Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT03943290 · ClinicalTrials.gov registry record · Phase 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)

A Phase 2 study, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

Terminated
Registry status
Phase 2
Development phase
62
Enrollment target

NCT03943290: Clinical Trial Phase 2 study, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

NCT03943290 is a Phase 2 study that was terminated before completion, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA. The registered enrollment target is 62 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT03943290, a Phase 2 study, was terminated before completion, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

TERMINATED
Registry status
Phase 2
Development phase
62 participants
Enrollment target

Study 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.

Primary Outcome

The number of participants that had a least one Treatment Emergent Adverse Event during the Part 2 of the study. The pre-specified analysis for this outcome measure was for those participants in the part 2 cohorts of this study therefore, only data of part 2 portion of the study are reported.

Interventions

  • DRUG ACE-083

Trial Details

FieldValue
Enrollment Target 62 participants
Start Date 2019-05-10
Est. Completion 2020-03-11
Phase Phase 2

Why NCT03943290 stopped before completion

NCT03943290 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 62 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower).

The record links to 0 conditions, and to 1 intervention - of which ACE-083 is the first listed.

NCT03943290 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT03943290 about?

NCT03943290 is a clinical study titled "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)". 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...

What is the current status of trial NCT03943290?

This trial is currently terminated. It is a Phase 2 study. The enrollment target is 62 participants. The study started on 2019-05-10. Estimated completion is 2020-03-11.

What interventions are being tested in trial NCT03943290?

The interventions under investigation include: ACE-083 (DRUG).

Who is sponsoring clinical trial NCT03943290?

This trial is sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA, which has 14 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT03943290's enrollment target sits among peer trials

62 1016th of 2000 higher than 975 of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

Nationwide trials with similar profiles

Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.

Similar enrollment target

  • NCT03231150 · 62 participants · NA

    Plantar Fasciitis Randomized Clinical Control Trial

  • NCT03458676 · 62 participants · NA

    Radiologic Pathologic Correlation of Advanced MR Imaging to Guide the Biopsy of Cerebral Malignancies

  • NCT03596086 · 62 participants · Phase 1

    HSV-tk + Valacyclovir + SBRT + Chemotherapy for Recurrent GBM

  • NCT03603405 · 62 participants · Phase 1

    HSV-tk and XRT and Chemotherapy for Newly Diagnosed GBM

Similar registry start date

  • NCT05962346 · started 2026-12 · NA

    Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia

  • NCT07125183 · started 2026-12 · Phase 2

    Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma

  • NCT07292298 · started 2026-11 · Phase 2

    Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates

  • NCT04263285 · started 2026-10 · NA

    Treatment of Depression Post-SCI

Source: ClinicalTrials.gov NCT03943290, the US trial registry maintained by the National Library of Medicine. NCT03943290 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.