Clinical trials: Delivery Delayed
- Condition Delivery Delayed
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Crigler-Najjar Syndrome
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year.
- Trial name
- VALENS (NCT03223194)
- Testing
- AT342
- Where
- 4 sites in United States, Israel and 1 more
- Volunteers
- 1 people
- Sponsor
- Audentes Therapeutics
HIV-1 Infection, Receipt of Cal-1 Modified Hematopoietic Cellular Products
Long Term Follow up for the Detection of Delayed Adverse Events in Cal-1 Recipients
Long term safety follow-up of Cal-1 recipients
- Trial name
- NCT02390297
- Testing
- Blood tests
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Calimmune, Inc.
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies