We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 21 of 87 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02927080
Recruitment Status : Recruiting
First Posted : October 6, 2016
Last Update Posted : October 24, 2017
Sponsor:
Information provided by (Responsible Party):

October 5, 2016
October 6, 2016
October 24, 2017
November 2016
January 2019   (Final data collection date for primary outcome measure)
  • Part 1: Safety and Tolerability (adverse events) [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141). ]
  • Part 2: Percent change from baseline in muscle volume of injected muscle by MRI [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141). ]
Same as current
Complete list of historical versions of study NCT02927080 on ClinicalTrials.gov Archive Site
  • Estimation of systemic exposure to ACE-083 by quantitative LC-MS assay of serum samples following local intramuscular administration [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141) ]
  • Percent change from baseline in strength of injected muscle by quantitative muscle testing [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]
  • Percent change from baseline in function of injected muscle by motor function tests [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]
  • Change from baseline in Health-Related-Quality-of-Life (HRQoL) by FSHD-Health Index questionnaire [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]
Same as current
Not Provided
Not Provided
 
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy
Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

Part 1 (dose escalation, open-label) Part 1 will consist of up to 6 cohorts (A to F) of patients and will evaluate multiple ascending dose levels of ACE-083 in either the tibialis anterior (TA) or biceps brachii (BB) muscle. Patients in each cohort will be enrolled in a 4-week screening period before beginning treatment. A Safety Review Team (SRT) will meet to review data for each cohort when at least 4 patients within a cohort have completed their Day 43 visit prior to dose escalation.

Part 2 (randomized, double-blind, placebo-controlled) Prior to the initiation of Part 2, a review of safety and efficacy data from Part 1 will be conducted to determine whether cohorts for one or both muscles will be pursued in Part 2, as well as the recommended dose level for each muscle. A total of up to 40 new patients (20 patients per muscle) may be enrolled and randomized (3:2) to receive either ACE-083 (n=12) or placebo (n=8) unilaterally or bilaterally (if both sides are affected per inclusion criteria) to either the TA or BB muscles (but not both). The SRT will meet to review Part 2 patient safety data after the first 10 patients reach Day 43 in the study and approximately every 3 months thereafter.

Study duration for Parts 1 and 2 for each patient will be approximately 24 weeks, including a 4-week screening period, a 12-week treatment period, and an 8-week follow-up period after the last dose

Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Facioscapulohumeral Muscular Dystrophy
  • Drug: ACE-083
    Recombinant fusion protein.
  • Drug: ACE-083 or placebo
    Recombinant fusion protein or buffer solution.
  • Experimental: ACE-083 (Part 1, Cohort 1)
    ACE-083 150 mg IM, once every 3 weeks for up to 5 doses.
    Intervention: Drug: ACE-083
  • Experimental: ACE-083 (Part 1, Cohort 2)
    ACE-083 200 mg IM, once every 3 weeks for up to 5 doses.
    Intervention: Drug: ACE-083
  • Experimental: ACE-083 (Part 1, Cohort 3)
    ACE-083 up to 250 mg IM, once every 3 weeks for up to 5 doses.
    Intervention: Drug: ACE-083
  • Experimental: ACE-083 or placebo (Part 2, Cohort 1)
    ACE-083 up to 250 mg IM (tibialis anterior muscle) or placebo, once every 3 weeks for up to 5 doses.
    Intervention: Drug: ACE-083 or placebo
  • Experimental: ACE-083 or placebo (Part 2, Cohort 2)
    ACE-083 up to 250 mg IM (biceps brachii muscle) or placebo, once every 3 weeks for up to 5 doses.
    Intervention: Drug: ACE-083 or placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
76
April 2019
January 2019   (Final data collection date for primary outcome measure)

Key Inclusion Criteria:

  1. Age ≥ 18 years
  2. Genetically-confirmed FSHD1 or FSHD2 (or a first-degree relative with genetically confirmed FSHD1 or FSHD2) and clinical findings meeting FSHD criteria
  3. For tibialis anterior (TA) cohorts:

    1. Able to walk independently for at least 10 meters, without a brace
    2. Mild to moderate weakness in left and/or right ankle dorsiflexion

    Note: Opposite side must not be severely affected.

    For biceps brachii (BB) cohorts: mild to moderate weakness in left and/or right elbow flexion.

  4. Females of childbearing potential must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods during study participation. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study even if he has undergone a successful vasectomy.
  5. Ability to adhere to the study visit schedule/procedures, and to understand and comply with protocol requirements
  6. Signed written informed consent

Key Exclusion Criteria:

  1. History of active malignancy, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
  2. Symptomatic cardiopulmonary disease, significant functional impairment, or other co morbidities that in the opinion of the investigator would limit a patient's ability to complete strength and/or functional assessments on study
  3. Renal impairment (serum creatinine ≥ 2 times the upper limit of normal [ULN])
  4. Aspartate transaminase (AST) and/or alanine transaminase (ALT) ≥ 3 times ULN
  5. Increased risk of bleeding (i.e., due to hemophilia, platelet disorders, or use of any anticoagulation/platelet modifying therapies up to 2 weeks prior to Study Day 1; low dose aspirin [≤ 100 mg daily] is permitted)
  6. Major surgery within 4 weeks prior to Study Day 1
  7. Systemic corticosteroids within 2 weeks before Study Day 1 and for duration of study; inhaled therapeutic or physiologic doses of corticosteroids are permitted
  8. Any change in medications potentially affecting muscle function within 4 weeks of Study Day 1 and for duration of study
  9. Previous exposure to any marketed or investigational agent potentially affecting muscle volume, strength, or function within 5 half-lives of last dose or 4 weeks of Study Day 1 if half-life is unknown, or any prior exposure to ACE-083
  10. Significant change in physical activity or exercise (e.g., significant increase or decrease in intensity or frequency) within 8 weeks before Study Day 1 or inability to maintain the baseline level of physical activity throughout the study
  11. Any condition that would prevent MRI scanning or compromise the ability to obtain a clear and interpretable scan of the TA or BB muscles, as applicable (e.g., pacemaker, knee/hip replacement, or metallic implants)
  12. Known active substance abuse, including alcohol
  13. History of sensitivity to protein pharmaceuticals
  14. Female that is lactating/breast-feeding
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact: Clinical Trial Manager clinicaltrials083@acceleronpharma.com
Canada,   United States
 
 
NCT02927080
A083-02
ACE-083 ( Other Identifier: Acceleron Pharma Inc. )
Yes
Not Provided
Not Provided
Acceleron Pharma, Inc.
Acceleron Pharma, Inc.
Not Provided
Not Provided
Acceleron Pharma, Inc.
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP