Ignite Creation Date:
2024-05-05 @ 5:23 PM
Last Modification Date:
2024-10-26 @ 9:31 AM
Study NCT ID:
NCT00443066
Status:
COMPLETED
Last Update Posted:
2023-03-21
First Post:
2007-03-01
Brief Title:
Clinical Study of Spinal Muscular Atrophy
Sponsor:
Columbia University
Organization:
Columbia University
Study Overview
Official Title:
Clinical Study of Spinal Muscular Atrophy
Status:
COMPLETED
Status Verified Date:
2023-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The investigators propose to prepare for clinical trials where SMA patients are asked to join the research effort The visits will include questions physical exam blood drawing and sometimes X-rays and a skin biopsy The investigators will use modern computer methods to process the information during which the investigators will plan a clinical trial Once the clinical trial begins the investigators will offer SMA patients participation if they meet the criteria for that trial
Identifying an effective SMA treatment is very important because there is currently none Clinical trials are the only way to decide whether a new treatment works in SMA patients or not
Identifying an effective SMA treatment is very important because there is currently none Clinical trials are the only way to decide whether a new treatment works in SMA patients or not
Detailed Description:
Spinal Muscular Atrophy SMA is one of the most devastating neurological diseases of childhood Affected infants and children suffer from progressive muscle weakness caused by degeneration of lower motor neurons in the spinal cord and brainstem Clinically four phenotypes are distinguished within the continuous spectrum of disease severity based on the age of onset and the highest motor milestone ever achieved SMA is caused by homozygous deletion of the survival motor neuron-1 SMN1 gene A related gene SMN2 produces low levels of full-length SMN protein due to inefficient splicing There is an inverse correlation between SMN copy number and disease severity presumably mediated by levels of full length SMN protein Therefore increasing the amount of full-length SMN protein is a promising treatment strategy Several drugs targeting splicing efficiency have resulted in increased SMN protein in preclinical assays and are now awaiting clinical testing
With the future objective to conduct clinical trials in SMA the proposed project has 3 specific aims 1 To establish a web-based database that will serve to enroll the patient population and that will facilitate timely recruitment for future clinical trials 2 to plan for clinical trials by a developing reliable outcome measures and b establishing the infrastructure needed to carry out efficient clinical trials c convening meetings of preclinical and clinical researchers involved in SMA drug development to select candidate drugs and 3 to characterize the patient population from a clinical and molecular point of view
With the future objective to conduct clinical trials in SMA the proposed project has 3 specific aims 1 To establish a web-based database that will serve to enroll the patient population and that will facilitate timely recruitment for future clinical trials 2 to plan for clinical trials by a developing reliable outcome measures and b establishing the infrastructure needed to carry out efficient clinical trials c convening meetings of preclinical and clinical researchers involved in SMA drug development to select candidate drugs and 3 to characterize the patient population from a clinical and molecular point of view
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CU52029001 | OTHER | SMA | None |