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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01906658
• Date of registration: 15/07/2013
• Prospective Registration: No
• Primary sponsor: Mallinckrodt
• Public title: A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
• Scientific title: A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
• Date of first enrolment: July 2013
• Target sample size: 43
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01906658
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion 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 < 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.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Repository corticotropin injection

Primary Outcome(s)

Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication [Time Frame: Baseline to Week 8]

Secondary Outcome(s)

Proportion of Subjects With Treatment Emergent Suicidality [Time Frame: Baseline to Week 36]

Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation [Time Frame: Baseline to Week 8]

Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication [Time Frame: Baseline to Week 8]

Secondary ID(s)

QSC01-ALS-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/01/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01906658