NCT01083667

Brief Summary

The objective of this study will be to evaluate the safety, tolerability and effect on SOD1 levels by pyrimethamine in patients with familial amyotrophic lateral sclerosis.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Nov 2009

Longer than P75 for phase_1

Geographic Reach
4 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2009

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

December 17, 2009

Completed
3 months until next milestone

First Posted

Study publicly available on registry

March 10, 2010

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2016

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

June 19, 2017

Completed
Last Updated

June 19, 2017

Status Verified

May 1, 2017

Enrollment Period

5.1 years

First QC Date

December 17, 2009

Results QC Date

February 17, 2017

Last Update Submit

May 19, 2017

Conditions

Familial Amyotrophic Lateral Sclerosis

Keywords

ALSFamilial ALSSOD1 Gene Mutation

Outcome Measures

Primary Outcomes (1)

  • Mean Change in SOD1 CSF

    Reported change in mean SOD1 CSf from baseline to visit 6 (week 18) and end of study for all subjects who completed the measure

    baseline, Visit 6 week 18, end of study

Secondary Outcomes (1)

  • Appel ALS Score

    Week 0, 6, 18, and end of study

Study Arms (1)

Pyrimethamine

EXPERIMENTAL

Open label. Only one arm will receive the intervention.

Drug: Pyrimethamine

Interventions

PyrimethamineDRUG

Open Label, dose escalating,

Also known as: Daraprim
Pyrimethamine

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects with definite, probable, or laboratory supported probable ALS will be eligible.
  • ALS diagnosed as probable, laboratory supported probable or definite according to the World Federation of Neurology El Escorial criteria \[Brooks et al. 2000\]
  • Age 18 or older
  • Capable of providing informed consent and complying with trial procedures
  • SOD1 mutation confirmation by study team
  • Not taking Riluzole (Rilutek) or on a stable dose for 30 days
  • Not taking Coenzyme QR10R or on a stable dose and brand for 30 days

You may not qualify if:

  • History or evidence of malabsorption syndromes
  • Exposure to any experimental agent within 30 days of onset of this protocol
  • Women who are pregnant or planning to become pregnant
  • Women of childbearing potential not practicing contraception
  • Women who are breastfeeding
  • Enrollment in another research study within 30 days of or during this trial
  • Alcoholism
  • Patients taking phenytoin (Dilantin) or other therapy affecting folate levels
  • Dementia (MMSE \<22)
  • Seizure disorder
  • Folate deficiency
  • Megaloblastic anemia
  • Cardiovascular disorder/arrhythmia
  • Impaired kidney function, defined as creatinine levels of 2.5 x ULN
  • Impaired liver function, defined as AST or ALT of 3 X ULN
  • +4 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Weill Cornell Medical Center/New York Presbyterian Hospital

New York, New York, 10021, United States

Location

Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

Universitäts- und Rehabilitationskliniken Ulm

Ulm, Germany

Location

Milano Neurological Institute

Milan, Italy

Location

Umea University

Umeå, Sweden

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Pyrimethamine

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

PyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Results Point of Contact

Title
Dale Lange
Organization
HSS/WCMC
langed@hss.edu
6467978917

Study Officials

  • Dale J. Lange, M.D.

    Hospital for Special Surgery/Weill Cornell Medical Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Neurologist in Chief

Study Record Dates

First Submitted

December 17, 2009

First Posted

March 10, 2010

Study Start

November 1, 2009

Primary Completion

December 1, 2014

Study Completion

May 1, 2016

Last Updated

June 19, 2017

Results First Posted

June 19, 2017

Record last verified: 2017-05

Locations

US(2)SE(1)IT(1)DE(1)