TerminatedPhase 2

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Stopped: Investigation of ACE-083 for use in patients with CMT and FSHD is being discontinued in this extension study as functional secondary endpoints were not achieved in the A083-023 or A083-03 trial (the parent trials).

United States62 participantsStarted 2019-05-10

Plain-language summary

This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX previously enrolled in Study A083-03. This study will be conducted in two Parts: Part 1, which is a loading phase of 6 months' duration, and Part 2, the maintenance phase, which will last up to 24 months.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Completion of treatment with study drug per protocol and completion of the end of treatment (ET) visit in Study A083-02 or Study A083-03.
✓. Females of childbearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy or are not naturally postmenopausal ≥ 24 consecutive months) must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation and for 8 weeks following the last dose of ACE-083. Hormonal birth control use must be stable for at least 14 days prior to Day 1. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study and for 8 weeks following the last dose of ACE-083, even if they have undergone a vasectomy. Subjects must be counseled about contraception prior to the first dose of ACE-083 and every three months thereafter during the study.
✓. Ability to adhere to the study visit schedule/procedures and to understand and comply with protocol requirements
✓. Signed written informed consent

Exclusion criteria

✕. Current/active malignancy (e.g., remission less than 5 years' duration), with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin

What they're measuring

Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2

Timeframe: From baseline to end of participation of the Part 2 portion of the study

Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study.

Timeframe: From baseline to the end of the part 2 portion of the study

Part 2: Change in Total Muscle Volume - Percent Change From Baseline to Day 113 in Total Muscle Volume of Injected Muscle by Magnetic Resonance Imaging (MRI) During the Part 2 Portion

Timeframe: During the Part 2 portion of the study: Baseline to Day 113

Trial details

NCT IDNCT03943290

SponsorAcceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-03-11

Results submitted2021-03-11

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