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NCT01588522

Compound 31543 (Calcitriol, USP) in Patients Receiving Taxane-based Chemotherapy Regimens for the Treatment of Chemotherapy-Induced Alopecia

Completed Phase 1 Results posted Last updated 28 May 2025
What this trial tests

Phase 1 trial testing Compound 31543 Calcitriol 5 μg/mL in Chemotherapy-Induced Alopecia in 28 participants. Completed in 16 February 2017.

Timeline
17 September 2012
Primary endpoint
16 February 2017
16 February 2017

Quick facts

Lead sponsorBPGbio
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designsequential
Maskingnone
Primary purposeprevention
Enrollment28
Start date17 September 2012
Primary completion16 February 2017
Estimated completion16 February 2017
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

BPGbio — full company profile →

Who can join

18 and older, any sex, with Chemotherapy-Induced Alopecia. 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.

Dose-limiting Toxicities (DLT) of Topical Compound 31543 Primary · 28 days

DLTs were graded according to the CTCAE v4.0. A DLT was defined as a clinically significant Grade 3 or 4 (severe or life-threatening, respectively) non-hematologic toxicity occurring during the first 28-day treatment cycle, and determined to be possibly, probably, or definitely related to BPM31543 and not the taxane-based regimen as best determined by investigators.

GroupValue95% CI
Compound 31543 Calcitriol - 5 μg/mL0
Compound 31543 Calcitriol - 10 μg/mL0
Compound 31543 Calcitriol - 20 μg/mL0
Compound 31543 Calcitriol - 40 μg/mL0
Compound 31543 Calcitriol - 60 μg/mL0
Compound 31543 Calcitriol - 80 μg/mL0
Pre-dose Concentrations of Compound 31543 Secondary · pre-dose on day 1 of Week 1

Pre-dose concentrations of Compound 31543 from serum samples collected on Week 1 Day 1.

GroupValue95% CI
Compound 31543 Calcitriol - 5 ug/mL49.0± 5.8
Compound 31543 Calcitriol - 10 ug/mL43.2± 14.8
Compound 31543 Calcitriol - 20 ug/mL54.3± 31.5
Compound 31543 Calcitriol - 40 ug/mL58.4± 19.4
Compound 31543 Calcitriol - 60 ug/mL32.8± 5.2
Compound 31543 Calcitriol - 80 ug/mL37.8± 15.5
Cmax of Compound 31543 on Week 1 Day 1 Secondary · 8 hours

Cmax of Compound 31543 was calculated using serum samples collected on Week 1 Day 1 pre-dose and at 2 ± 0.5 hours, 4 ± 0.5 hours, and 8 ± 1 hours post-dose. The 8-hour Week 1 Day 1 sample was collected before the second of the daily doses.

GroupValue95% CI
5 μg/mL51.6± 6.9
10 μg/mL47.2± 13.6
20 μg/mL42.5± 8.5
40 μg/mL81.0± 8.1
60 μg/mL36.0± 9.8
80 μg/mL53.5± 16.1
AUC0-t of Compound 31543 on Week 1 Day 1 Secondary · 8 hours

Area under the curve (AUC0-t) of Compound 31543 was calculated from serum samples collected on Week 1 Day 1 pre-dose and at 2 ± 0.5 hours, 4 ± 0.5 hours, and 8 ± 1 hours post-dose. The 8-hour Week 1 Day 1 sample was collected before the second of the daily doses.

GroupValue95% CI
Compound 31543 Calcitriol - 5 ug/mL346± 10
Compound 31543 Calcitriol - 10 ug/mL334± 163
Compound 31543 Calcitriol - 20 ug/mL259± 60
Compound 31543 Calcitriol - 40 ug/mL530± 165
Compound 31543 Calcitriol - 60 ug/mL230± 87
Compound 31543 Calcitriol - 80 ug/mL370± 99
Top View Photographic Review Score Secondary · 7 and 15 weeks

Top of head/scalp view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-1± 0
10 μg/mL-1.5± 0.71
20 μg/mL-1
40 μg/mL-3± 0
60 μg/mL-1± 0
80 μg/mL-2± 1
Week 15
GroupValue95% CI
5 μg/mL-2
20 μg/mL-1
40 μg/mL-3
60 μg/mL-2± 1.41
80 μg/mL-3± 0
Right Side Photographic Review Score Secondary · 7 and 15 weeks

Right side of head/scalp view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-1± 0
10 μg/mL-2
20 μg/mL-1± 0
40 μg/mL-2.3± 1.15
60 μg/mL-1± 0
80 μg/mL-1.7± 0.58
Week 15
GroupValue95% CI
5 μg/mL-2
20 μg/mL-1.5± 0.71
40 μg/mL-3
60 μg/mL-2± 1.41
80 μg/mL-2.5± .71
Left Side Photographic Review Score Secondary · 7 and 15 weeks

Left side of head/scalp view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-0.5± 0.71
10 μg/mL-1± 0
20 μg/mL-1± 0
40 μg/mL-2.3± 1.15
60 μg/mL-1± 0
80 μg/mL-1.7± 0.58
Week 15
GroupValue95% CI
5 μg/mL-2
20 μg/mL-1.5± 0.71
40 μg/mL-3
60 μg/mL-2± 1.41
80 μg/mL-2.5± 0.71
Front Photographic Review Score Secondary · 7 and 15 weeks

Front of head/face view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-0.5± 0.71
10 μg/mL-0.5± 0.71
20 μg/mL-1± 0
40 μg/mL-1.7± 0.58
60 μg/mL-1± 0
80 μg/mL-2.3± 0.58
Week 15
GroupValue95% CI
5 μg/mL-2
20 μg/mL-1.5± 0.71
40 μg/mL-2
60 μg/mL-2± 1.41
80 μg/mL-2.5± 0.71
Back Photographic Review Score Secondary · 7 and 15 weeks

Back of head/face view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-0.5± 0.71
10 μg/mL-0.5± 0.71
20 μg/mL-1± 0
40 μg/mL-2± 1
60 μg/mL-1± 0
80 μg/mL-2± 1
Week 15
GroupValue95% CI
5 μg/mL-2
20 μg/mL-1± 0
40 μg/mL-3
60 μg/mL-2± 1.41
80 μg/mL-2.5± 0.71
Superior (Mid Pattern) Photographic Review Score Secondary · 7 and 15 weeks

Mid-pattern of the scalp from a superior view at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
5 μg/mL-1
10 μg/mL-1± 0
20 μg/mL-1± 0
40 μg/mL-1.7± 0.58
60 μg/mL-0.5± 0.71
80 μg/mL-1.7± 0.58
Week 15
GroupValue95% CI
20 μg/mL-1± 0
40 μg/mL-2
60 μg/mL-2± 1.41
80 μg/mL-2.5± 0.71
Vertex Photographic Review Score Secondary · 7 and 15 weeks

Vertex view with hair parted in the center at 7 and 15 weeks were compared to baseline images and scored on a 7-point evaluation scale: -3 = \>75% great hair loss; -2 = 50-75% moderate hair loss; -1 = 25-49% slight hair loss; 0 = 0-24% hair loss/gain, 1 = 25-49% slight hair gain; 2 = 50-75% moderate hair gain; 3 = \>75% greatly increased/hair gain.

Week 7
GroupValue95% CI
20 μg/mL-1± 0
40 μg/mL-1.7± 0.58
60 μg/mL-0.5± 0.71
80 μg/mL-1.7± 0.58
Week 15
GroupValue95% CI
20 μg/mL-1± 0
40 μg/mL-2
60 μg/mL-1.5± 2.12
80 μg/mL-2.5± 0.71

Adverse events — posted to ClinicalTrials.gov

Time frame: 2 weeks (or 7 days ± 2 days if the patient was unable or unwilling to undergo 2 weeks of pre-treatment) prior to the first dose of chemotherapy and continued the application twice daily until termination of chemotherapy treatment (up to a maximum of 54 weeks). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Compound 31543 Calcitriol - 5 μg/mL
Serious: 0/3 (0%)
Deaths: 0/3
Compound 31543 Calcitriol - 10 μg/mL
Serious: 1/3 (33%)
Deaths: 0/3
Compound 31543 Calcitriol - 20 μg/mL
Serious: 1/6 (17%)
Deaths: 0/6
Compound 31543 Calcitriol - 40 μg/mL
Serious: 4/4 (100%)
Deaths: 0/4
Compound 31543 Calcitriol - 60 μg/mL
Serious: 1/4 (25%)
Deaths: 0/4
Compound 31543 Calcitriol - 80 μg/mL
Serious: 2/3 (67%)
Deaths: 0/3

Serious adverse events (26 terms)

ReactionSystemCompound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …
Abdominal PainGastrointestinal disorders
Supraventicular TachycardiaCardiac disorders
Muscular WeaknessMusculoskeletal and connective tissue disorders
PainGeneral disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
AlkalosisMetabolism and nutrition disorders
HypokalaemiaMetabolism and nutrition disorders
HypomagnesaemiaMetabolism and nutrition disorders
PyrexiaGeneral disorders
Flank PainMusculoskeletal and connective tissue disorders
Bone PainMusculoskeletal and connective tissue disorders
PollakiuriaRenal and urinary disorders
ConstipationGastrointestinal disorders
Large Intestinal ObstructionGastrointestinal disorders
DehyrdationMetabolism and nutrition disorders
SyncopeNervous system disorders
DiarrhoeaGastrointestinal disorders
Chest PainCardiac disorders
FatigueGeneral disorders
ArthralgiaMusculoskeletal and connective tissue disorders
InsomniaPsychiatric disorders
CoughRespiratory, thoracic and mediastinal disorders
Oropharyngeal PainRespiratory, thoracic and mediastinal disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
Other adverse events (22 terms — click to expand)

ReactionSystemCompound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …Compound 31543 Calcitriol …
AlopeciaSkin and subcutaneous tissue disorders
Pain of SkinSkin and subcutaneous tissue disorders
FatigueGeneral disorders
Rash Maculo-papularSkin and subcutaneous tissue disorders
Dry SkinSkin and subcutaneous tissue disorders
Dermatitis AcneiformSkin and subcutaneous tissue disorders
Abdominal PainGeneral disorders
ConstipationGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
PainGeneral disorders
Blood Alkaline Phosphate IncreasedInvestigations
Vitamin D IncreasedInvestigations
Pain in ExtremityMusculoskeletal and connective tissue disorders
Peripheral Sensory NeuropathyNervous system disorders
InsomniaPsychiatric disorders
CoughRespiratory, thoracic and mediastinal disorders
Oedema PeripheralGeneral disorders
ArthralgiaMusculoskeletal and connective tissue disorders
AnxietyPsychiatric disorders
HypertensionVascular disorders

Most-reported serious reactions: Abdominal Pain, Supraventicular Tachycardia, Muscular Weakness, Pain, Nausea, Vomiting, Alkalosis, Hypokalaemia.

Data from ClinicalTrials.gov NCT01588522 adverse events section.

Sponsor's own description

The goal of this clinical trial is to learn if Compound 31543 (topical calcitriol) for treatment of hair-loss in patients with a diagnosis of breast cancer, gynecologic cancer or sarcomas receiving a taxane-based chemotherapy is safe. The main questions it aims to answer are: * What medical problems do participants have when using compound 31543? * How much Compound 31543 is in blood after treatment? * How much hair is lost during treatment? Participants will: * Apply Compound 31543 to their scalp twice a day, starting at least 5 days before starting chemotherapy for at least 3 months or until chemotherapy treatment is completed. * Have blood drawn on weeks 1, 5, 9, 13, and 54. * Have photographs taken of their hair before starting and at weeks 7, 15, 27, and 54. * Keep a diary of the condition of their hair.

Publications & conference data

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

  1. Prevention and Treatment of Chemotherapy-Induced Alopecia: What Is Available and What Is Coming?
    Wikramanayake TC, Haberland NI, Akhundlu A, Laboy Nieves A, et al · · 2023 · cited 37× · PMID 37185388 · DOI 10.3390/curroncol30040275
  2. The future of pharmaceuticals: Artificial intelligence in drug discovery and development.
    Fu C, Chen Q. · · 2025 · cited 18× · PMID 40893437 · DOI 10.1016/j.jpha.2025.101248
  3. Dickkopf Proteins and Their Role in Cancer: A Family of Wnt Antagonists with a Dual Role.
    Giralt I, Gallo-Oller G, Navarro N, Zarzosa P, et al · · 2021 · cited 15× · PMID 34451907 · DOI 10.3390/ph14080810
  4. A New Era of Artificial Intelligence (AI): Transforming Drug Discovery and Development.
    Ali S, Tian X, Chen H, Zhou J. · · 2025 · cited 7× · PMID 41306069 · DOI 10.1021/acs.jmedchem.5c03159
  5. A phase I safety study of topical calcitriol (BPM31543) for the prevention of chemotherapy-induced alopecia.
    Lacouture ME, Dion H, Ravipaty S, Jimenez JJ, et al · · 2021 · cited 7× · PMID 33206291 · DOI 10.1007/s10549-020-06005-6

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