Familial amyotrophic lateral sclerosis (FALS) MedDRA version: 14.1 Level: LLT Classification code 10036704 Term: Primary lateral sclerosis System Organ Class: 100000004852
Conditions
Interventions
Trade Name: Daraprim
Pharmaceutical Form: Tablet
INN or Proposed INN: PYRIMETHAMINE
CAS Number: 58-14-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
Sponsors
Weill Medical College of Cornell University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. ALS diagnosed as probable, laboratory supported probable or definite according to the World Federation of Neurology El Escorial criteria 2. Age 18 or older 3. Capable of providing informed consent and complying with trial procedures 4. SOD1 mutation confirmation by study team 5. Not taking Riluzole (Rilutek) or on a stable dose for 30 days 6. Not taking Coenzyme QR10R or on a stable dose and brand for 30 days Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 7
Exclusion criteria
Exclusion criteria: 1. History or evidence of malabsorption syndromes 2. Exposure to any experimental agent within 30 days of onset of this protocol 3. Women who are pregnant or planning to become pregnant 4. Women of childbearing potential not practicing contraception 5. Women who are breastfeeding 6. Enrollment in another research study within 30 days of or during this trial 7. Alcoholism 8. Patients taking phenytoin (Dilantin) or other therapy affecting folate levels 9. Dementia (MMSE <22) 10. Seizure disorder 11. Folate deficiency 12. Megaloblastic anemia 13. Cardiovascular disorder/arrhythmia 14. Impaired kidney function, defined as creatinine levels of 2.5 x ULN 15. Impaired liver function, defined as AST or ALT of 3 X ULN 16. Advanced ALS patients, defined as those with any of the following: forced vital capacity <60% (use of BIPAP is allowed); traecheostomy; or mechanical ventilation 17. Use of any of the following medications: cytosine, arabinoside, methotrexate, daunorubicin, sulfonamides, zidovudine, lorazapam, coumadin, sulfamethoxazole, and trimethoprim 18. Patients taking Lithium within 30 days of or during this trial 19. Incapable of providing informed consent and complying with trial procedures
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety and tolerability of pyrimethan in patients with FALS due to measure safety and tolerability by adverse events, laboratory data, and ability to remain on assigned treatment.;Timepoint(s) of evaluation of this end point: Safety and tolerability will be assessed every week, either during the study visit or by phone interview. | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the ability of pyrimethamine to reduce SOD1 protein by 15% in the cerebrospinal fluid in patients with FALS at doses of 75 mg or less.;Secondary Objective: To evaluate the safety and tolerability of pyrimethamine in patients with FALS.;Primary end point(s): reduction of SOD1 protein for 15 % in the cerebrospinal fluid in patients with FALS with pyrimethamin ;Timepoint(s) of evaluation of this end point: last visit (week 36) | — |
Countries
Germany, Italy, United States
Contacts
Public ContactAlbert C. Ludolph
Klinik für Neurologie der Universität Ulm
Outcome results
None listed