# VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

> **NCT02898753** · PHASE1,PHASE2 · TERMINATED · sponsor: **Valerion Therapeutics, LLC** · enrollment: 12 (actual)

## Conditions studied

- Pompe Disease

## Interventions

- **DRUG:** VAL-1221
- **DRUG:** RhGAA

## Key facts

- **NCT ID:** NCT02898753
- **Lead sponsor:** Valerion Therapeutics, LLC
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2017-06-21
- **Primary completion:** 2020-03-25
- **Final completion:** 2020-03-25
- **Target enrollment:** 12 (ACTUAL)
- **Why stopped:** Company closure due to lack of funding
- **Last updated:** 2020-06-02


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02898753

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02898753, "VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT02898753. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*