# In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

> **NCT02986698** · PHASE1 · TERMINATED · sponsor: **University of California, San Francisco** · enrollment: 6 (actual)

## Conditions studied

- Alpha Thalassemia Major
- Hemoglobinopathy; With Thalassemia
- Hemoglobinopathies
- Fetal Anemia
- Fetal Hydrops
- Alpha; Thalassemia
- Thalassemia Major
- Thalassemia Alpha
- A-Thalassemia

## Interventions

- **BIOLOGICAL:** in utero hematopoietic stem cell transplantation

## Key facts

- **NCT ID:** NCT02986698
- **Lead sponsor:** University of California, San Francisco
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2017-10-05
- **Primary completion:** 2024-04-30
- **Final completion:** 2024-12-31
- **Target enrollment:** 6 (ACTUAL)
- **Why stopped:** Enrollment ended early due to interim data suggesting a lack of efficacy. Interim data did demonstrate that IUHSCT was safe and well tolerated, but the PI and DSMB agreed that enrollment should not continue under the current treatment protocol.
- **Last updated:** 2025-01-27

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02986698

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02986698, "In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)". Retrieved via AI Analytics 2026-06-09 from https://api.ai-analytics.org/clinical/NCT02986698. Licensed CC0.

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