Phase 2
N=43
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT01906658 ↗Enrolled (actual)
43
Serious AEs
16.3%
Results posted
Jan 2017
Primary outcome: Primary: Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication — 2; 0; 2; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Repository corticotropin injection (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Mallinckrodt
- Primary completion
- Nov 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication |
2; 0; 2; 0 | — |
| SECONDARY Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation |
2; 0; 2; 0 | — |
| SECONDARY Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication |
0; 0; 0; 0 | — |
| SECONDARY Proportion of Subjects With Treatment Emergent Suicidality |
1; 3; 0; 0 | — |
Summary
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.
Eligibility Criteria
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 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.
- 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.
- 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.
- For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening.
- 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.
- For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
Data sourced from ClinicalTrials.gov (NCT01906658). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.