ACTH in Progressive Forms of MS

Phase 2TerminatedNCT01950234

University of Minnesota59 enrolled

Overview

This is a phase II, randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety, tolerability, and efficacy of adrenocorticotropic hormone (ACTH, Acthar gel) administered as a pulsed regimen consisting of injections on three consecutive days per month in patients with progressive forms of Multiple Sclerosis (MS). Patients will be randomly assigned to either an ACTH arm or a placebo arm. The main hypotheses are that 1) pulsed ACTH will be safe and well-tolerated, and 2) pulsed ACTH will slow progression of clinical and paraclinical measures of MS progression compared to placebo.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Secondary Progressive Multiple Sclerosis Primary Progressive Multiple Sclerosis Progressive Relapsing Multiple Sclerosis

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female patients with a confirmed diagnosis of MS by McDonald criteria * Age \>/= 18 years * SPMS, PPMS, or PRMS phenotype, according to Lublin and Reingold criteria * EDSS 2.0 - 6.0, inclusive * Able to understand the consent process Exclusion Criteria: * Known intolerance of ACTH or corticosteroids * Diabetes mellitus, defined as pre-existing diagnosis, fasting blood glucose \> 125 mg/dl, or glycosylated hemoglobin \>/= 6.5% * Osteoporosis, defined as pre-existing diagnosis or T-score on dual-energy x-ray absorptiometry (DEXA) scan of \</= -2.5. * Current serious medical condition which may interfere with subject's ability to complete the study, or for which pulsed ACTH therapy is contraindicated or might complicate current therapy (e.g., cancer, severe psychiatric illness, chronic infections, autoimmune disorders) * Treatment with cytotoxic agents (including but not necessarily limited to mitoxantrone, cyclophosphamide, alemtuzumab, or rituximab) within 3 years prior to randomization * Treatment with non-cytotoxic immunosuppressive agents (including but not necessarily limited to corticosteroids, ACTH, azathioprine, mycophenolate mofetil, methotrexate or natalizumab) within 3 months prior to randomization * Treatment with FDA-approved first-line MS disease-modifying therapies (B-interferon, glatiramer acetate, fingolimod, teriflunomide or dimethyl fumarate) will be permitted, as long as treatment has been ongoing and stable for at least 3 months prior to randomization * Treatment with dalfampridine or compounded 4-aminopyridine (4-AP) will be permitted as long as treatment has been ongoing and stable for at least 3 months prior to randomization * Stimulant medications for fatigue (such as methylphenidate, modafinil, armodafinil, amantadine or dextroamphetamine) will be permitted, but subjects will be asked to not take these medications on study visit days until all study procedures/assessments are completed.

Outcomes

Primary Outcomes

Proportion of Patients Exhibiting a 20% Worsening in T25FW at 36 Months

Time frame: Month 36

Secondary Outcomes

Safety and Tolerability of ACTH: Menstrual Changes [Female]

Count of participants self reporting at any time throuhgout the study noticing any changes in menstrual cycle or period.

Time frame: Month 36

Safety and Tolerability of ACTH: DEXA Scans

The number is a T-score. There is no maximum or minimum value. Mean change in average of 5 DEXA scan values dual-energy x-ray absorptiometry; measures bone mineral density. Higher numbers are better. A DEXA T-score has a mean of 0 and standard deviation of 1, meaning that a T-score represents how many standard deviations a person's bone density is away from the average bone density of a healthy young adult, with a score of 0 indicating average bone density. Higher scores are better, indicating better bone density. A T-score of -1 to +1 is considered normal bone density, a score between -1 and -2.5 indicates osteopenia (low bone mass), and a score of -2.5 or lower indicates osteoporosis.

Time frame: Month 36

Safety and Tolerability of ACTH: Bruising

Count of participants self reporting at any time throuhgout the study that they bruise more easily

Time frame: Month 36

Safety and Tolerability of ACTH: Swelling Ankles

Count of participants self reporting at any time throuhgout the study that have noticed swelling in their ankles

Time frame: Month 36

Safety and Tolerability of ACTH: Hair Loss

Count of participants self reporting at any time throuhgout the study that have noticed losing more hair

Time frame: Month 36

Safety and Tolerability of ACTH: Acne

Count of participants self reporting at any time throuhgout the study that have noticed in increase in acne

Time frame: Month 36

Safety and Tolerability of ACTH: Insomnia

Count of participants self reporting at any time throuhgout the study that they have been having trouble sleeping

Time frame: Month 36

Safety and Tolerability of ACTH: Mood Change

Count of participants self reporting at any time throuhgout the study that they have noticed changes in mood

Time frame: Month 36

Safety and Tolerability of ACTH: GI Upset

Count of participants self reporting at any time throuhgout the study that they have had upset stomach, indigestion, diarrhea, or bloating

Time frame: Month 36

Safety and Tolerability of ACTH: Fatigue

Count of participants self reporting at any time throuhgout the study that noticed feeling tired or worn out

Time frame: Month 36

Safety and Tolerability of ACTH: Unpleasant Taste

Count of participants self reporting at any time throuhgout the study noticing a metallic taste in their mouth

Time frame: Month 36

Safety and Tolerability of ACTH: Blurred Vision

Count of participants self reporting at any time throuhgout the study noticing any blurred vision

Time frame: Month 36

Safety and Tolerability of ACTH: Moon Facies

New physician assessed moon facies over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Skin Thinning

New physician assessed skin thinning over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Hirsuitism

New physician assessed hirsuitism over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Bruising

New physician assessed bruising over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Ankle Swelling

New physician assessed ankle swelling over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Hair Loss

Any new physician assessed hair loss over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Cataracts

New physician assessed cataracts over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Myopathy

New physician assessed myopathy over study duration

Time frame: Month 36

Safety and Tolerability of ACTH: Change in Hemoglobin A1c

Change in hemoglobin A1c

Time frame: Month 36

ACTH in Progressive Forms of MS

Overview

Conditions

Interventions

Eligibility

Locations (3)

Outcomes

Primary Outcomes

Secondary Outcomes