Clinical Trials Logo

Clinical Trial Details — Status: No longer available

Administrative data

NCT number NCT03062631
Other study ID # 223019
Secondary ID
Status No longer available
Phase
First received
Last updated

Study information

Verified date May 2023
Source University of California, Davis
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial Summary

Congenital myasthenia is a potentially lethal disorder, which, even with careful management, significantly impedes participation in normal daily functions. Currently approved therapies have had little impact on promoting a normal quality of life activity in these patients. The goal is to systematically examine the effect of 3,4-DAP on the natural course of this disease and to gain additional experience in titrating 3,4-DAP with other available therapies to maximize clinical function and development in this patient population. The specific aim of this study is to evaluate the use of 3,4 Diaminopyridine (DAP) on selected patients proven by genetic or serum antibody testing to have Congenital Myasthenic Syndrome (CMS), prescribe 3,4 DAP, and then clinically evaluate the response.


Description:

Read more »
Read more »

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
3,4-Diaminopyridine
The investigator will prescribe an initial dose of 3,4 diaminopyridine (DAP) by mouth based on the participant's weight. This initial dose may be modified later according to the participant's response to treatment.

Locations

Country Name City State
United States University of California, Davis Medical Center Sacramento California

Sponsors (2)

Lead Sponsor Collaborator
Ricardo Maselli Catalyst Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

See also
  Status Clinical Trial Phase
Completed NCT01203592 - Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes Phase 1
Recruiting NCT01403402 - Congenital Muscle Disease Study of Patient and Family Reported Medical Information
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT05408702 - Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes
Not yet recruiting NCT06436742 - A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) Phase 1
Completed NCT01474980 - Pregnancy Outcomes in Congenital Myasthenie Syndrome N/A
Recruiting NCT06078553 - A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)
No longer available NCT02189720 - Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Approved for marketing NCT00872950 - 3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)