Spinal and Bulbar Muscular AtrophyPhase 2NCT02024932

Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

A Phase 2 study testing BVS857 in people with Spinal and Bulbar Muscular Atrophy, run by Novartis Pharmaceuticals at 6 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA). Read more about this study

Who is it for?: Ages 18 years and older, men only
Conditions the study is for: Spinal and Bulbar Muscular Atrophy
What is being tested?: BVS857
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 37 participants
Where: 6 sitesin 4 countries
Study dates: 4 Feb 2014to 13 Apr 2016
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

Genetic diagnosis of SBMA with symptomatic muscle weakness
Able to complete 2 minute timed walk
Serum IGF-1 level less than or equal to 170 ng/mL

Who cannot join

Medically treated diabetes mellitus or known history of hypoglycemia
History of Bell's palsy
Treatment with systemic steroids > 10 mg/day (or equivalent dose); androgens or androgen reducing agents; systemic beta agonists; or other muscle anabolic drugs within the previous 3 months
History of cancer, other than non-melanomatous skin cancer
Retinopathy
Papilledema Other protocol defined inclusion/exclusion criteria may apply

Trial locations and contacts

6 sites in 4 countries.

Country

City, postcode or hospital

6 sites in 4 countries

United States3 sites

National Institutes of Health
Bethesda, Maryland, 20892
Novartis Investigative Site
Columbus, Ohio, 43210
Novartis Investigative Site
Orange, California, 92868

Other trials in United States

Denmark1 site

Novartis Investigative Site
Copenhagen, 2100

Other trials in Denmark

Germany1 site

Novartis Investigative Site
Ulm, 89081

Other trials in Germany

Italy1 site

Novartis Investigative Site
Padova, PD, 35128

Other trials in Italy

About this study

Official title: A Two-part Placebo-controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).

Conditions: Spinal and Bulbar Muscular Atrophy
Being tested: BVS857, Placebo
Medical subject headings: Bulbo-Spinal Atrophy, X-Linked

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BVS857Drug
BVS857 lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..
PlaceboDrug
Placebo lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..

Study groups in NCT02024932
Study group	Role	What they receive
BVS857 Part A Open label (Cohort 1)	Experimental	Participants received single doses of 0.01 mg/kg BVS857 intravenously (i.v.) on day 1, 0.01 mg/kg BVS857 subcutaneously (s.c.) on day 15, 0.03 mg/kg BVS857 s.c. on day 29, 0.06 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. Receives: Drug: BVS857
BVS857 Part A double blind (Cohort 2)	Experimental	Participants received single doses of 0.03 mg/kg BVS857 i.v on day 1, 0.03 mg/kg BVS857 s.c. on day 15, 0.06 mg/kg BVS857 s.c. on day 29, 0.10 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. (BVS857 concentrations differed on days 43 and 57.) Receives: Drug: BVS857
Placebo Part A double blind (Cohort 2)	Placebo comparator	Participants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57. Receives: Drug: Placebo
BVS857 Part B open-label (Cohort 4)	Experimental	Participants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks. Receives: Drug: BVS857
BVS857 Part B double blind (Cohort 5)	Experimental	Participants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks. Receives: Drug: BVS857
Placebo Part B double blind (Cohort 5)	Placebo comparator	Participants received matching placebo i.v. to BVS857 weekly for 12 weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability
Safety was monitored throughout the study.
Measured: After 78 days in Part A and after 85 days in Part B.
Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability
Safety was monitored throughout the study.
Measured: After 78 days in Part A and after 85 days in Part B.
Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5
Thigh muscle volume was assessed by magnetic resonance imaging (MRI). Change from baseline was calculated from the ratio of the post-baseline mean value to the baseline mean value: [(Day 85/baseline) - 1)] x 100. A positive change from baseline indicates improvement.
Measured: Baseline, Day 85

Secondary outcomes (21)

Mean Change From Baseline in Score on the Adult Myopathy Assessment Tool (AMAT) in Part B, Cohort 5
The AMAT rated physical function and muscle endurance, with higher scores indicating better performance. The tool includes 7 timed functional tasks rated on a scale from 0 - 21 and 6 endurance tasks rated on a scale from 0 - 24. The range for the total score was from 0 (worst) to 45 (best). A positive change from baseline indicates improvement.
Measured: Baseline, Day 85
Mean Change From Baseline in Total Lean Body Mass (LBM) in Part B, Cohort 5
LBM was assessed by dual-energy X-ray (DXA) absorptiometry. A positive change from baseline indicate improvement.
Measured: Baseline, Day 85
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 1
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 2
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 1
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 2
Serum samples were obtained for PK assessment.
Measured: Day 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 1
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 2
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 1
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 2
Serum samples were obtained for PK assessment.
Measured: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 4
Serum samples were obtained for PK assessment.
Measured: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 5
Serum samples were obtained for PK assessment.
Measured: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 4
Serum samples were obtained for PK assessment.
Measured: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 5
Serum samples were obtained for PK assessment.
Measured: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part B, Cohort 5
Serum samples were obtained for PK assessment.
Measured: Day 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 4
Serum samples were obtained for the PK assessment.
Measured: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 5
Measured: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the End of the Dosing Interval Tau (AUCtau)
Serum samples were obtained for PK assessment.
Measured: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Terminal Elimination Half-life (T1/2)
Serum samples were obtained for PK assessment.
Measured: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to Infinity (AUCinf)
Serum samples were obtained for PK assessment.
Measured: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Compare Dose Normalized Log-transformed AUCinf Following IV and SC Administrations
Serum samples were obtained for PK assessment.
Measured: In Part A: days 1 and 15, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Feb 201413 Apr 2016

