Trial Search Results

A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies.

This study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and tolerable at that time have the option to continue into the open-label extension period. A 3-week treatment taper and a follow-up visit are planned for all patients enrolled in the study, beginning either at Week 8 or at Week 36 if a patient continues into the optional open-label extension period.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Mallinckrodt

Stanford Investigator(s):

Intervention(s):

  • Drug: Repository corticotropin injection

Phase:

Phase 2

Eligibility


Inclusion Criteria:

   - Able to provide informed consent.

   - Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS,
   clinically probable ALS, or clinically possible ALS based on the revised El Escorial
   criteria.

   - Patients with ALS ≤ 3 years since symptom onset. Symptom onset is defined as date of
   first muscle weakness or dysarthria.

   - Upright slow vital capacity (SVC)≥ 60% of predicted.

   - If taking riluzole and/or Nuedexta®, stable regimen is required for ≥ 30 days prior to
   screening.

   - Medically (either independently or with caregiver assistance) able to comply with
   study procedures, including subcutaneous (SC) injections of study medication and
   adherence to concomitant medication restrictions.

Exclusion Criteria:

   - Any medical condition known to have an association with motor neuron dysfunction which
   might confound or obscure the diagnosis of ALS.

   - Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type
   (e.g., bilevel positive airway pressure) for treatment of ALS-related respiratory
   dysfunction (vital capacity of < 60% predicted, nocturnal desaturation, and/or
   nocturnal hypoventilation). Patients on assisted ventilation for other reasons require
   approval from the Medical Monitor. (Supplemental oxygen is acceptable).

   - Recorded diagnosis or evidence of major psychiatric disorder.

   - Clinically evident cognitive and/or behavioral impairment that in the opinion of the
   Investigator would impair the ability of the patient to comply with the study
   procedures.

   - Therapies and/or Medications:

      1. History of prior sensitivity to Acthar or other porcine protein products.

      2. Chronic systemic corticosteroid use, defined as > 20 mg of prednisone or
      equivalent systemic corticosteroid taken for more than 4 consecutive weeks within
      6 months prior to randomization. Topical, inhaled, or intra-articular
      corticosteroids are allowed.

      3. Planned treatment with live or live attenuated vaccines once enrolled in the
      study.

   - Participation in another therapeutic (drug or device) investigational study within 30
   days prior to screening.

   - Type 1 or type 2 diabetes mellitus, or patients currently taking hypoglycemic
   medication.

   - Contraindication per Acthar Prescribing Information, Appendix D Section 4:
   scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent
   surgery, history of or the presence of peptic ulcer, congestive heart failure,
   uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical
   hyperfunction.

      1. For the purposes of this study, osteoporosis is defined as a history of a lumbar
      spine and/or femoral neck T-score ≤ -2.5 on bone densitometry (DXA), OR
      osteoporosis requiring pharmacologic therapy, OR a history of non-traumatic low
      impact hip or vertebral fracture, OR patient reported history of osteoporosis.

      2. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months
      prior to screening.

      3. For the purposes of this study, uncontrolled hypertension is defined as mean
      systolic blood pressure ≥ 140 mmHg and diastolic blood pressure ≥ 90 mmHg on ≥ 3
      seated readings taken at least 5 minutes apart during the screening period.

      4. For the purposes of this study, congestive heart failure is defined as New York
      Heart Association Functional Class III-IV.

Ages Eligible for Study

18 Years - 80 Years

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shirley Paulose
650-724-3792
Not Recruiting