Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06594094
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-10
Brief Title:
An Open-label Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients MUSCLE
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Investigator-initiated Clinical Study Evaluating the CRISPR-hfCas12Max Gene Editing Therapy in the Treatment of Duchenne Muscular Dystrophy DMD
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MUSCLE
Brief Summary:
Duchenne muscular dystrophin DMD is an X-linked fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins with symptom onset before age of 6 years in boys These mutations abolish dystrophin production in the muscle leading to dystrophin deficiency at the myofiber membrane continued fiber degeneration the need for assisted ventilation respiratory inflammation loss of walking ability in their teens followed by respiratory and cardiac decline and eventually premature death before the age of 30
Currently there are only glucocorticoids for the standard supportive therapy of DMD which can improve disease symptoms but do not change the outcome of the disease Three antisense oligonucleotide ASOs medicines have been approved to treat DMD with exon 45-55 hotspot region mutations However they can only restore trace amounts of dystrophin protein which is insufficient to bring real clinical benefits Gene replacement therapy has been approved using adeno-associated virus AAV vectors to deliver the mini-dystrophin gene Yet mini-dystrophin gene-expression versions of truncated dystrophin functionality are sacrificed and limited
HG302 uses a single AAV vector to deliver the CRISPRhfCas12Max DNA editing system in the human DMD exon 51 splice donor site Preclinical studies have shown that a single intravenous injection of HG302 significantly restores dystrophin protein expression in muscle fibers and rescues their muscle function in humanized DMD mice to wild-type levels with long-lasting and durable efficacy
Currently there are only glucocorticoids for the standard supportive therapy of DMD which can improve disease symptoms but do not change the outcome of the disease Three antisense oligonucleotide ASOs medicines have been approved to treat DMD with exon 45-55 hotspot region mutations However they can only restore trace amounts of dystrophin protein which is insufficient to bring real clinical benefits Gene replacement therapy has been approved using adeno-associated virus AAV vectors to deliver the mini-dystrophin gene Yet mini-dystrophin gene-expression versions of truncated dystrophin functionality are sacrificed and limited
HG302 uses a single AAV vector to deliver the CRISPRhfCas12Max DNA editing system in the human DMD exon 51 splice donor site Preclinical studies have shown that a single intravenous injection of HG302 significantly restores dystrophin protein expression in muscle fibers and rescues their muscle function in humanized DMD mice to wild-type levels with long-lasting and durable efficacy
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None