First posted31 Dec 2013 (estimated)
Study start4 Feb 2014
Primary completion13 Apr 2016
Study completion13 Apr 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Spinal and Bulbar Muscular AtrophyStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 37 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02024932
Sponsor's protocol ID: CBVS857X2202
Status last verified: Mar 2019
First submitted: 29 Dec 2013
Last updated: 5 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Grunseich C, Miller R, Swan T, Glass DJ, El Mouelhi M, Fornaro M, Petricoul O, Vostiar I, Roubenoff R, Meriggioli MN, Kokkinis A, Guber RD, Budron MS, Vissing J, Soraru G, Mozaffar T, Ludolph A, Kissel JT, Fischbeck KH; BVS857 study group. Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2018 Dec;17(12):1043-1052. doi: 10.1016/S1474-4422(18)30320-X. Epub 2018 Oct 15.
PubMed 30337273

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Two-part Placebo-controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
ClinicalTrials.gov ID: NCT02024932
Acronym / sponsor ID: CBVS857X2202
Conditions: Spinal and Bulbar Muscular Atrophy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 37 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 4 Feb 2014 (actual)
Primary completion: 13 Apr 2016 (actual)
Study completion: 13 Apr 2016 (actual)
First posted: 31 Dec 2013 (estimated)
Results: Posted 11 Aug 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 6

Arm	Type	Receives
BVS857 Part A Open label (Cohort 1)	Experimental	Drug: BVS857Participants received single doses of 0.01 mg/kg BVS857 intravenously (i.v.) on day 1, 0.01 mg/kg BVS857 subcutaneously (s.c.) on day 15, 0.03 mg/kg BVS857 s.c. on day 29, 0.06 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57.
BVS857 Part A double blind (Cohort 2)	Experimental	Drug: BVS857Participants received single doses of 0.03 mg/kg BVS857 i.v on day 1, 0.03 mg/kg BVS857 s.c. on day 15, 0.06 mg/kg BVS857 s.c. on day 29, 0.10 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. (BVS857 concentrations differed on days 43 and 57.)
Placebo Part A double blind (Cohort 2)	Placebo comparator	Drug: PlaceboParticipants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57.
BVS857 Part B open-label (Cohort 4)	Experimental	Drug: BVS857Participants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks.
BVS857 Part B double blind (Cohort 5)	Experimental	Drug: BVS857Participants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks.
Placebo Part B double blind (Cohort 5)	Placebo comparator	Drug: PlaceboParticipants received matching placebo i.v. to BVS857 weekly for 12 weeks.

Who may qualify

Ages: 18 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

Genetic diagnosis of SBMA with symptomatic muscle weakness
Able to complete 2 minute timed walk
Serum IGF-1 level less than or equal to 170 ng/mL

Key exclusion criteria (6 listed)

Medically treated diabetes mellitus or known history of hypoglycemia
History of Bell's palsy
Treatment with systemic steroids > 10 mg/day (or equivalent dose); androgens or androgen reducing agents; systemic beta agonists; or other muscle anabolic drugs within the previous 3 months
History of cancer, other than non-melanomatous skin cancer
Retinopathy

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BVS857BVS857 lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..	Drug	BVS857 Part A Open label (Cohort 1), BVS857 Part A double blind (Cohort 2), BVS857 Part B double blind (Cohort 5), BVS857 Part B open-label (Cohort 4)
PlaceboPlacebo lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..	Drug	Placebo Part A double blind (Cohort 2), Placebo Part B double blind (Cohort 5)

Primary outcome

Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability
Time frame: After 78 days in Part A and after 85 days in Part B.
Safety was monitored throughout the study.
Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability
Time frame: After 78 days in Part A and after 85 days in Part B.
Safety was monitored throughout the study.
Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5
Time frame: Baseline, Day 85
Thigh muscle volume was assessed by magnetic resonance imaging (MRI). Change from baseline was calculated from the ratio of the post-baseline mean value to the baseline mean value: [(Day 85/baseline) - 1)] x 100. A positive change from baseline indicates improvement.

