# Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

> **NCT04232085** · PHASE2 · RECRUITING · sponsor: **Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins** · enrollment: 27 (estimated)

## Conditions studied

- Primary Immune Deficiency Disorder
- Immune Deficiency Disease
- Bone Marrow Failure
- Short Telomere Length
- Fanconi Anemia
- Non Fanconi DNA-DSB Repair Disorder
- Hoyeraal-Hreidarsson Syndrome
- Dyskeratosis Congenita
- Telomere Biology Disorder
- Short Telomere Syndrome

## Interventions

- **DRUG:** Alemtuzumab
- **DRUG:** Fludarabine
- **DRUG:** Melphalan
- **RADIATION:** Low Dose Total Body Irradiation
- **DRUG:** Cyclophosphamide
- **DRUG:** Tacrolimus
- **DRUG:** Mycophenolate Mofetil

## Key facts

- **NCT ID:** NCT04232085
- **Lead sponsor:** Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2020-02-12
- **Primary completion:** 2027-12-31
- **Final completion:** 2028-12-31
- **Target enrollment:** 27 (ESTIMATED)
- **Last updated:** 2025-11-28

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04232085

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04232085, "Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures". Retrieved via AI Analytics 2026-06-30 from https://api.ai-analytics.org/clinical/NCT04232085. Licensed CC0.

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