# A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

> **NCT04525352** · PHASE1 · TERMINATED · sponsor: **Rocket Pharmaceuticals Inc.** · enrollment: 1 (actual)

## Conditions studied

- Infantile Malignant Osteopetrosis

## Interventions

- **BIOLOGICAL:** RP-L401

## Key facts

- **NCT ID:** NCT04525352
- **Lead sponsor:** Rocket Pharmaceuticals Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2020-11-19
- **Primary completion:** 2021-05-21
- **Final completion:** 2021-05-21
- **Target enrollment:** 1 (ACTUAL)
- **Why stopped:** Study discontinued due to feasibility.
- **Last updated:** 2022-07-13

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04525352

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04525352, "A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis". Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/clinical/NCT04525352. Licensed CC0.

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