at clinicaltrials.gov
NPC GM1 GM2: AZ-3102 (AZA-001-301)
Short Summary:
This Phase 3 clinical trial is evaluating the safety and effectiveness of an investigational oral medication called nizubaglustat (AZ-3102) for children and young people living with certain rare genetic conditions, including Niemann-Pick type C disease, GM1 gangliosidosis, and GM2 gangliosidosis. The study will take place over approximately 18 months and will compare nizubaglustat to a placebo to better understand how the treatment may help manage these conditions.
Who May Qualify:
You or your child may qualify for this study if you:
- Are 4 years of age or older
- Have a confirmed diagnosis of Niemann-Pick type C (NPC), GM1 gangliosidosis, or GM2 gangliosidosis
- Are unable or unwilling to take miglustat
- Have genetic testing results confirming the diagnosis through disease-causing genetic variants identified by the study team
Contacts:
Additional Information:
Please see the clinical trials page at clinicaltrials.gov
PKU: Palynziq Registry (Biomarin 165-504)
Learn more about one of our latest studies Pegvaliase: a global, multicenter study to assess maternal, fetal and infant outcomes of exposure to Palynziq® (pegvaliase) during pregnancy and breastfeeding. Read more about the study here.
Noonan Syndrome: BMN111-211
Short Summary:
This Phase 2 clinical trial is studying an investigational medication called vosoritide in children with Noonan syndrome who have experienced limited growth during or after treatment with human growth hormone (hGH). Researchers are evaluating the safety of different dosing options and how well the medication may help support growth, including its potential impact on final adult height.
Who May Qualify:
Your child may qualify for this study if they:
- Are between 3 and 11 years old (girls) or 3 and 12 years old (boys)
- Have a confirmed diagnosis of Noonan syndrome
- Are shorter than expected for their age and sex and have experienced limited growth despite current or previous growth hormone treatment
- Have not yet started puberty at the time of study enrollment
Contacts:
Additional Information:
Please see the clinical trials page at clinicaltrials.gov