Pilot-Study of Thalidomide in Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00231140
• Date of registration: 30/09/2005
• Prospective Registration: Yes
• Primary sponsor: Charite University, Berlin, Germany
• Public title: Pilot-Study of Thalidomide in Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: Randomized, Open, Parallel Group Study for the Evaluation of an Oral Dose of 100 mg Thalidomide and Subsequent Dose Escalation of 400 mg Thalidomide in Combination With Riluzole in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: December 2005
• Target sample size: 40
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT00231140
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Germany

Contacts

• Name: Thomas Meyer, MD
• Affiliation: Charité University Hospital, Berlin, Germany

Key inclusion & exclusion criteria

Inclusion Criteria:

- patients aged 25 and 80 years

- female patients who are either postmenopausal for at least 24 month or who are
willing and able to practice the methods of contraception following the Pharmion-Risk
Managment Program (PRMP)

- Male patients who are willing and able to practice the methods of contraception along
with their female partners of childbearing potential following the PRMP

- Clinical diagnosis of probable and definite ALS

- Sporadic or familial ALS

- Onset of pareses for no more than 4 years

- Vital capacity equal to or more than 65% of the predicted value

- Treatment with riluzole 100mg/day

- Patients who are willing to give informed consent

Exclusion Criteria:

- pregnancy or breast feeding

- female patients who are unwilling or unable to practice the methods of contraception
following the Pharmion-Risk Managment Program (PRMP)

- Male patients who are willing and able to practice the methods of contraception along
with their female partners of childbearing potential following the PRMP

- Patients unlikely to comply with the PRMP and other study requirements

- Patients with significant sensory abnormalities, dementia, uncompensated medical
illnesses and psychiatric disorders

- Laboratory abnormalities consistent with clinically significant cardiovascular,
respiratory, haematological, metabolic, hepatic and renal disease

- Infectious disease including HIV, hepatitis B and C

- monoclonal gammopathy of unknown significance (MGUS)

- History of substance abuse within the past year

- History of recurrent thrombosis

- Continuous non-invasive ventilation (ventilation-free interval equal to or less than
2 hours daily)

- Tracheotomy and invasive ventilation

- Treatment with investigational drug within 3 months prior to screening

- patients with clinically signifikant sensory polyneuropathy (inflammatory neuropathy
cause and treatment sensory sum score - ISS = 2)

- patients with sleep disorder (Epworth Sleeping Scale-ESS = 10)



Age minimum: 25 Years
Age maximum: 80 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis (ALS)

Intervention(s)

Drug: Thalidomide (drug)

Primary Outcome(s)

to evaluate the long-term safety and tolerability of thalidomide

to compare the total number of adverse events (AE), abnormal laboratory tests, and number of patients who completed the study between groups

Secondary Outcome(s)

to determine the number of patients who require percutanous endoscopic gastrostomy (PEG)

to evaluate the sleep quality and somnolence using the Epworth Sleeping Scale: ESS = 18

to determine the number of patients who require continuous non-invasive ventilation or invasive ventilation

to evaluate the frequency and severity of sensory neuropathy using the inflammatory neuropathy cause and treatment sensory sum score – ISS = 4

to investigate the effects of thalidomide on pulmonary function (forced vital capacity) over a 24 week treatment period

to evaluate the frequency of thrombotic events

to evaluate the clinical effect of two oral doses of the thalidomide on the rate of functional decline in ALS patients measured by the ALS Functional Rating Scale-revised (ALS-FRS-R) over a 24 week treatment period

to evaluate the survival time or the time point until invasive ventilation is started

Secondary ID(s)

THL-ALS01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors