NCT01474681 is a Phase 1, PHASE2 clinical trial of HSC835 in Acute Myelocytic Leukemia, sponsored by Novartis Pharmaceuticals.

Who is sponsoring NCT01474681?

NCT01474681 is sponsored by Novartis Pharmaceuticals.

What drugs are being tested in NCT01474681?

Interventions in NCT01474681: HSC835.

What condition does NCT01474681 study?

NCT01474681 studies Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, Marginal Zone Lymphoma, Follicular Lymphomas, Large-cell Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, High Grade Lymphomas, Mantle-cell Lymphoma, Lymphoplasmacytic Lymphoma.

Is NCT01474681 still recruiting?

NCT01474681 is currently completed. Last updated 30 December 2020.

Are results published for NCT01474681?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2020-12-30 · How we verify

NCT01474681

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Completed Phase 1, PHASE2 Results posted Last updated 30 December 2020

What this trial tests

Phase 1, PHASE2 trial testing HSC835 in Acute Myelocytic Leukemia in 27 participants. Completed in 3 October 2016.

Timeline

9 January 2012

Primary endpoint
3 October 2016

3 October 2016

Quick facts

Lead sponsor	Novartis Pharmaceuticals
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	27
Start date	9 January 2012
Primary completion	3 October 2016
Estimated completion	3 October 2016
Sites	1 location across United States

Drugs / interventions tested

HSC835 — full drug profile →

Conditions studied

Acute Myelocytic Leukemia — all drugs for Acute Myelocytic Leukemia →
Acute Lymphocytic Leukemia — all drugs for Acute Lymphocytic Leukemia →
Chronic Myelogenous Leukemia — all drugs for Chronic Myelogenous Leukemia →
Myelodysplastic Syndrome — all drugs for Myelodysplastic Syndrome →

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 10 to 55, any sex, with Acute Myelocytic Leukemia or Acute Lymphocytic Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT. Primary · 32 days

The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others.

Infusional Toxicity 48 hrs after transplant

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	0
DUCBT < 18 Yrs	0
DUCBT >= 18 Yrs	1

Absence of graft failure after 32 days

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	0
DUCBT < 18 Yrs	0
DUCBT >= 18 Yrs	0

Incidence of Neutrophil Recovery Within 42 Days Secondary · 42 days

Neutrophil recovery (engraftment) is defined as the first of three consecutive days with ANC \> 0.5 x 109/L which occurred for all patients before 42 days post transplant.

Group	Value	95% CI
SUCBT < 18 Yrs	1
SUCBT >= 18 Yrs	8
DUCBT < 18 Yrs	3
DUCBT >= 18 Yrs	15

Incidence of Platelet Recovery Within Six Months Secondary · 6 months

Incidence of platelet recovery within six months. Number of participants recovering platelet to ≥50,000 × 109/L for at least one week without transfusion in the prior 7 days to the first measurement.

Group	Value	95% CI
SUCBT < 18 Yrs	1
SUCBT >= 18 Yrs	6
DUCBT < 18 Yrs	2
DUCBT >= 18 Yrs	11

Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only) Secondary · Day 100

Frequency of expanded unit predominance at day 100 (DUCBT recipients only) unit predominance was assessed by differences in microsatellite patterns between the recipient, HSC835 and the unmanipulated cord blood unit. Evaluation of sorted CD15-positive/CD33-positive myeloid and CD3-positive T cells in the peripheral blood, revealed three patterns: predominance of HSC835, Mixed dominance an unique chimerism pattern was observed with the CD15/CD33 population predominantly derived from HSC835 and the CD3 population almost exclusively derived from the unmanipulated unit, and predominance of the unm

Predominance of HSC835

Group	Value	95% CI
DUCBT < 18 Yrs	0
DUCBT >= 18 Yrs	6

Mixed dominance

Group	Value	95% CI
DUCBT < 18 Yrs	3
DUCBT >= 18 Yrs	3

Predominance of the unmaninpulated unit

Group	Value	95% CI
DUCBT < 18 Yrs	0
DUCBT >= 18 Yrs	6

Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year Secondary · Day 100 and Month 12

Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year

Day 100

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	1
DUCBT < 18 Yrs	1
DUCBT >= 18 Yrs	3

