A placebo-controlled double blind randomized trial to investigate the efficacy and safety of the combination of Penicillin G / Hydrocortisone treatment in ALS patients (PHALS).

Phase 2

Completed

• Conditions: Amyotrophic lateral sclerosis; 10029317

• Registration Number: NL-OMON44566
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 2020-07-15
• Last Update Posted: 28 February 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 16

Inclusion Criteria

1. Patients must be over 18 years of age.
2. Patients diagnosed with laboratory supported, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria (Brooks, 1994).
3. Disease duration from symptoms onset no longer than 24 months at the screening visit.
4. Patient with a FVC (Forced Vital Capacity) equal to or more than 80% predicted normal value for gender, height, and age at the screening visit
5. Patients must have bulbar involvement (defined as clinically evident dysarthria or a >=1 point drop on questions 1-3 of the revised version of the ALS functional rating scale (ALS-FRS-R).
6. Patient treated with a stable dose of riluzole (100 mg/day) for at least 30 days prior to screening.

Exclusion Criteria

1. Patients with concomitant frontotemporal dementia (FTD).
2. Patients who underwent gastrostomy.
3. Patients with syphilis or a medical history of syphilis.
4. Patients with known penicillin allergy or patients with a positive penicillin allergy skin test.
5. Patients with a contra-indication for using penicillin (use of methotrexate, renal insufficiency).
6. Patients with a medical history of epilepsy.
7. Patients with a contra-indication for using hydrocortisone (uncontrolled hypertension or diabetes mellitus, ulcus ventriculi or ulcus duodeni, patients with acute infections)
8. In the case of a female with childbearing potential, the patient must not be pregnant or breast-feeding. Women of childbearing potential should use adequate contraception.
9. Patients with a concomitant infection.
10.Patients currently treated with corticosteroids.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Change from baseline to week 48 in Amyotrophic Lateral Sclerosis Functional<br /><br>Rating Scale - Revised (ALSFRS-R).</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Changes in both treatment arms:<br /><br><br /><br>- Survival<br /><br>- Change in sVC (measure for lungcapacity)<br /><br>- Change in muscle strength<br /><br>- Change in plasma creatinin<br /><br>- Mean time spent in each stage of the King*s staging system and ALS<br /><br>Milano-Torino Staging system (ALS-MITOS).<br /><br>- Time to gastrostomy defined as the time from randomization to the time of<br /><br>the gastrostomy.<br /><br>- Changes in score on the EQ-5D-5L.<br /><br>- Change in the Center for Neurological Study - Bulbar Functional Scale<br /><br>(CNS-BFS)<br /><br>- Safety: Number of Serious Adverse Events (SAEs), changes on clinical<br /><br>examination including vital signs and weight, laboratory exams (biochemistry<br /><br>and hematology).</p><br>