21 secondary outcomes

Mean Change From Baseline in Score on the Adult Myopathy Assessment Tool (AMAT) in Part B, Cohort 5
Time frame: Baseline, Day 85
Mean Change From Baseline in Total Lean Body Mass (LBM) in Part B, Cohort 5
Time frame: Baseline, Day 85
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 1
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 2
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 1
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 2
Time frame: Day 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 1
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 2
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 1
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 2
Time frame: Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 4
Time frame: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 5
Time frame: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 4
Time frame: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 5
Time frame: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part B, Cohort 5
Time frame: Day 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 4
Time frame: Days 1: pre-dose, 1, 4, 24, 48 hours post-dose
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 5
Time frame: Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the End of the Dosing Interval Tau (AUCtau)
Time frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Terminal Elimination Half-life (T1/2)
Time frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to Infinity (AUCinf)
Time frame: Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose.
Compare Dose Normalized Log-transformed AUCinf Following IV and SC Administrations
Time frame: In Part A: days 1 and 15, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose.

Results and safety

Results posted to the registry on 11 Aug 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BVS857 Part A Open Label (Cohort 1)	2	0
BVS857 Part A Double Blind (Cohort 2)	4	1
Placebo Part A Double Blind (Cohort 2)	2	2
BVS857 Part B Open-label (Cohort 4)	2	0
BVS857 Part B Double Blind (Cohort 5)	18	16
Placebo Part B Double Blind (Cohort 5)	9	9

Primary outcome: Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability

Time frame: After 78 days in Part A and after 85 days in Part B.. Number. Unit: Participants

Measure	BVS857 Part A Open Label (Cohort 1) n = 2	BVS857 Part A Double Blind (Cohort 2) n = 4	Placebo Part A Double Blind (Cohort 2) n = 2	BVS857 Part B Open-label (Cohort 4) n = 2	BVS857 Part B Double Blind (Cohort 5) n = 18	Placebo Part B Double Blind (Cohort 5) n = 9
Non-serious AEs	2	4	2	1	17	8
SAEs	0	0	0	0	0	0
Deaths	0	0	0	0	0	0

Population analysed: The safety analysis set, which included participants who received any study drug, was analyzed.

Primary outcome: Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability

Time frame: After 78 days in Part A and after 85 days in Part B.. Number. Unit: Participants

Measure	BVS857 Part A Open Label (Cohort 1) n = 2	BVS857 Part A Double Blind (Cohort 2) n = 4	Placebo Part A Double Blind (Cohort 2) n = 2	BVS857 Part B Double Blind (Cohort 5) n = 18	Placebo Part B Double Blind (Cohort 5) n = 9
Mild	1	2	2	5	4
Moderate	1	2	0	11	3
Severe	0	0	0	1	1

Population analysed: The safety analysis set, which included participants who received any study drug, was analyzed.

Primary outcome: Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5

Time frame: Baseline, Day 85. Mean (Standard Deviation). Unit: Percent change

Measure	BVS857 Part B Double Blind (Cohort 5) n = 15	Placebo Part B Double Blind (Cohort 5) n = 9
Value	0.0 (2.42)	-3.4 (4.79)

BVS857 Part B Double Blind (Cohort 5) vs Placebo Part B Double Blind (Cohort 5): Geo-mean ratio: 1.037 (90% CI 1.009 to 1.065), p = 0.0164, ANCOVA

Population analysed: The PD analysis set was considered for the analysis. However, only participants who had evaluable data at both baseline and day 85, were included in the analysis. The PD set included participants with evaluable PD data who received any study drug and had no protocol deviations with relevant impact on PD data.

21 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BVS857 Part A Open Label (Cohort 1)	0 of 2 (0%)	2 of 2 (100%)	Not reported
Placebo Part A Double Blind (Cohort 2)	0 of 2 (0%)	2 of 2 (100%)	Not reported
BVS857 Part A Double Blind (Cohort 2)	0 of 4 (0%)	4 of 4 (100%)	Not reported
BVS857 Part B Open-label (Cohort 4)	0 of 2 (0%)	1 of 2 (50%)	Not reported
BVS857 Part B Double Blind (Cohort 5)	0 of 18 (0%)	17 of 18 (94.4%)	Not reported
Placebo Part B Double Blind (Cohort 5)	0 of 9 (0%)	8 of 9 (88.9%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 30337273Grunseich C, Miller R, Swan T, Glass DJ, El Mouelhi M, Fornaro M, Petricoul O, Vostiar I, Roubenoff R, Meriggioli MN, Kokkinis A, Guber RD, Budron MS, Vissing J, Soraru G, Mozaffar T, Ludolph A, Kissel JT, Fischbeck KH; BVS857 study group. Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2018 Dec;17(12):1043-1052. doi: 10.1016/S1474-4422(18)30320-X. Epub 2018 Oct 15.
A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

6 sites in 4 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BVS857 in people with Spinal and Bulbar Muscular Atrophy, run by Novartis Pharmaceuticals at 6 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, Denmark, Germany, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Aug 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability (time frame: After 78 days in Part A and after 85 days in Part B.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02024932, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.