Month 12

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	1
DUCBT < 18 Yrs	1
DUCBT >= 18 Yrs	6

Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year Secondary · Day 100 and Monnth 12

Number of participants with incidence of Acute Graft Versus Host Disease (aGVHD) within 100 days and Chronic Graft Versus Host Disease (cGVHD) within 1 year

aGVHD-Day 100

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	5
DUCBT < 18 Yrs	3
DUCBT >= 18 Yrs	8

cGVHD-Month12

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	1
DUCBT < 18 Yrs	0
DUCBT >= 18 Yrs	2

Incidence of Relapse Within One Year Secondary · Month 12

Number of participants with Incidence of relapse within one year

Group	Value	95% CI
SUCBT < 18 Yrs	0
SUCBT >= 18 Yrs	2
DUCBT < 18 Yrs	1
DUCBT >= 18 Yrs	0

Overall Survival (OS) Within One Year Secondary · Month 12

Number of participants with Overall survival (OS) within one year

Group	Value	95% CI
SUCBT < 18 Yrs	1
SUCBT >= 18 Yrs	5
DUCBT < 18 Yrs	1
DUCBT >= 18 Yrs	9

Disease Free Survival (DFS) Within One Year Secondary · Month 12

Number of participants with Disease Free Survival (DFS) within one year. Patients are considered to have achieved DFS or relapse-free survival if they had not experienced either relapse or death (of any cause)

Group	Value	95% CI
SUCBT < 18 Yrs	1
SUCBT >= 18 Yrs	5
DUCBT < 18 Yrs	1
DUCBT >= 18 Yrs	9

Adverse events — posted to ClinicalTrials.gov

Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

SUCBT < 18 Yrs

Serious: 1/1 (100%)

Deaths: —

SUCBT >= 18 Yrs

Serious: 7/8 (88%)

Deaths: —

DUCBT < 18 Yrs

Serious: 3/3 (100%)

Deaths: —

DUCBT >= 18 Yrs

Serious: 14/15 (93%)

Deaths: —

SUCBT

Serious: 8/9 (89%)

Deaths: —

DUCBT

Serious: 17/18 (94%)

Deaths: —

Serious adverse events (53 terms)

Reaction	System	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs	SUCBT	DUCBT
Acute graft versus host disease	Immune system disorders	—	—	—	—	—	—
Acute kidney injury	Renal and urinary disorders	—	—	—	—	—	—
Pulmonary alveolar haemorrhage	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Engraftment syndrome	Immune system disorders	—	—	—	—	—	—
Human herpesvirus 6 infection	Infections and infestations	—	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—	—
Pneumonia cytomegaloviral	Infections and infestations	—	—	—	—	—	—
Acute respiratory distress syndrome	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Chronic graft versus host disease	Immune system disorders	—	—	—	—	—	—
BK virus infection	Infections and infestations	—	—	—	—	—	—
Cytomegalovirus viraemia	Infections and infestations	—	—	—	—	—	—
Enterococcal bacteraemia	Infections and infestations	—	—	—	—	—	—
Influenza	Infections and infestations	—	—	—	—	—	—
Pneumonia fungal	Infections and infestations	—	—	—	—	—	—
Staphylococcal bacteraemia	Infections and infestations	—	—	—	—	—	—
Staphylococcal sepsis	Infections and infestations	—	—	—	—	—	—
Acute myeloid leukaemia recurrent	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Thrombotic thrombocytopenic purpura	Blood and lymphatic system disorders	—	—	—	—	—	—
Gastrointestinal haemorrhage	Gastrointestinal disorders	—	—	—	—	—	—
Impaired gastric emptying	Gastrointestinal disorders	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Cholecystitis	Hepatobiliary disorders	—	—	—	—	—	—
Acute graft versus host disease in skin	Immune system disorders	—	—	—	—	—	—
Adenovirus infection	Infections and infestations	—	—	—	—	—	—

Other adverse events (81 terms — click to expand)

