Pompe Disease Infantile-Onset Clinical Trials
3 recruiting trials for Pompe Disease Infantile-Onset. Eligibility criteria explained in plain English.
TrialFinderData lists 3 Pompe Disease Infantile-Onset clinical trials drawn from ClinicalTrials.gov, all of which are currently recruiting participants.
Every phased trial in this set is Phase 1 (1 of the listed studies); the remaining records are observational or have no phase recorded on ClinicalTrials.gov.
Research is led by University of California, San Francisco (2), Assistance Publique Hopitaux De Marseille (1), among the most active sponsors registered for these trials.
The most frequently studied intervention is There is no intervention (other, 1 trial), followed by Aldurazyme (laronidase), ARN extraction from muscle biopsies.
Track Pompe Disease Infantile-Onset trials
Subscribe for TrialFinderData updates by email. No spam, unsubscribe anytime.
Recruiting Trials
Clinical trial data sourced from the ClinicalTrials.gov registry, maintained by the National Library of Medicine. Always consult your doctor before considering any clinical trial.
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the...
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in...
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle...
Explore Other Conditions
Frequently Asked Questions
There are currently 3 clinical trials for Pompe Disease Infantile-Onset, with 3 actively recruiting participants. These include trials across all phases from early-stage Phase 1 to late-stage Phase 3.
To join a clinical trial for Pompe Disease Infantile-Onset, review the eligibility criteria on the trial detail pages, then talk to your doctor about whether a trial is right for you. Your doctor can help you evaluate the potential benefits and risks.
Phase 3 trials are large-scale studies that test whether a treatment is effective and monitor side effects. There are 0 Phase 3 trials for Pompe Disease Infantile-Onset, representing treatments closest to potential FDA approval.
Clinical trials follow strict safety protocols overseen by Institutional Review Boards (IRBs) and the FDA. Participants are monitored closely and can withdraw at any time. Always discuss risks and benefits with your healthcare provider before enrolling.
Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.