Reaction	System	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs	SUCBT	DUCBT
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—	—	—
Stomatitis	Gastrointestinal disorders	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Staphylococcal bacteraemia	Infections and infestations	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—
Fluid overload	Metabolism and nutrition disorders	—	—	—	—	—	—
Chills	General disorders	—	—	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
BK virus infection	Infections and infestations	—	—	—	—	—	—
Cytomegalovirus viraemia	Infections and infestations	—	—	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Clostridium difficile infection	Infections and infestations	—	—	—	—	—	—
Human herpesvirus 6 infection	Infections and infestations	—	—	—	—	—	—
Hyperglycaemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Sinus tachycardia	Cardiac disorders	—	—	—	—	—	—
Tachycardia	Cardiac disorders	—	—	—	—	—	—
Face oedema	General disorders	—	—	—	—	—	—
Pain	General disorders	—	—	—	—	—	—
Adenovirus infection	Infections and infestations	—	—	—	—	—	—
Enterococcal bacteraemia	Infections and infestations	—	—	—	—	—	—
Enterococcal infection	Infections and infestations	—	—	—	—	—	—
Pneumonia fungal	Infections and infestations	—	—	—	—	—	—
Staphylococcal infection	Infections and infestations	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Neuropathy peripheral	Nervous system disorders	—	—	—	—	—	—
Anxiety	Psychiatric disorders	—	—	—	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Hiccups	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—
Atrial fibrillation	Cardiac disorders	—	—	—	—	—	—
Eye pain	Eye disorders	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Dyspepsia	Gastrointestinal disorders	—	—	—	—	—	—

Most-reported serious reactions: Acute graft versus host disease, Acute kidney injury, Pulmonary alveolar haemorrhage, Engraftment syndrome, Human herpesvirus 6 infection, Pneumonia, Pneumonia cytomegaloviral, Acute respiratory distress syndrome.

Data from ClinicalTrials.gov NCT01474681 adverse events section.

Sponsor's own description

This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Phase I/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft.
Wagner JE, Brunstein CG, Boitano AE, DeFor TE, et al · · 2016 · cited 260× · PMID 26669897 · DOI 10.1016/j.stem.2015.10.004
Advances in umbilical cord blood manipulation-from niche to bedside.
Lund TC, Boitano AE, Delaney CS, Shpall EJ, et al · · 2015 · cited 60× · PMID 25511187 · DOI 10.1038/nrclinonc.2014.215
Optimizing cord blood selection.
Ruggeri A. · · 2019 · cited 21× · PMID 31808851 · DOI 10.1182/hematology.2019000056
Development and clinical advancement of small molecules for <i>ex vivo</i> expansion of hematopoietic stem cell.
Li J, Wang X, Ding J, Zhu Y, et al · · 2022 · cited 15× · PMID 35755294 · DOI 10.1016/j.apsb.2021.12.006
Clinical Progress and Preclinical Insights Into Umbilical Cord Blood Transplantation Improvement.
Sun Z, Yao B, Xie H, Su X. · · 2022 · cited 9× · PMID 35972332 · DOI 10.1093/stcltm/szac056
How to Improve Cord Blood Engraftment?
Beksac M, Yurdakul P. · · 2016 · cited 9× · PMID 26925402 · DOI 10.3389/fmed.2016.00007
Umbilical cord blood derived cell expansion: a potential neuroprotective therapy.
Penny TR, Jenkin G, Miller SL, McDonald CA. · · 2024 · cited 8× · PMID 39075614 · DOI 10.1186/s13287-024-03830-0
Expansion of Haematopoietic Stem and Progenitor Cells: Paving the Way for Next-Generation Haematopoietic Stem Cell Transplantation.
Bari S, Chong P, Hwang WYK. · · 2019 · PMID 37588101 · DOI 10.31547/bct-2019-004

Verify or expand the search:

Other trials of HSC835

Trials testing the same drug.

NCT01930162 — Safety and Tolerability of HSC835 in Patients With Hematological Malignancies Undergoing Single Umbilical Cord Blood Tra · Phase 2 · completed

Other recruiting trials for Acute Myelocytic Leukemia

Currently open trials in the same condition.

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Other Novartis Pharmaceuticals trials

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Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01474681 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 30 December 2020

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01474681.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing

NCT01474681

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Serious adverse events (53 terms)

Sponsor's own description

Publications & conference data

Related trials

Other trials of HSC835

Other recruiting trials for Acute Myelocytic Leukemia

Other Novartis Pharmaceuticals trials

Verify against primary sources